BioMarin reports five-year Phase 3 PROPEL extension data for Pombiliti plus Opfolda
BMRN•BioMarin disclosed five-year Phase 3 extension results for Pombiliti with Opfolda in late-onset Pompe disease at the World Muscle Society congress. Follow-up showed durable walking ability, broadly stable lung function and no new safety signals.
1. Pompe disease results
Long-term follow-up showed durable walking ability and broadly stable lung function. The safety profile remained broadly consistent with prior experience, with no new safety signals; some patients stopped treatment due to adverse events.
2. Duchenne drug update
BioMarin also presented separate Phase 1/2 updates for investigational Duchenne drug nivudirsen, showing dystrophin production signals with generally favorable tolerability.
