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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.

4 A B C D E F G H I J K L M N O P Q R S T U V W X Y Z
Company Name
Phone Number & Email
Company Acknowledgement

NanoCarrier Co., Ltd.

Expected Application Timeframe
10 business days
Single-Patient EA Policies/Criteria

A. Patient Eligibility Criteria
To be eligible for access to an investigational product, patients must meet the following criteria:
• Suffer from a serious or immediately life-threatening disease or condition.
• Have undergone appropriate standard treatments without success and no comparable or satisfactory alternative treatment is available or exists to treat the disease or condition.
• Are ineligible for participation in any ongoing clinical study of the investigational product, which includes lack of access due to geographic limitations.
• The patient has a disease for which there is sufficient evidence of a projected benefit from the use of the investigational product and the benefit outweighs the known or anticipated risks.
• There is adequate information to support appropriate dosing for a special patient population such as pediatric, elderly, renal or hepatic disease, etc.
• Any other pertinent medical criteria for access to the investigational product, as established by the NanoCarrier clinically or medically responsible individual.

B. Investigational Product Criteria
In addition to the patient eligibility requirements, the investigational product must meet the following criteria:
• The product is under investigation in one or more clinical studies.
• There is sufficient evidence to expect that the investigational product will have an acceptable safety profile for the intended patient population.
• The provision of the investigational product will not interfere with or compromise the clinical development of the product.

Available Therapies via Single-Patient EA

NC-6300 (nanoparticle epirubicin) for the treatment of patients with advanced or metastatic angiosarcoma not amenable to curative treatment with radiation or surgery

Disease/Category-Specific EA Policies/Criteria

https://www.nanocarrier.co.jp/en/pipeline/expanded_access_policy/

NeoMatrix Therapeutics, Inc.

Expected Application Timeframe
N/A
Single-Patient EA Policies/Criteria

NeoMatrix Therapeutics (the Company) is the developer of a novel investigational drug product, cP12. The investigational drug product is being evaluated for the treatment of serious burn injuries and is still in the early stages of clinical development.
At the current stage of clinical development, NeoMatrix Therapeutics has chosen not to offer Expanded Access (EA) and will not accept EA requests for the investigational drug product. The justification of the restriction on EA is as follows:
•    There is insufficient safety and efficacy data to determine the risk-benefit profile for the treatment of serious burn injuries.
•    The Company’s ability to conduct clinical trials is limited. Offering EA under any setting at this time will impact the Company’s ability to execute its trials.
•    The organization does not have the personnel and other resources to offer EA on a fair and equitable basis.
•    The Company’s investigational drug product cP12 requires special handling and shipping.
As the company is not currently accepting EA requests, the specified policy information is not provided at this time. The status of EA restriction will be re-evaluated if early data from future clinical trials suggest that cP12 investigational treatment can offer benefits for patients facing serious or life-threatening conditions.

NeoMatrix Therapeutics, Inc. Expanded Access Policy, Rev: May 17, 2019

Neuraptive Therapeutics, Inc.

Phone Number & Email
Expected Application Timeframe
5 business days
Single-Patient EA Policies/Criteria

At Neuraptive, we are committed to developing first-in-class treatments for patients with peripheral nerve injuries (PNI) and physicians who treat them.

NTX-001 has been designed and is being developed as an adjunctive treatment for patients with nerve injuries and may restore and improve the recovery of neurons following nerve repair surgery. We will be conducting clinical studies to demonstrate the safety and efficacy of NTX-001, and to obtain regulatory approval and ultimately make NTX-001 available.

Before regulatory approval by the U.S. Food and Drug Administration (FDA) and foreign regulatory authorities, patients gain access to investigational treatments, such as NTX-001, by participating in clinical trials and by ‘Expanded access,’ also referred to as compassionate use, ‘Early Access,’ and ‘Emergency Use’. Patients who seek access to investigational medicines outside of an established clinical study and before health authority marketing authorization may wish to do so because standard treatments have failed, because they cannot tolerate already approved medicines, because they are unable to participate in a clinical study or because there are no comparable or satisfactory therapy options available outside of clinical trials. The primary purpose of expanded access is to use the investigational drug for patient treatment purposes rather than to gather data on safety, tolerability, and effectiveness.

Currently, Neuraptive does not offer an expanded access program and does not accept expanded access requests outside of clinical trials. We believe that access to NTX-001 should be limited to clinical trials until its safety, tolerability, and effectiveness for a particular indication has been determined and confirmed by regulatory authorities. Patient access to NTX-001 outside of a clinical trial may interfere with the conduct of our ongoing and future clinical trials, and may also disrupt the progress of our development program, which would, in turn, delay access to many patients in need.

