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Company Directory

Use this Company Directory to identify investigational drugs, link to clinical trials, and search for expanded access policies, company contact information, and program listings.

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If you know the name of a drug manufacturer, you can easily search using the alphabetical menu below. Or you can search in free text – simply type the disease, drug, company name, or other detail into the Search bar below and click on the magnifying glass. For broadest results, type the terms without quotation marks; to narrow your search to an exact match, put your terms in quotation marks (i.e.; “breast cancer”). See Search Tips for tips and definitions to make your searches more effective.

The web links in this directory are public information provided to the Foundation and the acknowledgement times listed are the number of days or hours for a company to acknowledge receipt of request, not to make a decision. Data contained in this directory is provided either directly by the company or by ClinicalTrials.gov for the companies listed in this directory.

The Expanded Access listings provided on this website are limited to those companies currently in the Company Directory and include listings for expanded access which are Available, Temporarily Not Available and Approved for Marketing, which in some cases includes treatments which have not yet reached the market. In addition, many companies are willing to consider expanded access requests even if they do not show active Expanded Access Listings in this directory. Contact the company to explore the possibility of individual access to an investigational therapy.

To learn more about clinical trials and expanded access, please visit our patient and physician guides on this website.

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Company Name
Phone Number & Email
Company Acknowledgement

Abbvie

Phone Number & Email
2 business days

Expanded Access Listings

Conditions:
Multiple Myeloma
The objective of this expanded access program is to provide treatment with elotuzumab in combination with lenalidomide and dexamethasone for patients with relapsed or refractory multiple myeloma at Japanese sites where licensed physicians determine clinical need.
Conditions:
Primary Tauopathy
This is an expanded access program (EAP) for eligible participants. This program is designed to provide access to ABBV-8E12 prior to approval by the local regulatory agency. Availability will depend on territory eligibility. Participating sites will be added as they apply for and are approved for the EAP. A medical doctor must decide whether the potential benefit outweighs the risk of receiving an investigational therapy based on the individual patient's medical history and program eligibility criteria.
Conditions:
Glioblastoma or Solid Tumors, Epidermal Growth Factor Receptor (EGFR) Diagnosis
This is an expanded access program (EAP) for eligible participants. This program is designed to provide access to ABT-414 prior to approval by the local regulatory agency. Availability will depend on territory eligibility. Participating sites will be added as they apply for and are approved for the EAP. A medical doctor must decide whether the potential benefit outweighs the risk of receiving an investigational therapy based on the individual patient's medical history and program eligibility criteria.
Conditions:
Hepatitis C Virus Infection
This is an expanded access program (EAP) for eligible participants. This program is designed to provide access to glecaprevir/ pibrentasvir prior to approval by the local regulatory agency. Availability will depend on territory eligibility. Participating sites will be added as they apply for and are approved for the EAP. A medical doctor must decide whether the potential benefit outweighs the risk of receiving an investigational therapy based on the individual patient's medical history and program eligibility criteria.
Conditions:
Myelofibrosis, Acute Lymphocytic Leukemia (ALL), Lymphoblastic Lymphoma, Thyroid Cancer
This is an expanded access program (EAP) for eligible participants. This program is designed to provide access to navitoclax prior to approval by the local regulatory agency. Availability will depend on territory eligibility. Participating sites will be added as they apply for and are approved for the EAP. A medical doctor must decide whether the potential benefit outweighs the risk of receiving an investigational therapy based on the individual patient's medical history and program eligibility criteria.
Conditions:
Small Cell Lung Cancer
This is an expanded access program (EAP) for eligible participants. This program is designed to provide access to Rovalpituzumab Tesirine prior to approval by the local regulatory agency. Availability will depend on territory eligibility. Participating sites will be added as they apply for and are approved for the EAP. A medical doctor must decide whether the potential benefit outweighs the risk of receiving an investigational therapy based on the individual patient's medical history and program eligibility criteria.
Conditions:
Solid Tumors With Documented BRCA, BARD, or PALB or Other Acceptable DNA Mutations or Anomalies That Are Scientifically Sound, Triple Negative Breast Cancer (TNBC), High Grade Serous Ovarian Cancer
Expanded Access
Conditions:
Chronic Lymphocytic Leukemia (CLL), Multiple Myeloma, Acute Myeloid Leukemia (AML), Non-Hodgkin's Lymphoma, Acute Lymphoblastic Leukemia (ALL), Chronic Myelogenous Leukemia (CML), Amyloidosis
This is an expanded access program (EAP) for eligible participants. This program is designed to provide access to Venetoclax prior to approval by the local regulatory agency. Availability will depend on territory eligibility. Participating sites will be added as they apply for and are approved for the EAP. A medical doctor must decide whether the potential benefit outweighs the risk of receiving an investigational therapy based on the individual patient's medical history and program eligibility criteria.
Conditions:
Metastatic Breast Cancer With BRCA 1 or BRCA 2 Genetic Mutation, Triple-Negative Breast Cancer
This is an expanded access protocol to allow continued maintenance therapy with ABT-888 (veliparib) for three patients with metastatic triple negative breast cancer who are currently receiving the investigational product in association with clinical trial participation. Additionally, the protocol will enroll up to 7 new patients with metastatic BRCA associated or triple negative breast cancer to allow for additional access to veliparib monotherapy, or at the investigator's discretion, veliparib in combination with cisplatin and/or vinorelbine.

