BioMarin Announces Five-Year Phase 3 Data for POMBILITI® (cipaglucosidase alfa-atga) + OPFOLDA® (miglustat) at World Muscle Society Annual Congress
PR Newswire•02/10/2026•11:00 ET
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Key Highlights
- ➤POMBILITI + OPFOLDA showed durable motor-function outcomes through five years
- ➤Pulmonary function declined 2.8% in ERT-experienced and 4.4% in ERT-naïve cohorts
- ➤82 participants received continuous POMBILITI + OPFOLDA treatment through year five
- ➤51.2% experienced treatment-related adverse events; five discontinued for safety reasons
- ➤Nivudirsen showed positive near full-length dystrophin expression and favorable tolerability
Expert Statements
Greg Friberg, M.D., Executive Vice President and Chief Research & Development Officer at BioMarin
“These five-year results demonstrate that POMBILITI and OPFOLDA can provide durable, long-term benefits for people living with late-onset Pompe disease, including those who had been on enzyme replacement therapy for years before enrolling in the study.”
Greg Friberg, M.D., Executive Vice President and Chief Research & Development Officer at BioMarin
“Sustaining motor function and limiting pulmonary decline for up to five years in a progressive disease like LOPD is clinically meaningful, and we remain committed to continuing to share long-term evidence to inform the care of people with this rare genetic condition.”
BioMarin Announces Five-Year Phase 3 Data for POMBILITI® (cipaglucosidase alfa-atga) + OPFOLDA® (miglustat) at World Muscle Society Annual Congress PR Newswire
Long-term data reinforce durable efficacy and safety of POMBILITI + OPFOLDA in late-onset Pompe disease
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