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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.

EA Webpage Clinicaltrials.gov
Phone Number & Email
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.

Phone Number & Email
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.

EA Webpage Clinicaltrials.gov
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

EA Webpage Clinicaltrials.gov
Phone Number & Email
Expected Application Timeframe
Immediately

Global Managed Access Program Cohort for Remibrutinib in Adult Patients With Chronic Spontaneous Urticaria

Conditions
Chronic Spontaneous Urticaria

The purpose of this Managed Access Program (MAP) Cohort Treatment Plan is to provide access to remibrutinib for adult patients with chronic spontaneous urticaria (CSU)

MAP Cohort CLNP023B12004M to Provide Access to Iptacopan for C3 Glomerulopathy

Conditions
C3 Glomerulopathy (C3G)

The purpose of this Managed Access Program (MAP) Cohort Treatment Plan is to provide guidance to the Physician for the treatment and monitoring of patients in the Cohort MAP. The Physician should follow the suggested treatment guidelines. Furthermore, the Physician must comply with the MAP Agreement Letter and applicable local laws and regulations.

MAP to Provide Access to Midostaurin, for Patients 18 Years of Age and Older With Newly-diagnosed FLT3-mutated AML and Eligible for Induction and Consolidation Chemotherapy

Conditions
FMS-Like Tyrosine Kinase 3-mutated Acute Myeloid Leukemia

The purpose of this Cohort Treatment Plan is to allow access to midostaurin in combination with standard chemotherapy for eligible patients newly diagnosed with FLT3-mutated AML. The patient's Treating Physician should follow the suggested treatment guidelines and comply with all local health authority regulations.

MAP to Provide Access to Ruxolitinib, for Patients With Polycythemia Vera

Conditions
Polycythemia Vera

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

Global Managed Access Program Cohort for Ligelizumab in CSU

Conditions
Chronic Spontaneous Urticaria

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

177Lu-PSMA-617 Managed Access Program for mCRPC Patients

Conditions
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.

MAP to Provide Access to Eltrombopag, for Treatment of Patients With Medically Significant Thrombocytopenia

Conditions
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.

MAP to Provide Access to Ruxolitinib, for Patients With PMF or PPV MF or PET-MF

Conditions
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.

Treatment Plan CINC280A02001M to Provide Access to Capmatinib, for MET Exon 14 Skipping Non-Small Cell Lung Cancer (NSCLC)

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

The purpose of this Managed Access Program (MAP) Cohort Treatment Plan is to provide guidance to the Physician for the treatment and monitoring of patients in the Cohort MAP. The Physician should follow the suggested treatment guidelines. Furthermore, the Physician must comply with the MAP Agreement Letter and applicable local laws and regulations.

Managed Access Program to Provide Access to Alpelisib for Patients With Advanced Breast Cancer

Conditions
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.

Compassionate Use Program to Access Trametinib and Dabrafenib for Patients With BRAFmutation-positive Metastatic Melanoma

Conditions
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.

Managed Access Program Cohort Treatment Plan CTMT212X2002I to Provide Access to Trametinib and Dabrafenib Combination Therapy for Patients With BRAF V600 Mutation-positive Advanced Non-Small Cell Lung Cancer

Conditions
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.

Nilotininb, for Patients With CML-CP, CML-AP, or CML-BC

Conditions
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.

Compassionate Use Program BRAF Mutation-positive Patients in the Adjuvant Treatment of Melanoma After Surgical Resection

Conditions
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.

Managed Access Program (MAP) for Patients Diagnosed With Secondary Progressive Multiple Sclerosis With Active Disease

Conditions
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.

CTL019 Out of Specification MAP for ALL or DLBCL Patients

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.

MAP to Provide Access to Crizanlizumab, for Sickle Cell Disease Patients

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.

Managed Access Program (MAP) to Provide Access to Alpelisib (BYL719) for Patients With PIK3CA-Related Overgrowth Spectrum (PROS)

Conditions
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

Lamprene Multiple Patient Program

Conditions
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.

Managed Access Program (MAP)* to Provide Access to Asciminib for Patients With CML in Chronic Phase

Conditions
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

EA Webpage Clinicaltrials.gov
Phone Number & Email
Expected Application Timeframe
5 business days

NS Pharma, Inc.

EA Webpage Clinicaltrials.gov
Phone Number & Email
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

Nuvation Bio, Inc.

EA Webpage Clinicaltrials.gov
Expected Application Timeframe
N/A
Single-Patient EA Policies/Criteria

At this time, Nuvation Bio products are considered investigational, which means that they have not been approved as safe and effective by regulatory health authorities, such as the United States Food and Drug Administration (FDA). Currently, participation in clinical trials is the only way for patients to gain access to Nuvation Bio’s investigational drugs. As more clinical data on the safety and efficacy of these investigational drugs become available, Nuvation Bio will review and update its policy on Expanded Access. For information on Nuvation Bio’s clinical trials, please email ClinicalTrials@nuvationbio.com.

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