BioMarin reports five-year Phase 3 PROPEL extension data for Pombiliti plus Opfolda in late-onset Pompe disease

By Public Technologies
  • BioMarin disclosed five-year Phase 3 extension results for Pombiliti with Opfolda in late-onset Pompe disease at the World Muscle Society congress.
  • Long-term follow-up showed durable walking ability with broadly stable lung function, supporting sustained benefit in a progressive condition.
  • Safety profile remained broadly consistent with prior experience, with no new safety signals; some patients stopped treatment due to adverse events.
  • Separate Phase 1/2 updates for investigational Duchenne drug nivudirsen were presented, showing dystrophin production signals with generally favorable tolerability.


Disclaimer: This news brief was created by Public Technologies (PUBT) using generative artificial intelligence. While PUBT strives to provide accurate and timely information, this AI-generated content is for informational purposes only and should not be interpreted as financial, investment, or legal advice. BioMarin Pharmaceutical Inc. published the original content used to generate this news brief via PR Newswire (Ref. ID: 202610021100PR_NEWS_USPR_____SF62144) on October 02, 2026, and is solely responsible for the information contained therein.

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