Alexion

Phone Number & Email
3 business days

Alkermes

2 weeks

Additional Information

Alnylam Pharmaceuticals

Phone Number & Email
3 business days

Additional Information

DISEASE/CATEGORY-SPECIFIC EA POLICIES/CRITERIA

http://www.alnylam.com/medical-professionals/expanded-access-program/

Amgen

3 business days

Expanded Access Listings

Conditions:
Pancreatic Cancer
The purpose of this study is to compare best supportive care plus oxaliplatin/ folinic acid/ 5-FU versus best supportive alone in patients with gemcitabine refractory pancreatic cancer.
Conditions:
Celiac Disease
Expanded access requests for AMG 714 may be considered for adult patients with biopsy proven Refractory Celiac Disease Type II who have failed all available treatment options and do not have EATL. To request access, use Responsible Party contact information provided in this record.
Conditions:
Multiple Myeloma
The purpose of this study is to expand upon the safety data for carfilzomib by providing expanded access to patients with relapsed and refractory multiple myeloma who are unable to enroll in any other ongoing carfilzomib trial.
Conditions:
Relapsed/Refractory B-Precursor Acute Lymphoblastic Leukemia
Primary Objective: To estimate the incidence of treatment-emergent and treatment-related adverse events during treatment with blinatumomab in pediatric and adolescent subjects with B-precursor ALL in second or later bone marrow relapse, in any marrow relapse after alloHSCT, or refractory to other treatments Secondary Objective(s): To describe key efficacy outcomes, including incidence of complete response (CR) within 2 cycles of blinatumomab, minimal residual disease (MRD) remission within 2 cycles of blinatumomab, relapse free survival (RFS), overall survival (OS), incidence of alloHSCT, and 100-day mortality after alloHSCT. Hypotheses: A formal statistical hypothesis will not be tested. The incidence of treatment-emergent and treatment-related adverse events will be estimated. Study Endpoints: - Incidence of treatment-emergent and treatment-related adverse events - Incidence of CR within 2 cycles of blinatumomab - MRD remission within 2 cycles of blinatumomab - RFS - OS - Incidence of alloHSCT - 100-day mortality after alloHSCT Study Design: Multi-center, open-label, single-arm expanded access protocol

AMICUS THERAPEUTICS

Phone Number & Email
3 business days

Expanded Access Listings

Conditions:
Pompe Disease
This is an expanded access program (EAP) for eligible participants designed to provide access to ATB200/AT2221.
Conditions:
Fabry Disease
This program allows physicians to request permission from Amicus Therapeutics (Amicus) for treatment access to migalastat hydrochloride (HCl) for specific adult patients with Fabry disease. Treatment is open label for 6 months with renewal every 6 months.

Additional Information

Available therapies via single-patient EA

Requests for Early Access cannot be made directly by an individual patient or a patient’s parent/legal guardian or caregiver. Patients and caregivers seeking general information may reach out to Amicus’ Global Patient & Professional Advocacy Department at patientadvocacy@amicusrx.com or call toll-free in the United States at 1-866-9AMICUS (1-866-926-4287), or +44-1753-888-567 for International inquiries.

Astellas Pharma US

7 days

Expanded Access Listings

Conditions:
Acute Myeloid Leukemia (AML), FMS-like Tyrosine Kinase-3 (FLT3) Mutations
The purpose of this study is to provide expanded access to ASP2215 for subjects with FLT3-mutated relapsed or refractory AML or FLT3-mutated AML in composite complete remission (CRc) (complete remission [CR], complete remission with incomplete hematologic recovery [CRi], complete remission with incomplete platelet recovery [CRp]) with MRD without access to comparable or alternative therapy.
Conditions:
Metastatic Castration-Resistant Prostate Cancer
The purpose of this treatment protocol is to provide expanded access to MDV3100 and monitor its safety in patients with metastatic castration-resistant prostate cancer previously treated with docetaxel-based chemotherapy.

AstraZeneca Pharmaceuticals LP

Immediate

Expanded Access Listings

Conditions:
Non Small Cell Lung Cancer, Cancer of the Head and Neck
The purpose of this study is to provide ZD1839 for those patients with locally advanced and/or metastatic non-operable non-small cell lung cancer (stage III or IV) or recurrent and/or metastatic squamous cell head and neck cancer who receive the therapy on an expanded access basis due to their inability to meet eligibility criteria for on-going recruiting trials, inability to participate in other clinical trials (e.g., poor performance status, lack of geographic proximity), or because other medical interventions are not considered appropriate or acceptable.
Conditions:
NF type1 With Inoperable Plexiform Neurofibromas
This will be an open-label, single-arm, multicenter intermediate access protocol which provides treatment access to selumetinib for eligible patients with neurofibromatosis type 1 (NF1) who have inoperable, progressive/symptomatic plexiform neurofibromas (PN) without any alternative therapeutic options. All patients will continue to receive drug while they are deriving clinical benefit . Approximately 100 patients in the US will be treated as part of this protocol
Conditions:
Ovarian Cancer
This is an open-label, single-arm, international, multicenter Multiple Patient Expanded Access Program (MPEAP). The program is designed to provide treatment access to olaparib tablets for patients with platinum-sensitive relapsed high-grade epithelial ovarian, fallopian tube or primary peritoneal cancer without other treatment options or eligible for an olaparib clinical trials.
Conditions:
Non Small Cell Lung Cancer (NSCLC)
The purpose of this study is to provide gefitinib treatment to patients who, on completion or closure of other gefitinib clinical studies, were either receiving placebo treatment, or are continuing on the same dose and regimen of gefitinib established in their preceding study, for as long as the patients continue to derive benefit.

Biogen

Phone Number & Email
1 business day

Expanded Access Listings

Conditions:
Infantile-onset Spinal Muscular Atrophy
To provide access to nusinersen to eligible patients with Infantile-onset Spinal Muscular Atrophy (SMA) (consistent with Type 1) to address a high-unmet medical need.

Biohaven Pharmaceutical Holding Company Ltd.

2 business days

Expanded Access Listings

Conditions:
Amyotrophic Lateral Sclerosis, ALS, Lou Gehrig Disease, Lou Gehrig's Disease, Lou-Gehrigs Disease, Motor Neuron Disease, Amyotrophic Lateral Sclerosis
This is an open label expanded access protocol for the treatment of up to approximately 250 adult patients with amyotrophic lateral sclerosis (ALS) who have difficulty swallowing oral riluzole tablets and may be able to derive benefit from treatment with an alternative oral formulation of riluzole.

Additional Information

Available therapies via single-patient EA

Requests for Early Access must be made by a physician on behalf of their patient. A licensed physician overseeing the patient’s care, who is able to comply with Biohaven’s requirements, may contact Biohaven at BHV0223.ExpandedAccess@earlyaccesscare.com or call toll-free in the United States at 1-888-315-5797 (Option 6).

