Clinical TrialJun 10, 2026, 07:56 AM
Rafael Holdings Completes Phase 3 TransportNPC™ Study
AI Summary
Rafael Holdings, Inc. announced the completion of the last patient's final 96-week study visit in its pivotal Phase 3 TransportNPC™ study evaluating Trappsol® Cyclo™ for the treatment of Niemann-Pick Disease Type C. The company anticipates announcing topline data from the main study cohort and submitting a New Drug Application (NDA) in the second half of 2026, having already completed a pre-NDA meeting with the FDA. Additionally, the FDA has acknowledged the company's US Expanded Access Program.
Key Highlights
- Last patient completed final 96-week study visit in pivotal Phase 3 TransportNPC™ study.
- Topline data from the main study cohort is expected in the second half of 2026.
- Rafael Holdings expects to submit a New Drug Application (NDA) in H2 2026.
- The company completed a pre-NDA meeting with the FDA.
- The US Expanded Access Program (EAP) for Trappsol® Cyclo™ has been acknowledged by the FDA.
- The Phase 3 study enrolled 94 patients across 27 sites in 13 countries.
- Pediatric sub-study data showed 80% of patients' CGI scores improved or remained stable.
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