Rafael Holdings subsidiary Cyclo Therapeutics seeks FDA approval for remibetadex to treat NPC
RFL•Cyclo Therapeutics submitted an FDA application for remibetadex to treat Niemann-Pick disease type C in adults and children from birth. Its Phase 3 trial missed its primary endpoint, while a prespecified subgroup showed 71% relative slowing of disease progression versus placebo (p=0.046).
1. Trial results and safety
The Phase 3 trial showed 64% relative slowing of disease progression versus placebo, but missed its primary endpoint (p=0.19). In a prespecified subgroup receiving background therapy, about 83% of participants, progression slowed by 71% (p=0.046). A survival analysis linked remibetadex to an 85% lower mortality risk in infantile-onset patients versus matched registry controls (p=0.044). Serious adverse events occurred in 35.9% of remibetadex recipients and 16.7% of placebo recipients; no new safety signals emerged.