Boehringer Ingelheim Pharmaceuticals

Phone Number & Email
5 business days

Expanded Access Listings

Conditions:
Carcinoma, Non-Small-Cell Lung
This is an open-label, multi-center, single-arm trial, designed to provide early access to afatinib and to provide additional information on the safety and efficacy of afatinib in advanced NSCLC patients who harbor an EGFR mutation.
Conditions:
Lung Diseases, Interstitial
This Expanded Access Program is intended to facilitate the availability of OFEV to patients suffering from non Idiopathic Pulmonary Fibrosis-Interstitial Lung Disease (non IPF-ILD) with a progressive clinical course despite Standard of Care treatment and for whom no satisfactory authorised alternative therapy exists or who cannot enter a clinical trial.
Conditions:
HIV Infections
The purpose of this Open Label Safety Study is to provide access to and evaluate the safety and tolerability of TPV/r in treatment-experienced patients with advanced HIV-1 infection who have failed at least two PI-containing regimens, and have limited treatment options.
Conditions:
Idiopathic Pulmonary Fibrosis
To provide early access and to evaluate the safety and tolerability of nintedanib in patients with idiopathic pulmonary fibrosis (IPF).
Conditions:
Carcinoma, Non-Small-Cell Lung
To provide expanded access and to evaluate the safety, tolerability and efficacy of afatinib in patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) harboring Epidermal Growth Factor Receptor (EGFR) mutation(s) and have never been treated with an EGFR tyrosine kinase inhibitor (TKI)
Conditions:
Carcinoma, Non-Small-Cell Lung
The program will provide early access to the investigational drug BIBW 2992 to treat patients with advanced NSCLC who have failed at least 12 weeks on erlotinib or gefitinib. The Compassionate Use Programme will also provide additional safety and efficacy information on BIBW 2992 use. Named Patient Use (NPU)
Conditions:
HIV Infections
This study was designed to provide early access to and evaluate the safety of TPV/r in PI-experienced patients with HIV-1 infection.
Conditions:
HIV Infections
To provide early access to tipranavir and evaluate the safety and tolerance of tipranavir combined with low dose of ritonavir in patients with progressive, HIV-1 disease who have failed or are intolerant to currently approved treatments for HIV infection, who are unable to participate in another tipranavir controlled clinical trial and have an urgent need for anti-HIV treatment.

Bristol-Myers Squibb

Phone Number & Email
48 hours

Expanded Access Listings

Conditions:
Renal Transplantation
To make belatacept available for recipients of a renal allograft who are currently intolerant to or have contraindications to CNIs and/or m-TOR inhibitors and are either: - unable to construct an adequate immunosuppression regimen due to non-renal toxicity / contraindication (and withdrawing the causative agent would lead to renal graft loss) OR - at imminent risk of losing the allograft kidney due to nephrotoxicity and have no other options for renal replacement therapy
Conditions:
Multiple Myeloma
The objective of this expanded access program is to provide treatment with elotuzumab in combination with lenalidomide and dexamethasone for patients with relapsed or refractory multiple myeloma at Japanese sites where licensed physicians determine clinical need.
Conditions:
Multiple Myeloma
At BMS, we work with physicians/investigators to make investigational products available to patients with life-threatening diseases that have exhausted other treatment options and where there is a reasonable expectation of benefit over risk. When contacted by a treating physician, BMS will consider requests for providing early patient access to Elotuzumab in patients with multiple myeloma who are residents of Belgium, Thailand, Turkey, Argentina, and Colombia.
Conditions:
Pediatric Cancer
At BMS, we work with physicians/investigators to make investigational products available to patients with life-threatening diseases that have exhausted other treatment options and where there is a reasonable expectation of benefit over risk. When contacted by a treating physician, BMS will consider requests for providing early patient access to Nivolumab in pediatric patients exhibiting a high mutational load.
Conditions:
Multiple Myeloma
The objective of this expanded access program is to provide treatment with elotuzumab in combination with lenalidomide and dexamethasone for patients with relapsed or refractory multiple myeloma at U.S. sites where licensed physicians determine clinical need.
Conditions:
Leukemia
This protocol allows CML and Ph+ ALL subjects who are resistant to or intolerant of imatinib mesylate, to potentially benefit from dasatinib. It is intended to provide patients with access to dasatinib while awaiting reimbursement decision in Korea and it will also provide additional data on the safety of dasatinib in Korean population

Celgene

Phone Number & Email
24 hours

Expanded Access Listings

Conditions:
Myelodysplastic Syndrome
This is an expanded access program (EAP) for eligible participants designed to provide access to ACE-011.
Conditions:
Myelodysplastic Syndrome (MDS)
This is an expanded access program (EAP) for eligible participants designed to provide access to CC-486.
Conditions:
Myelofibrosis
This is an expanded access program (EAP) for eligible participants designed to provide access to Fedratinib.
Conditions:
Multiple Myeloma
To provide pomalidomide access to relapsed/refractory multiple myeloma subjects with a likelihood of benefit from the pomalidomide treatment while the medication is not commercially available

Celldex Therapeutics

5 business days

Additional Information

SINGLE-PATIENT EA POLICIES/CRITERIA

None

DISEASE/CATEGORY-SPECIFIC EA POLICIES/CRITERIA

https://celldex.com/docs/Compassionate_Use_Policy_MAY2018.pdf

Daiichi Sankyo

5 business days

Expanded Access Listings

Conditions:
Acute Myeloid Leukemia With Gene Mutations
Quizartinib is an experimental product being developed by Daiichi Sankyo that has not been approved yet by the FDA for relapsed or refractory FLT3-ITD mutated AML. The main objective of this program is to provide treatment with quizartinib as monotherapy to patients with relapsed or refractory FLT3-ITD mutated AML who are not eligible to participate in our current clinical trials. Expanded access treatment with quizartinib as monotherapy is currently available in the United States only, and will consist of 28-day continuous cycles. However, treatment may be placed on hold or permanently discontinued by the Sponsor at any point for reasons not limited to drug supply shortage, marketing authorization decline, or safety concerns.

Additional Information

Available therapies via single-patient EA

At Daiichi Sankyo, we recognize that there are instances when a patient has a serious or life-threatening disease or condition, for which all currently available treatment options have been exhausted and enrollment into a clinical trial is not possible. In these cases, in particular, a treating physician can request the use of an investigational Daiichi Sankyo product prior to regulatory approval for the particular condition or indication, provided it is allowed by the applicable local laws.

