Comment from Healx Limited

Healx Ltd. submits the attached response to Docket FDA-2026-N-4492. Healx is an AI-enabled rare disease drug repurposing company working across a portfolio of rare and ultra-rare disease programmes. More than 7,000 rare diseases affect approximately 30 million Americans, and approved treatments exist for only a small minority. AI-enabled sponsors, patient organisations and nonprofits can now identify credible repurposing candidates at scale, but two barriers repeatedly make otherwise viable programmes uneconomic. First, safety data from shelved or failed assets is often inaccessible, locked in dormant sponsor files or lost through bankruptcy. Second, sponsors are asked to duplicate safety work already completed and reviewed by trusted ICH-member regulators (EMA, PMDA, MHRA, Health Canada, Swissmedic, TGA). Both barriers scale across every asset in a portfolio, and both fall hardest on paediatric rare disease repurposing, which is structurally excluded from the Rare Pediatric Disease Priority Review Voucher programme under FDCA 529. The attached response proposes five administrative reforms, all within FDA's existing authorities: A published, tiered reliance framework applying 21 CFR 312.120 and 314.106 more predictably to repurposing candidates approved by trusted ICH-member regulators, modelled on the MHRA International Recognition Procedure. Regulatory continuity for third-party acquirers of orphaned dossiers from failed sponsors, using existing transfer provisions under 21 CFR 314.72. Repurposing-specific 505(b)(2) guidance for academic, nonprofit and patient-organisation sponsors. A Rare Disease Repurposing Fast Track assembled from existing authorities (Fast Track, Orphan Drug Designation, OOPD engagement, pre-IND support). Structured off-label evidence capture through expanded CURE ID and NCATS infrastructure. None of these proposals asks FDA to lower the substantial evidence standard under FDCA 505(d), delegate review authority, or override trade secret protections. They ask FDA to make better use of safety and regulatory work already completed to internationally harmonised standards. Healx welcomes the opportunity to engage further with FDA on any of the proposals set out in the attached response.