NasdaqGS:PRAXBiotechs
FDA-Endorsed Elsunersen Trial Redesign Could Be A Game Changer For Praxis Precision Medicines (PRAX)
Praxis Precision Medicines recently completed a Type C meeting with the FDA, securing agreement to convert its EMBRAVE3 trial of elsunersen for early-onset SCN2A developmental and epileptic encephalopathy into a smaller, single-arm, baseline-controlled study focused on seizure reduction over 24 weeks plus an open-label extension.
This shift, alongside prior orphan and rare pediatric designations, underscores elsunersen’s potential as a disease-modifying therapy in an ultra-rare, genetically...