Daiichi Sankyo strives for an equitable balance between the public interest in securing the approval of a new drug and allowing access to certain investigational medicinal products that may have the potential to treat seriously ill patients who cannot be satisfactorily treated with commercially available products. The following criteria are typically required before Daiichi Sankyo would consider patients eligible for expanded access to a Daiichi Sankyo investigational product. All criteria are subject to local laws and regulations:
--The patient suffers from a life threatening or serious disease or condition, where there is an unmet medical need that cannot be met by existing products.
--The patient is ineligible or unable to participate in any ongoing clinical study of the investigational product.
--The investigational product is under active development (a dose and regimen has been defined and the product is typically in Phase 2 development or beyond) and has not been placed on regulatory or any other hold.
--The benefit-risk profile of the drug in the indication and the patient population is deemed, to date, to be positive.
--Daiichi Sankyo has decided to seek marketing approval in at least one major market globally.
--The expanded access use of the investigational product will not jeopardize the development of the drug (for instance, granting access to an investigational unapproved drug through expanded access should not delay, interfere with, or compromise the completion of clinical trials that are intended to support approval by regulatory authorities).
--The use of the investigational product in an expanded access protocol is compliant with local rules and regulations.

Daiichi Sankyo cannot approve expanded access use requests in cases where doing so would violate company policy, local laws or regulations, or if access would present an unacceptable benefit/risk profile, as deemed by medical personnel of the company. We commit to a careful and fair evaluation of each request by the appropriate medical experts at Daiichi Sankyo within the boundaries of local laws and regulations.

SINGLE-PATIENT EA POLICIES/CRITERIA

We have a formal program for quizartinib (relapsed/refractory FLT3-ITD AML)

Daiichi Sankyo does not currently have formal expanded access programs for our other investigational products. We consider granting expanded access to products other than quizartinib on a case-by-case basis, as long as such provision will not delay, interfere with or compromise the completion of clinical trials that are intended to support approval by regulatory authorities, which, in turn, provides access to the medication for the greatest number of individuals.

DISEASE/CATEGORY-SPECIFIC EA POLICIES/CRITERIA

https://www.daiichisankyo.com/rd/pipeline/index.html

Eisai

Phone Number & Email
2 business days

Expanded Access Listings

Conditions:
Differentiated Thyroid Cancer
This Expanded Access Program (EAP) consists of a Prerandomization Phase and a Randomization Phase. Only subjects with radioiodine-refractory DTC who fulfill the eligibility criteria will be treated. These subjects will be treated until progression of disease or unacceptable toxicity.
Conditions:
Lennox Gastaut Syndrome
This is an extended access study for participants who have completed Rufinamide Study E2080-G000-303 to continue to have access to rufinamide until it becomes commercially available in Poland or until no participants remain in the EAP.
Conditions:
Primary Generalized Tonic-Clonic or Partial Onset Seizures
The main objective of this EAP is to ensure that participants participating in Study E2007-G000-332, Study 311 or EAP 401 continue to have access to perampanel until such time that the appropriate formulation of perampanel becomes commercially available in the country in which they reside or until no participants remain in the EAP.
Conditions:
Partial Onset Seizures
The Extended Access Program (EAP) is a managed access programme for Perampanel. The main objective of this EAP is to ensure that patients participating in studies E2007-A001-207, E2007-G000-307, or E2007-G000-235 continue to have access to perampanel until such time perampanel tablets become commercially available for the treatment of Partial Onset Seizures (POS) in the country in which they reside. This EAP will consist of 2 phases: - Screening: The patient will start the program once the Screening assessments are completed and the patient is qualified for participation. - Treatment: Additional assessments, physical examinations, and dosage changes will be clinically determined by the treating physician. Patients will enter this program on the same dose of perampanel that they were receiving at the end of their participation in previous study. Doses of perampanel and of concomitant anti-epileptic drugs (AEDs) can be adjusted (i.e., added,removed, or changed in dose) based on clinical judgment. Treatment will be prescribed as long as clinically appropriate according to the judgement of the treating physician and the approved Summary of Product Characteristics (SmPC). The program will complete in a staggered fashion, country by country, as and when perampanel becomes commercially available for the treatment of POS in each country.
Conditions:
Metastatic Breast Cancer
This expanded access program is an open-label, multi-center study, which will consist of a PreTreatment Phase and a Treatment Phase. Patients with locally advanced or metastatic breast cancer who fulfill the eligibility criteria will be treated. The program will continue in each country until eribulin is approved, reimbursed and launched in that country, or termination of the program by the Sponsor.
Conditions:
Thyroid Cancer
This is an Expanded Access Program to make lenvatinib available to participants with radioiodine-refractory differentiated thyroid cancer in Brazil. Participants who have no other treatment options available, and who, in the opinion and clinical judgment of the treating physician, would benefit from treatment with lenvatinib will be enrolled. This is a multicenter, open-label program consisting of 2 phases: a 28-day pretreatment phase (including screening) and a treatment phase. Treatment will be provided as long as there is a clinical benefit based on tumor assessments performed according to the center's standard of care and the judgment of the participant's treating physician.

Eli Lilly

Phone Number & Email
5 business days

Expanded Access Listings

Conditions:
Mesothelioma
This study is to evaluate the effects (good and bad) of ALIMTA and Cisplatin or ALIMTA alone on you and your malignant pleural mesothelioma as well as make ALIMTA available to patients who qualify for treatment.
Conditions:
Metastatic Breast Cancer
The treating physician/investigator contacts Lilly when, based on their medical opinion, a patient meets the criteria for inclusion in the expanded access program.
Conditions:
Cancer
This study is for patients who have participated in a previous study and who continue to receive benefit to have continued access to study drug and/or treatment.
Conditions:
Metastatic Breast Cancer
The treating physician/investigator contacts Lilly when, based on their medical opinion, a patient meets the criteria for inclusion in the expanded access program.

EMD Serono

Phone Number & Email
5 business days

Genentech

Phone Number & Email
3 business days

Expanded Access Listings

Conditions:
Basal Cell Carcinoma
This is an open-label, non-comparative, multicenter, expanded access study of Vismodegib (GDC-0449) in patients with locally advanced basal cell carcinoma (BCC) or metastatic BCC (mBCC) who are otherwise without satisfactory treatment options.
Conditions:
Hemophilia A
This open-label, multicenter expanded access program (EAP) is designed to provide emicizumab to eligible participants with hemophilia A with factor VIII (FVIII) inhibitors before it is commercially available in the United States for the indication of hemophilia A with FVIII inhibitors. Discontinuation may occur earlier if participant or physician decides to discontinue treatment or the sponsor discontinues emicizumab clinical development.
Conditions:
Urothelial Carcinoma
This is an open-label, multicenter, single-arm, expanded access program (EAP) designed to provide atezolizumab access to participants with locally advanced or metastatic urothelial carcinoma that has progressed on, or is intolerant to, a platinum-containing chemotherapy regimen.
Conditions:
Idiopathic Pulmonary Fibrosis
This is an open label multi-center program to allow patients in the US with IPF access to treatment with pirfenidone.

