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
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