Accordingly, while clinical trials are ongoing, we believe participation in our clinical trials is the only safe and appropriate way to access NTX-001. 

If you have questions about Neuraptive’s expanded access policy, please contact Neuraptive at info@Neuraptive.com. Please expect a response within five business days.

In line with the 21ST Century Cures Act, Neuraptive may revise this policy at any time.

Novartis Pharmaceuticals

Phone Number & Email
Expected Application Timeframe
Immediately
Chronic Spontaneous Urticaria

Global Cohort Managed Access Program (MAP) to provide access to ligelizumab (QGE031) for chronic spontaneous urticaria (CSU)

Metastatic Castration-resistant Prostate Cancer (mCRPC)

The purpose of this Cohort Treatment Plan is to allow access to 177Lu-PSMA-617 for eligible patients diagnosed with metastatic castration-resistant prostate cancer (mCRPC). The patient's treating physician should follow the suggested treatment guidelines and comply with all local health authority regulations.

Thrombocytopenia

The purpose of this Cohort Treatment Plan is to allow access to eltrombopag for eligible patients diagnosed with medically significant thrombocytopenia. The patient's Treating Physician should follow the suggested treatment guidelines and comply with all local health authority regulations.

Carcinoma, Non-Small Cell Lung
Non-Small Cell Lung Cancer
Non-Small-Cell Lung Carcinoma
Nonsmall Cell Lung Cancer

The purpose of this Cohort Treatment Plan is to allow access to INC280 for eligible patients diagnosed with MET dysregulated NSCLC. The patient's Treating Physician should follow the suggested treatment guidelines and comply with all local health authority regulations.

Primary Myelofibrosis (PMF)
Post Polycythemia Myelofibrosis (PPV MF)
Thrombocythemia Myelofibrosis (PET-MF)

The purpose of this Cohort Treatment Plan is to allow access to ruxolitinib for eligible patients diagnosed with PMF, PPV MF or PET MF. The patient's Treating Physician should follow the suggested treatment guidelines and comply with all local health authority regulations. The requesting Treating Physician submitted a request for access to drug (often referred to as Compassionate Use) to Novartis which was reviewed and approved by the medical team experienced with the drug and indication.

HR+, HER2-, Advanced Breast Cancer

The purpose of this Cohort Treatment Plan is to allow access to alpelisib for patients diagnosed with HR-positive, HER2-negative advanced breast cancer with mutated phosphoinositide 3-kinase who progressed on or after AI treatment. The patient's Treating Physician should follow the suggested treatment guidelines and comply with all local health authority regulations.

Cytokine Release Syndrome in COVID-19-induced Pneumonia

This is a global Managed Access Program (MAP) to provide access to canakinumab to patients with cytokine release syndrome resulting from COVID-19 pneumonia

Melanoma

The purpose of this Cohort Treatment Plan is to allow access to trametinib (monotherapy or in combination) and dabrafenib (monotherapy or in combination) for eligible patients diagnosed with metastatic melanoma BRAF mutation-positive.

Hypereosinophilic Syndrome (HES)

The purpose of this program is to allow access to nilotinib for eligible patients diagnosed with Hypereosinophilic syndrome (HES). The patient's Treating Physician should follow the suggested treatment guidelines and comply with all local health authority regulations. The requesting Treating Physician submitted a request for access to drug (often referred to as Compassionate Use) to Novartis which was reviewed and approved by the medical team experienced with the drug and indication. Please refer to the latest Investigator's Brochure (IB) or approved label for overview of drug including: non-clinical and clinical experience, risk and benefits. Novartis will continue to provide any new safety information to the Treating Physician as they emerge.

Small Cell Lung Carcinoma

The purpose of this Managed Access Program (MAP) Cohort Treatment Plan is to provide guidance to the Physician for the treatment and monitoring of eligible patients diagnosed with BRAF V600E/K activating mutation-positive advanced NSCLC using trametinib/dabrafenib in the Cohort MAP CTMT212X2002I. The Physician should follow the suggested treatment guidelines. Furthermore, the Physician must comply with the MAP Agreement Letter and applicable local laws and regulations.

Chronic Myelogenous Leukemia (CML)

The purpose of this Cohort Treatment Plan is to allow access to Nilotinib for eligible patients diagnosed with imatinib-intolerant and/or resistant Philadelphia Chromosome positive (Ph+) Chronic Myelogenous Leukemia in Chronic Phase (CML-CP), Chronic Myelogenous Leukemia in Accelerated Phase (CML-AP), or Chronic Myelogenous Leukemia in Blast Crisis Phase (CML-BC). The patient's Treating Physician should follow the suggested treatment guidelines and comply with all local health authority regulations.