Additional Information

Available therapies via single-patient EA

https://www.gene.com/patients/investigational-medicines/criteria

GlaxoSmithKline

24 hours

Expanded Access Listings

Conditions:
Multiple Myeloma
Compassionate use access to GSK2857916 for eligible participants with refractory/relapsing multiple myeloma
Conditions:
Solid Tumours
Compassionate use access to molibresib/GSK525762 for eligible participant with NUT Midline Carcinoma; indication is a seriously debilitating or life-threatening disease.
Conditions:
Hypereosinophilic Syndrome
104317: The market authorisation application for mepolizumab for the indication of hypereosinophilic syndrome (HES) was filed in 2008, but later the file was withdrawn due to outstanding questions from regulator's raised from the application. On the basis of sponsor's evaluation, participants with life-threatening HES who have documented failure (lack of efficacy or a contra-indication) to at least 3 standard HES therapies (compassionate use) and participants who have participated in a previous GSK sponsored study in HES (long-term access) can be consider for mepolizumab treatment where the country regulation permits. In this study, participants will receive mepolizumab in an open-labelled manner, and limited data will be collected to evaluate the long-term safety and efficacy of mepolizumab. 201956: This is a Long-term Access Programme (LAP) which aims to support provision of mepolizumab, until it is commercially available, to eligible subjects with severe asthma who participated in a GSK-sponsored mepolizumab clinical study in severe asthma. Eligible subjects will initiate mepolizumab within a 6-month period following the individual subject's last scheduled visit in their preceding clinical study. For each subject benefit versus risk will be assessed throughout the study to support continued treatment with mepolizumab. 112562: To provide a mechanism for expanded access to mepolizumab therapy for eligible patients with HES. Whenever possible, use of an investigational medicinal product by a patient as part of a clinical trial is preferable. However, when patient enrollment in a clinical trial is not possible (such as when the patient is not eligible for ongoing clinical trials or the patient is not able to attend investigational sites), appropriate patients may receive mepolizumab through expanded access. This expanded access protocol was designed to allow access to mepolizumab for HES patients with seriously debilitating or life-threatening disease that are not able to enroll in clinical trials, including those patients that have already participated in a mepolizumab clinical trial.

Additional Information

Available therapies via single-patient EA

http://www.gsk.com/media/3368/compassionate-use.pdf

Go to GSK Compassionate Use Request Portal at https://gsk-cu-portal.idea-point.com/ to make a request.

Immix Biopharma

Phone Number & Email
24hr to 48hr

Additional Information

Available therapies via single-patient EA

http://immixbio.com/pipeline/compassionate-use-policy/

SINGLE-PATIENT EA POLICIES/CRITERIA

imx-110 - all advanced solid tumors

Janssen

Immediate

Expanded Access Listings

Conditions:
Crohn Disease
The purpose of this program is to provide early access to ustekinumab where it is commercially unavailable for the treatment of participants with moderately to severely active Crohn's disease who have failed treatment with conventional Crohn's disease therapy (example, immunomodulators or corticosteroids) and Tumor Necrosis Factor alpha (TNF alpha) antagonist therapy (e.g., infliximab, adalimumab, certolizumab pegol, or their biosimilars), or who are intolerant to, or have a contraindication to these treatments. During the course of this early access program (EAP), through the reporting of serious adverse events (SAEs) and non-serious adverse drug reactions (ADRs) by participating physicians, information on the safety and tolerability of ustekinumab will be captured.
Conditions:
Relapsed or Refractory Mantle Cell Lymphoma
The purpose of this study is to provide patients who have relapsed or refractory Mantle Cell Lymphoma (MCL) with early access to an investigational medication called ibrutinib (PCI-32765) and to collect safety information about the drug.
Conditions:
Tuberculosis
The purpose of this is a study to provide early access of TMC207 to patients with pulmonary infection due to strains of Mycobacterium tuberculosis (M. tuberculosis) with resistance to isoniazid (INH), rifampin (RMP), and to a fluoroquinolone (FQ) and/or injectable second line tuberculosis (TB) drug (kanamycin, amikacin, or capreomycin) and who are unable/ineligible to participate in any other TMC207 study. In addition, information on safety and tolerability of TMC207 in combination with anti-TB drugs will be assessed and the results of microbiology assessments which are recommended to be performed during the early access study will be collected.
Conditions:
Multiple Myeloma
The objective of this study is to provide early access to daratumumab treatment and collect additional safety data while the medication is not commercially available or available through another protocol for subjects with multiple myeloma who have received at least 3 prior lines of therapy including a proteasome inhibitor (PI) and an immunomodulatory agent (IMiD) or whose disease is double refractory to both a PI and an IMiD.
Conditions:
Advanced Cancers and FGFR Genetic Alterations
The purpose of this program is to provide participants an early access to erdafitinib prior to market authorization (that is, Food and Drug Administration [FDA] approval in the United States). The program is limited to participants with advanced cancers and fibroblast growth factor receptor (FGFR) genetic alterations who have exhausted at least 2 lines of standard of care therapy and who are not eligible for an erdafitinib clinical trial.
Conditions:
Depressive Disorder, Treatment-Resistant
This is a pre-approval access program (PAAP) for eligible participants. The main purpose of this program is to provide access to esketamine nasal spray to eligible participant with treatment-resistant depression (TRD), who have exhausted all other treatment options, including all alternative treatment options with marketed therapies.
Conditions:
H7N9 Subtype of Influenza A Virus
The purpose of this program is to provide pre-approval access to pimodivir for the treatment of a patient(s) with H7N9 influenza A infection. Pre-approval access pertains to provision for therapeutic use of an investigational product prior to its marketing authorization. Such access may be considered for eligible patients with serious/life-threatening diseases or conditions, where alternative treatments do not exist or have been exhausted.
Conditions:
HIV
The main purpose of this pre-approval access program (PAAP) is to provide rilpivirine Long Acting (RPV LA) injectable suspension for the treatment of human immunodeficiency virus (HIV)-1 infection as single patient request to individuals who have no available treatment alternatives and/or limited treatment options (for example, who are unable to participate in the Phase III clinical studies or do not qualify). RPV LA can only be provided if participants are also eligible for cabotegravir Long Acting injections.

