Rafael submits remibetadex for FDA review despite a missed pivotal endpoint

Rafael Holdings ($RFL) said its Cyclo Therapeutics subsidiary submitted a US marketing application for remibetadex, also called Trappsol Cyclo, for neurological and internal-organ manifestations of Niemann-Pick disease type C. The proposed indication covers adults and children from birth. Submission starts an FDA filing assessment; it does not mean the application has been accepted, granted priority review or approved.

The supporting Phase 3 TransportNPC trial missed statistical significance on its overall primary endpoint, with p=0.19. A prespecified subgroup receiving background therapy showed 71% slower disease progression, with p=0.046. Serious adverse events occurred in 35.9% of remibetadex recipients versus 16.7% on placebo. Additional survival evidence uses external registry controls, limiting causal interpretation. The filing therefore advances the regulatory process while substantial efficacy and safety questions remain for FDA assessment.

Sources:

• GlobeNewswire