Melanoma
Adjuvant

The purpose of this Treatment Plan is to allow access to trametinib and dabrafenib for eligible high-risk BRAF mutation-positive patients in the adjuvant treatment of melanoma after surgical resection. The patient's Treating Physician should follow the suggested treatment guidelines and comply with all local health authority regulations.

Multiple Sclerosis

The purpose of this MAP Cohort Treatment Plan is to allow access to siponimod for eligible patients diagnosed with Secondary Progressive Multiple Sclerosis with active disease. The patient's Treating Physician should follow the suggested treatment guidelines and comply with all local health authority regulations. The requesting Treating Physician should submit a request for access to the drug (often referred to as Compassionate Use) to Novartis which will be reviewed and assessed by the medical team experienced with the drug and indication.

Acute Lymphoblastic Leukemia (ALL)

The purpose of this Cohort Treatment Plan is to allow access to nilotinib for eligible patients diagnosed with relapsed or refractory Philadelphia chromosome positive (Ph+) acute lymphoblastic leukemia (ALL).

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

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.

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.

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.

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.

Neuroendocrine Tumors

Advanced Accelerator Applications is currently pursuing marketing approval for 177Lu-DOTA0-Tyr3-Octreotate (Lutathera). This expanded access therapeutic protocol aims to allow patients suffering from inoperable, somatostatin receptor positive, neuroendocrine tumors, progressive under somatostatin analogue therapy to access the investigational product, 177Lu-DOTA0-Tyr3-Octreotate (Lutathera), prior to its commercial availability.

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.

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.

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.

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.

PIK3CA-Related Overgrowth Spectrum (PROS)

The purpose of this Cohort Treatment Plan is to allow access to alpelisib for patients diagnosed with PIK3CA-Related Overgrowth Spectrum (PROS) who fulfill certain eligibility criteria as specified in this document. The patient's Treating Physician should follow the suggested treatment guidelines and comply with all local health authority regulations

Non-Tuberculous Mycobacterial (NTM) Infections

Lamprene®/Clofazimine, is a product of the pharmaceutical company named Novartis Pharmaceuticals Corporation. Lamprene®/Clofazimine is approved by FDA (the U.S Food and Drug Administration) for the treatment of leprosy. It is no longer available through pharmacies in the US. It is being tested in non-Novartis clinical studies for drug resistant tuberculosis and non-tuberculous mycobacteria (NTM). To be eligible for participation in this expanded access program, patients must have an NTM diagnosis. The treating physician has decided that this infection can be treated with Lamprene®/Clofazimine. This medicine is provided to the physician in an expanded access program. This means that this medicine is not registered for the treatment of NTM, but it can be used in special situations where there are no other possible treatments. For example, this may be because the patient has a type of Mycobacterial infection that is resistant or failed to respond optimally to other drugs, or because the patient has had side effects that prevent the use of other drugs. The physician must submit a patient registration form to initiate the patient approval process.

Severe/Very Severe COVID-19 Illness

The purpose of this Cohort Treatment Plan is to allow access to ruxolitinib for eligible patients diagnosed with severe/very severe COVID-19 illness. The patient's Treating Physician should follow the suggested treatment guidelines and comply with all local health authority regulations. The requesting Treating Physician submitted a request for access to drug (often referred to as Compassionate Use) to Novartis which was reviewed and approved by the medical team experienced with the drug and indication. Please refer to the latest Investigator's Brochure (IB) or approved label for overview of ruxolitinib including: non-clinical and clinical experience, risk and benefits. Novartis will continue to provide any new safety information to the Treating Physician as they emerge.

Chronic Myeloid Leukemia

This program provides access to asciminib for patients with CML in chronic phase, with or without documented T315I mutation, without comparable or satisfactory alternative therapy to treat the disease

Available Therapies via Single-Patient EA

Novartis considers granting managed access to investigational or pre-approval products via single patient access provided the Managed Access criteria are fulfilled.

Novo Nordisk

Phone Number & Email
Expected Application Timeframe
5 business days

NS Pharma, Inc.

Expected Application Timeframe
Seven (7) business days
Single-Patient EA Policies/Criteria

Single-Patient Expanded Access Policies and Criteria - If a treating physician believes that expanded access may be the only option for a patient, the physician should contact NS Pharma to make a formal request on behalf of a patient. Physicians may contact Early Access Care by emailing nspharma.expandedaccess@earlyaccesscare.com. We may request additional information to assist with patient eligibility for an investigational medication.

Available Therapies via Single-Patient EA

Viltolarsen Expanded Access for Duchenne Muscular Dystrophy - Confirmed DMD mutation in the dystrophin gene that is amenable to skipping of exon 53

Disease/Category-Specific EA Policies/Criteria

https://www.earlyaccesscare.com/companies/ns+pharma+inc./viltolarsen