Additional Information

Johnson & Johnson

N/A

Kyowa Kirin Pharmaceutical Development, Inc.

Phone Number & Email
N/A

Expanded Access Listings

Conditions:
X-linked Hypophosphatemia, Tumor-Induced Osteomalacia
Individual patient expanded access requests may be considered for patients who have no other treatment options

Additional Information

Available therapies via single-patient EA

Poteligeo was approved by the FDA on August 8, 2018 for the treatment of adult patients with relapsed or refractory mycosis fungoides (MF) or Sézary syndrome (SS) after at least one prior systemic therapy. Only patients who have relapsed or refractory MF or SS after at least one prior systemic therapy will be eligible to participate.
The main purpose of the EA program is to facilitate the access of Poteligeo to patients who lack therapeutic alternatives and in which there is a reasonable expectation that the provision of access to Poteligeo will have a positive benefit/risk for the patient. The intention was for the EA program to fill the time between approval by the FDA and the date of availability of commercial supply to patients. Commercial supply is now available and the EA program ended on November 1, 2018.

Lundbeck

Phone Number & Email
10 business days

Melinta Therapeutics, Inc.

Phone Number & Email
Within 3 business days after receipt

Additional Information

Available therapies via single-patient EA

Consistent with Melinta Therapeutics’s mission to discover, develop, and commercialize a continual stream of novel antibiotics, we are focused on enrolling and conducting the clinical trials necessary to gain regulatory approvals and promptly make our medicines available to patients. At Melinta Therapeutics, we believe that wherever possible, the use of an investigational medicine for a patient as part of a clinical trial is preferable; however, when patients with an unmet medical need are unable to access our investigational products through a clinical trial and all other options have been exhausted, we may be able to provide access through our compassionate use or expanded access program.

The evaluation criteria to determine if Melinta Therapeutics can provide expanded access to a specific Melinta Therapeutics investigational agent outside of a clinical trial include all of the following:
• The patient has a serious or life-threatening illness or condition and is either no longer responsive to or no longer able to tolerate any available treatment option;
• The investigational drug is in active clinical development with sufficient data available to determine an appropriate dose and schedule for the patient’s specific condition;
• A benefit-risk analysis, based on both the available clinical data as well as the requesting physician’s assessment of the individual patient’s condition and history, supports making the investigational drug available;
• Access is compliant with local rules and laws;
• Making the investigational drug available will not negatively impact or delay the conduct of clinical trials or regulatory review or approval of the investigational drug for broader patient access; and
• Adequate supply of the investigational drug is available.

All requests must be submitted by the patient’s treating physician and Melinta Therapeutics may require more detailed information in order to fully evaluate a request. The treating physician must agree to obtain appropriate regulatory and ethics committee approvals and comply with regulatory obligations, including obtaining patient consent, patient monitoring and safety reporting. Treating physicians seeking expanded access, meeting the above-mentioned evaluation criteria, should submit their requests to medinfo@melinta.com. We regularly monitor this mailbox and will use our best efforts to acknowledge each submitted request within 3 business days after receipt. Melinta Therapeutics is committed to carefully evaluating requests on a case-by-case basis and in a fair and equitable manner; however, Melinta Therapeutics cannot guarantee access to any specific investigational drug by any individual patient.

If you are a patient with questions concerning access to our investigational products, either through a clinical trial or through expanded access, please consult with your treating physician.

SINGLE-PATIENT EA POLICIES/CRITERIA

fusidic acid (CEM-102)

Merck & Co.

Phone Number & Email
N/A

Expanded Access Listings

Conditions:
HIV Infections
This is a treatment use study to provide early access to MK0518 for the treatment of HIV-1 infection in patients who have limited or no treatment options due to virological failure, resistance, or intolerance to multiple antiretroviral regimens. Enrollment in this study is patient driven. Investigators are not proactively assigned. There is no target sample size and duration of the study is indefinite. For information on how to enroll in the study, see link below.
Conditions:
Mycoses
The purpose of this study is to provide posaconazole compassionate treatment to patients with invasive fungal infections: 1) which are resistant to standard antifungal therapies; 2) for which there are no effective therapies; 3) with a prior history of serious, severe, or life-threatening toxicities while receiving standard antifungal therapies, or 4) with pre-existing organ dysfunction which precludes the use of standard antifungal therapies.

Millendo Therapeutics (US), Inc.

15 days

Additional Information

SINGLE-PATIENT EA POLICIES/CRITERIA

Currently, none.

