Company Directory

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/

Single-Patient EA Policies/Criteria

(1) To be eligible for the Expanded Access Program, the patient must have a ① serious or life-threatening condition (e.g., Knee Osteoarthritis of K-L grade 3), ② a condition that does not have satisfactory alternative treatment options available, ③ and be unable to participate in ongoing clinical trials. This Expanded Access Program aims to provide a treatment opportunity with JointStem—an autologous adipose-derived mesenchymal stem cell product (1×10^8 cells/3 mL)—to patients with K-L grade 3 knee osteoarthritis whose symptoms have persisted for more than 3 months despite SOC treatment.
(2) This Expanded Access Program is intended for patients with osteoarthritis of the knee to assess the symptom-relieving effect of JointStem. Only patients who have received sufficient explanation of the program and voluntarily signed the informed consent form will be evaluated for patient eligibility during the screening phase. If eligible, they will undergo fat tissue and blood collection. Once the JointStem product is manufactured, patients will visit the site to receive a single dose of JointStem and will undergo regular follow-up evaluations for safety and efficacy over 24 weeks.

Available Therapies via Single-Patient EA

JointStem (Autologous Adipose Tissue derived Mesenchymal Stem Cells) for Knee Osteoarthritis of Kellgren & Lawrence grade 3

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

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.

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.

Available Therapies via Single-Patient EA

NexEos Bio’s position is that enrollment in a NexEos Bio-sponsored clinical trial is the preferred route of access to our investigational drug product. Information about ongoing clinical trials is posted publicly at www.ClinicalTrials.gov
None of NexEos Bio’s investigational drug products are available for expanded access at this time.

Available Therapies via Single-Patient EA

At NH TherAguix, our mission is to develop novel nanomedicine solutions applicable to precision radiotherapy in oncology. Our drug candidate AGuIX® is a nanodrug designed to meet the growing medical needs in brain cancer by improving the efficacy and precision of radiotherapy directly within tumors.
As defined by the US Food and Drug Administration, expanded access, sometimes called “compassionate use”, is a potential pathway for a patient with an immediately life-threatening condition or serious disease or condition to gain access to an investigational medical product (drug, biologic, or medical device) for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available.

Our goal is to provide access to our investigational drug at the appropriate time and in a manner that is most beneficial to the relevant patient population. We understand that patients, caregivers, or health professionals may wish to have access to our drug candidate before receive regulatory approval, outside the context of a clinical trial. At the present time, we are not making our product available for such pre-approval access. We believe enrollment in our ongoing clinical trial is the safest and most effective way of achieving this goal. We currently have ongoing Phase 1b/2 and Phase 2 clinical trials, in which the investigational drug is added to the standard of care that patients are already be receiving. You can find information about ongoing clinical trials of our drug candidate by visiting www.clinicaltrials.gov and entering “AGuIX” in the “other terms” search box.

NH TherAguix may revisit this policy as we gain additional scientific knowledge about our drug candidate through clinical trial data. Any such changes will be posted to this site.

Single-Patient EA Policies/Criteria

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

Disease/Category-Specific EA Policies/Criteria

Available Therapies via Single-Patient EA

Novavax promotes improved health by discovering, developing, and commercializing innovative vaccines to protect against serious infectious diseases. We offer a differentiated vaccine platform that combines a recombinant protein approach, innovative nanoparticle technology and Novavax's patented Matrix-M adjuvant to enhance the immune response.

Our vaccines are designed to be used prophylactically, meaning that they are designed and intended to prevent disease in healthy individuals, not for the treatment of existing disease. Like all vaccines, our products must be carefully evaluated in clinical trials to ensure that they can be used safely and effectively.

We understand that patients, caregivers, or healthcare professionals may wish to have access to our vaccine candidates before they receive regulatory approval, outside the context of a clinical trial. At the present time, we are not making our products available for such pre-approval access. This includes requests for pre-approval access under section 561(b) of the Federal Food, Drug, and Cosmetic Act, the Right to Try Act of 2017 (Pub. L. No. 115-176), or any state laws. We believe that participation in clinical trials, with their established eligibility criteria and ethical safeguards, is the most appropriate and equitable approach to making our investigational vaccines available before they are approved.

Novavax may revisit this policy as we gain additional scientific knowledge about our vaccine products through clinical trial data and real-world use after vaccine licensure or authorization, or if we develop therapeutic products in the future. Any such changes will be posted to this site.

You can find information about ongoing clinical trials of our vaccines by visiting www.clinicaltrials.gov and entering “Novavax” in the “other terms” search box. Any inquiries regarding this policy should be directed to https://www.novavax.com/contact-us. Novavax expects to acknowledge any such inquiries from patients, caregivers, or healthcare professionals within five 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

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.

Single-Patient EA Policies/Criteria

On our website (www.nuvectis.com) physicians and patients may explore information about our investigational drug candidate, NXP800.

Given the early stage of the NXP800 development program, we believe that participation in our clinical trial(s) is the most appropriate way to access NXP800, and therefore we are currently not making NXP800 available through an expanded access program.

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

Single-Patient EA Policies/Criteria

At this time, given the early stage of development, Nuvig Therapeutics is not offering Expanded access for NVG-2089