BioMarin Announces Five-Year Phase 3 Data for POMBILITI® (cipaglucosidase alfa-atga) + OPFOLDA® (miglustat) at World Muscle Society Annual Congress

Data ReadoutpositivePOMBILITIOPFOLDAPositive

AI Analysis

Summary

BioMarin announced five-year Phase 3 data for POMBILITI + OPFOLDA combination therapy in late-onset Pompe disease, demonstrating durable efficacy and safety at the World Muscle Society Annual Congress.

Clinical Trial Data

Phase

Phase 3

Primary Endpoint

Met

Outcome Details

Five-year Phase 3 data showing durable efficacy and safety of POMBILITI + OPFOLDA combination in late-onset Pompe disease

Importance:7/10

Sentiment:

0.70

Phase 3Data readoutPompe diseaseLong-term efficacySafety dataCombination therapy

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Published by PR Newswire Biotech on October 2, 2026 3:00 PM

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