Novartis Pharmaceuticals

Phone Number & Email
Immediately

Expanded Access Listings

Conditions:
Multiple Myeloma
The purpose of this study is to provide oral panobinostat (PAN) treatment to relapsed or relapsed and refractory multiple myeloma patients who are without satisfactory treatment alternatives prior to the commercial availability* and reimbursement of panobinostat during the regulatory approval process. This protocol will acquire additional safety data on the use of panobinostat in combination with bortezomib (BTZ) and dexamethasone (Dex) in patients with relapsed or relapsed and refractory multiple myeloma. In this protocol, PAN must be administered in the defined regimen in combination with both BTZ and DEX. *(Note: throughout this protocol "commercially available" means local health authority approval and a functional method for reimbursement)
Conditions:
Acromegaly
The present study is planned as an expanded treatment protocol to provide acromegalic patients for whom medical therapy is appropriate access to pasireotide LAR while regulatory approval for pasireotide is sought.
Conditions:
Acute Lymphoblastic Leukemia (ALL), Diffuse Large B-cell Lymphoma (DLBCL)
Managed Access Program (MAP) to provide access to CTL019, for acute lymphoblastic leukemia (ALL) or diffuse large b-cell lymphoma (DLBCL) patients with out of specification leukapheresis product and/or manufactured tisagenlecleucel out of specification for commercial release.
Conditions:
Neoplasms, Breast
This study will provide pre-approval drug access to lapatinib, in combination with capecitabine, to patients whose breast cancer had progressed on other therapies
Conditions:
Acute Lymphoblastic Leukemia
This is a single arm, open-label, multi-center, phase II study to determine the safety and efficacy of CTL019 in pediatric/young adult patients with r/r B-cell ALL.
Conditions:
Non-small Cell Lung Cancer (NSCLC)
Novartis-sponsored, open-label, multi-center, interventional ETP to provide LDK378 to patients with ALK (+)NSCLC, who have been pre-treated with an ALK inhibitor; except in countries where ALK inhibitors are not approved or available. The protocol will further evaluate the safety of LDK378 in patients with ALK(+) NSCLC.
Conditions:
HR+ Advanced or Metastatic Breast Cancer
The purpose of this Cohort Treatment Plan is to allow access to alpelisib for eligible patients diagnosed with hormone receptor positive, advanced or metastatic breast cancer harboring a phosphatidylinositol 3-kinase (PI3K)CA mutation. The patient's Treating Physician should follow the suggested treatment guidelines and comply with all local health authority regulations.
Conditions:
Sickle Cell Disease
The purpose of this Cohort Treatment Plan is to allow access to crizanlizumab (SEG101) for eligible patients diagnosed with sickle cell disease (SCD) to prevent or reduce the frequency of vaso-occlusive crises (VOC). The patient's Treating Physician should follow the suggested treatment guidelines and comply with all local health authority regulations.
Conditions:
Acute Myeloid Leukemia (AML) With, FLT3 Mutation, Internal Tandem Duplication (ITD) or Tyrosine Kinase Domain (TKD)
The purpose of this study is to provide access to Midostaurin and gather additional safety data on the combination of Midostaurin and standard of care for adult patients with newly diagnosed Fms-like tyrosine kinase receptor (FLT3) mutated Acute Myeloid Leukemia (AML) who are eligible for standard induction and consolidation chemotherapy.
Conditions:
Heart Failure With Reduced Ejection Fraction (HF-rEF)
Novartis has set up this global Multiple Patient Program (MPP) treatment plan to provide access to life-saving treatment with LCZ696 for patients that were not previously exposed to LCZ696 but have no other option to receive LCZ696 in their country prior to market authorization OR commercial availability, based on local regulatory and legal requirements.
Conditions:
Chronic Myeloid Leukemia
Multi-center, open-label, non-randomized trial to evaluate long-term safety and efficacy of nilotinib. Approximately 20 patients will be enrolled in this trial at 3 centers in Mexico, which means all ongoing patients participating on [CAMN107A2109] excluding discontinued patients. During this study, patients will receive nilotinib orally, at a dose of 400 mg b.i.d. Patients will normally receive nilotinib on an outpatient basis. This trial will have a maximum of 24 months of follow-up time.

Additional Information

SINGLE-PATIENT EA POLICIES/CRITERIA

Alpelisib - PIK3CA-Related Overgrowth Spectrum (PROS)
Capmatinib - MET mutated non-small cell lung cancer (NSCLC) and MET amplified NSCLC
Ceritinib - All ALK+ cancers
Clofazim – Pulmonary mycobacterium abscessus infection
Crizanlizumab - Sickle Cell Disease (SCD)
Dabrafenib/Trametinib - metastatic or unresectable melanoma, adjuvant (resected) melanoma, non-small cell lung cancer (NSCLC)
Eltrombopag – Chronic ITP, Congenital thrombocytopenia, Severe Aplastic Anemia, HCV patients requiring treatment with an interferon based therapy, or patients with low-and-int-1 MDS with severe thrombocytopenia
Everolimus – mTOR inhibition in Exploratory Development & special project
Lapatinib – HER2+ breast cancer
Nilotinib – CML
Panobinostat – Multiple Myeloma
Pasireotide – Exploratory/Tumor
Ribociclib – CDK4/6inhibitor

Novo Nordisk

Phone Number & Email
5 business days

Oncoceutics, Inc.

Receipt will generally be acknowledged within 48 hours

Expanded Access Listings

Conditions:
Glioma
The objective of this expanded access program is to provide ONC201 to eligible patients with previously-treated glioma that exhibits the H3 K27M mutation and/or that is located in the midline region of the brain.

Additional Information

Available therapies via single-patient EA

Oncoceutics will consider single - patient expanded access for patients who meet the criteria for the company's existing expanded access program, as outlined on clinicaltrials.gov

SINGLE-PATIENT EA POLICIES/CRITERIA

ONC201 - H3 K27M mutant gliomas

Otsuka America Pharmaceutical

Phone Number & Email
1 week

Pfizer

Phone Number & Email
5 business days

Expanded Access Listings

Conditions:
Gastrointestinal Neoplasm
The purpose of this study is to permit access to SU011248 for treatment use by patients with GIST given the following conditions: a) patients undergo screening, but are not eligible for participation in ongoing clinical studies such as A6181004; AND b) patients have GIST which standard treatments have not been able to control with acceptable toxicity AND c) patients have the potential to derive clinical benefit from treatment with SU011248.
Conditions:
Neoplasm
This is an open label expanded access protocol for the treatment of up to approximately 40 adult or pediatric (defined as age <18 years) patients with tumors harboring either a chromosomal translocation or activating mutation involving the ALK or ROS1 gene or an activating genetic alteration involving the cMET gene who cannot swallow the crizotinib capsule but may be able to derive benefit from treatment with an alternative oral formulation of crizotinib.
Conditions:
Metastatic Merkel Cell Carcinoma
Expanded access to Avelumab solution for infusion will be made available for adult patients with mMCC whose disease has progressed after receiving at least one prior chemotherapy.
Information relating to requesting compassionate access for this investigational drug can be found at the following link: http://www.pfizer.com/research/compassionate_use
Information relating to requesting compassionate access for this investigational drug can be found at the following link: http://www.pfizer.com/research/compassionate_use
Conditions:
Pulmonary Arterial Hypertension
The purpose of this study is to provide Sildenafil therapy for subjects who completed A1481156 study and are judged by the investigator to derive clinical benefit from continued treatment with Sildenafil, prior to reimbursement and availability for subjects in the Russian Federation.
Conditions:
Carcinoma, Renal Cell
The primary objective of this protocol is to provide access to SU011248 treatment for patients with metastatic RCC who are ineligible for participation in ongoing SU011248 clinical studies and have the potential to derive clinical benefit from treatment with SU011248 based on the judgment of the investigator.

