Clinical TrialJun 10, 2026, 07:31 AM
Rafael Holdings Completes Phase 3 NPC Trial; NDA Expected H2 2026
AI Summary
Rafael Holdings, Inc. announced the completion of the pivotal Phase 3 TransportNPC™ study for Trappsol® Cyclo™ in Niemann-Pick Disease Type C (NPC). Topline data from the study, which involved 94 patients globally, is expected in the second half of 2026. The company has already held a pre-NDA meeting with the FDA and plans to submit its New Drug Application (NDA) in the same period, aiming for an expedited path to market.
Key Highlights
- Rafael Holdings completed the pivotal Phase 3 TransportNPC™ study for Trappsol® Cyclo™ in Niemann-Pick Disease Type C (NPC).
- Topline data from the main study cohort is expected to be announced in the second half of calendar 2026.
- The company has completed a pre-NDA meeting with the FDA and expects to submit an NDA in H2 2026.
- The US Expanded Access Program (EAP) for Trappsol® Cyclo™ has received acknowledgement from the FDA.
- The Phase 3 study involved 94 patients across 27 sites in 13 countries.
- A single-arm pediatric sub-study showed 80% of patients' CGI scores improved or remained stable with no serious adverse events.
- Rafael Holdings aims to transition into a commercial-stage biotechnology company, unlocking long-term value for shareholders.
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