BioMarin Says 5-Year Data Shows Durable Efficacy of Pombiliti Plus Opfolda in Pompe Disease

11:56 AM EDT, 10/02/2026 (MT Newswires) -- BioMarin Pharmaceutical (BMRN) said Friday that five-year data from an open-label extension study showed that Pombiliti plus Opfolda therapy delivered durable motor function outcomes and relative stabilization of pulmonary function in patients with late-onset Pompe disease. The company said no new safety signals were identified, although 51.2% of the participants experienced treatment-related adverse events that were mostly manageable, and five individuals discontinued treatment for safety reasons. Shares of the company were down 1.4% in Friday trading. Price: 57.65, Change: -0.79, Percent Change: -1.36