Rafael Holdings Announces Submission of New Drug Application to FDA for Trappsol® Cyclo™ (remibetadex) for the Treatment of Niemann-Pick Disease Type C
Globe Newswire•29/10/2026•07:00 ET
0
Key Highlights
- ➤Rafael Holdings submitted FDA NDA for remibetadex targeting Niemann-Pick Disease Type C.
- ➤Phase 3 missed primary endpoint; disease progression slowed 64% versus placebo (p=0.19).
- ➤Background-therapy subgroup showed 71% relative slowing of disease progression (p=0.046).
- ➤Infantile-onset NPC analysis found 85% lower mortality risk versus matched registry controls (p=0.044).
- ➤Serious adverse events occurred more often with remibetadex than placebo, 35.9% versus 16.7%.
Expert Statements
Howard Jonas, Chairman and Chief Executive Officer of Rafael Holdings
“This submission is an important milestone for Rafael Holdings and, most importantly, for patients living with NPC. I believe the totality of the data, including the Phase 3 trial results and the overall survival analysis, supports the potential of remibetadex for patients with limited treatment options. Pending FDA approval, we are excited to bring remibetadex to patients and families who urgently need new options to treat NPC.”
Joshua Fine, Chief Operating Officer of Rafael Holdings
“The remibetadex NDA is the culmination of more than 15 years of research and investment and, if approved, could help address a significant unmet need in a rare, and fatal disease. With the data in the NDA and Orphan Drug, Fast Track and Rare Pediatric Disease designations in place, upon the FDA’s filing of the NDA, we believe remibetadex is well positioned for review, and an approval would make it eligible for a Priority Review Voucher, a potential source of non-dilutive capital. With this important milestone completed, we are preparing for a potential commercial launch, and we look forward to working with the FDA as the regulatory review process advances. We are grateful to the investigators, patients, families and advocates who made this milestone possible.”
NDA supported by the Phase 3 TransportNPC(™) study, data from earlier clinical studies, and an overall survival analysis versus external registry controls
Remibetadex has received Orphan Drug, Fast Track and Rare Pediatric Disease designations from the FDA; approval would make remibetadex eligible for a Rare Pediatric Disease Priority Review Voucher
Get started
Create a free account to read the full story.