Additional Information

Available therapies via single-patient EA

www.pfizercares.com

Pharnext SAS

NA

Additional Information

Available therapies via single-patient EA

ACCESS TO PHARNEXT INVESTIGATIONAL MEDICINES PRIOR TO REGULATORY APPROVAL
(FOR US PATIENTS ONLY)
Pharnext develops innovative new medicines designed to address unmet medical needs of patients in major therapeutic areas. We are focused on creating new medicines that address important, real world concerns of patients and their families. We currently have investigational treatments in clinical trials for a number of different diseases, as described in the PRODUCTS section of our website at https://www.pharnext.com/en/products/r-d-pipeline
Pharnext’s goal is to efficiently bring safe and effective medicines to all appropriate patients by conducting rigorous clinical trials and obtaining marketing approval by regulatory authorities, including the U.S. Food and Drug Administration (FDA). Prior to such approval, participation in one of our clinical trials is the optimal way for patients to gain access to our investigational medicines. However, we understand that some patients may not be able to participate in a clinical trial and may seek access to investigational treatments before they are approved. These situations are often referred to in various ways, including expanded access, early access, pre-approval access, compassionate use and emergency use.
If early data from clinical trials suggest that an investigational treatment can offer benefits for patients facing serious or life-threatening conditions, Pharnext may consider requests to provide access outside of a clinical trial. Our top priority when evaluating requests for expanded access to investigational medicines is to ensure that we are not putting patients at risk of unnecessary harm.
Pharnext will consider an expanded access program, or a single request for expanded access of an investigational medicine, only if all of the following criteria are met:
• The patient’s licensed physician submits an unsolicited request for expanded access
• The patient to be treated has a serious or immediately life-threatening illness and there is no satisfactory alternative therapy
• There are no comparable or satisfactory alternative therapies approved and available to treat the disease or condition
• There is preliminary clinical evidence of effectiveness and acceptable safety of the investigational treatment for that patient’s condition
• The potential benefit of the investigational medicine to the patients is greater than the potential risk
• Staff and other required resources are available to provide the product and there is an adequate supply of the investigational medicine
• Providing the investigational medicine will not interfere with the timely completion of clinical trials that could lead to marketing approval and thereby could delay its availability to other patients
• Expanded access will only occur in a country where Pharnext expects to file for regulatory approval of the product
Patients interested in obtaining access to a Pharnext investigational therapy must do so via their physician. Physicians should contact Pharnext Medical Information Center at medical@pharnext.com. All requests will be carefully considered, and a written or oral response will be provided to the treating physician within two weeks of receiving a request. For further information on available expanded access programs, visit www.clinicaltrials.gov and search ‘expanded access programs’.

SINGLE-PATIENT EA POLICIES/CRITERIA

NA

DISEASE/CATEGORY-SPECIFIC EA POLICIES/CRITERIA

NA - see above

Purdue Pharma L.P.

Phone Number & Email
N/A

Retrophin

7 business days

Sanofi

Phone Number & Email
1 business day

SCYNEXIS, Inc.

Phone Number & Email
SCYNEXIS will acknowledge a contact for requesting Expanded Access within 24 hours.

Additional Information

Available therapies via single-patient EA

SCYNEXIS is committed to making Investigational Products available to seriously ill patients who have exhausted other treatment options. A treating physician, who is able to comply with the SCYNEXIS requirements , may request information about how to apply for access to SCYNEXIS’ Investigational Products by contacting the company.

SINGLE-PATIENT EA POLICIES/CRITERIA

SCY-078 is a triterpenoid, glucan synthase inhibitor, antifungal agent undergoing investigation for:
• The treatment of invasive candidiasis, including candidemia (IC).
• The treatment of invasive aspergillosis (IA), including IA that is refractory to or intolerant of other antifungal therapies.
• The treatment of invasive candidiasis in patients who are refractory to or intolerant of other antifungal therapies.

Shire

Phone Number & Email
3 business days

Takeda Pharmaceuticals

Phone Number & Email
3 business days

Additional Information

DISEASE/CATEGORY-SPECIFIC EA POLICIES/CRITERIA

http://takedaoncology.com/medicines/access-to-investigational-medicines

UCB

Phone Number & Email
N/A

Expanded Access Listings

Conditions:
Epilepsy
This Compassionate Use Program (CUP) is setup to provide study patients with continuous access to Brivaracetam (BRV) for the time period between closures of the long-term follow up studies N01125, N01199, N01372, N01379, and N01315 and when BRV is commercially available.
Conditions:
Epilepsies, Partial
The objective of this Compassionate Use Program (CUP) is to provide continued access to Lacosamide (LCM) for monotherapy use for patients who were receiving LCM in SP0993 and SP0994 at the time of study unblinding and close of SP0994, and who benefited from the treatment per investigator assessment.
Conditions:
Crohn's Disease
The objective of this program is to allow treating physicians to supply/continue to supply Certolizumab Pegol (CIMZIA®, CZP) to adults suffering from Crohn's Disease (CD), and who are considered not suitable for treatment, intolerant, have medical contraindications or had insufficient response with an authorized conventional therapy, including other authorized biologics.
Conditions:
Rheumatoid Arthritis
The objective of this Named Patient Program (NPP) is to provide continued availability of Certolizumab Pegol (CZP) to adult Rheumatoid Arthritis (RA) patients who participated in the open label studies C87015 (CDP870 015), C87028 (CDP870-028) and C87051 (CDP870-051). Physicians may use the option to continue offering patients CZP treatment or to transition patients off CZP to a standard care regimen.

Additional Information

Ultragenyx

Phone Number & Email
N/A

Expanded Access Listings

Conditions:
Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency, Carnitine Palmitoyltransferase Deficiencies (CPT1, CPT2), Mitochondrial Trifunctional Protein Deficiency, Long-chain Hydroxyacyl-CoA Dehydrogenase Deficiency, Glycogen Storage Disorders, Pyruvate Carboxylase Deficiency Disease, ACYL-CoA DEHYDROGENASE FAMILY, MEMBER 9, DEFICIENCY of, Barth Syndrome
This is a compassionate use study to allow patients already taking triheptanoin (C7) through previous studies to continue to receive the supplement. It will also allow triheptanoin supplementation in patients with qualifying disorders if they are failing conventional therapy.
Conditions:
X-linked Hypophosphatemia, Tumor-Induced Osteomalacia
Individual patient expanded access requests may be considered for patients who have no other treatment options
Conditions:
MPS VII, Mucopolysaccharidosis VII, Sly Syndrome
Individual patient expanded access requests may be considered for patients who have no other treatment options
Conditions:
Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
Individual patient expanded access requests may be considered for patients who have no other treatment options

Additional Information

Available therapies via single-patient EA

Requests for early access to investigational therapies must be made by a qualified physician.