Drug Repurposing for Unmet Medical Needs; Request for Information

NCRI Health, a working group at Rutgers University, advocates for identifying ME/CFS, Long Covid, POTS, Lyme-IACCI, and Fibromyalgia as priority disease areas for drug repurposing. They specifically recommend that the FDA expedite the review of low-dose naltrexone for these conditions, citing clinical evidence, real-world data, and a lack of commercial sponsorship for the drug.

Vanda Pharmaceuticals Inc. supports the FDA's exploration of drug repurposing and argues that the agency should provide more regulatory flexibility, incentives, and streamlined processes for approving new uses of existing drugs. The company specifically advocates for the approval of its drugs, Hetlioz® and Nereus™, for various unmet medical needs, citing existing clinical evidence and criticizing current regulatory hurdles as barriers to innovation.

The Muscular Dystrophy Association (MDA) supports the FDA's initiative on drug repurposing, specifically advocating for neuromuscular diseases to be prioritized as a key area of focus. They suggest specific pathways for identifying candidates and call for collaboration with CMS and HHS to reduce insurance and reimbursement barriers for off-label therapies.

The American Society of Transplant Surgeons (ASTS) supports the FDA's interest in drug repurposing to expand access to immunosuppressive drugs for transplant recipients. They argue that creating a pathway to repurpose drugs already approved for kidney transplants could eliminate significant reimbursement barriers for patients receiving extra-renal transplants.

Matthew Halma, representing the Open Source Medicine Foundation (OSMF), supports the FDA's request for information on drug repurposing and proposes specific priority areas like infection-associated chronic conditions. He offers the organization's open-source tools, infrastructure, and biobank data to help the FDA develop non-commercial, decentralized clinical trial platforms.

Biostax Corp (Attune Biotech Inc.) submits a white paper supporting the FDA's initiative to modernize drug repurposing for unmet medical needs. They argue that low-dose naltrexone (LDN) is a prime candidate for this framework because it has established safety, widespread clinical use, and significant federal research support, but lacks a standardized, FDA-approved pharmaceutical product due to market failures.

The Association for Accessible Medicines (AAM) supports the FDA's efforts to explore drug repurposing for unmet medical needs, provided that the framework preserves existing intellectual property and regulatory standards. They recommend that the FDA consider the operational impacts on generic drug labeling, utilize real-world evidence, and maintain transparency and stakeholder engagement throughout the process.

StemCyte is advocating for the accelerated approval and repurposing of its cord blood cell therapy, REGENECYTE, for the treatment of Long COVID fatigue. They highlight positive Phase IIa trial data and request that the FDA address cost and coverage barriers to ensure patient access.

The commenter advocates for the FDA to consider the drug Ampligen (Rintatolimod) for repurposing to treat Long COVID, specifically for patients with moderate-to-severe fatigue. They highlight positive signals from Phase 2 and Phase 3 trials and request a severity-gated Phase 3 trial with expanded access.