Drug Repurposing for Unmet Medical Needs; Request for Information

Curada Public Benefit Corporation, an advocacy organization, supports the FDA's Drug Repurposing for Unmet Medical Needs RFI by nominating oral naltrexone hydrochloride 50-mg tablets for an event-driven regimen in alcohol use disorder. They argue that the existing evidence supports a high-priority review and offer to support a protocol-driven literature synthesis or prospective evidence consortium to facilitate the labeling process.

Heidi Cheng, a PharmD candidate, recommends that the FDA evaluate oral metformin as a Scenario 1 drug-repurposing candidate for treating antipsychotic-associated weight gain in adults with schizophrenia-spectrum disorders. She argues that the existing evidence base is strong and suggests that the FDA adopt transparent, criteria-based screening approaches to identify other repurposing candidates.

Fernando Sallés of AgeneBio, Inc. urges the FDA to prioritize and support the development of AGB-101, a repurposed levetiracetam formulation for mild cognitive impairment due to Alzheimer's disease. The commenter argues that the drug shows neuroprotective benefits and highlights the need for federal investment or incentives to overcome the high costs of the 505(b)(2) development pathway.

An individual consumer with Long Covid and a primary immunodeficiency advocates for the FDA to approve Pemgarda as a treatment for Long Covid. The commenter shares their personal experience with the drug's efficacy and urges the FDA to provide full approval and create financial incentives for further research into monoclonal antibody treatments.

The Children's Tumor Foundation (CTF) supports the FDA's drug repurposing initiative and recommends specific high-priority opportunities for treating neurofibromatosis. They advocate for regulatory facilitation to encourage pharmaceutical companies to pursue label expansions for drugs that show clinical benefit but lack commercial incentives for rare diseases.

A parent of a child with autism advocates for designating ASD as a priority area for drug repurposing. The commenter requests that the FDA pursue label expansion for existing drugs, create financial incentives for repurposing trials, and provide guidance on evidence for treatment-responsive subgroups.

Healx Ltd., an AI-enabled rare disease drug repurposing company, supports the FDA's request for information and proposes specific administrative reforms to facilitate drug repurposing. They argue for a tiered reliance framework on foreign regulatory data, improved access to safety data from shelved assets, and the creation of a Rare Disease Repurposing Fast Track to overcome economic and data barriers.

Ruchir Gupta, representing the American Society of Ketamine Physicians, Psychotherapists & Practitioners, advocates for the FDA to approve intravenous racemic ketamine for the treatment of refractory complex regional pain syndrome (CRPS). The organization argues that ketamine is an off-patent generic drug with a well-characterized safety profile and sufficient evidence to address a significant unmet medical need in patients with no other approved pharmacotherapy.

AmoSey Inc., a Canadian life sciences advisory firm, supports the FDA's initiative on drug repurposing and provides specific recommendations on candidate selection and overcoming commercial incentive barriers. They advocate for prioritizing candidates with shared biological mechanisms, utilizing human genetic evidence over pure AI outputs, and creating pathways for third-party-originated proposals to find sponsors.