India, Oct. 2 -- Friday, BioMarin Pharmaceutical Inc. (BMRN) reported five-year Phase 3 data for POMBILITI (cipaglucosidase alfa-atga) in combination with OPFOLDA (miglustat) in adults with late-onset Pompe disease (LOPD), while the company's shares traded lower.
The data were presented at the 31st Annual Congress of the World Muscle Society (WMS) in Hiroshima, Japan, including results from the PROPEL open-label extension study.
POMBILITI + OPFOLDA is a two-component therapy consisting of an enzyme replacement therapy and an oral enzyme stabilizer. The combination is approved for certain adults with LOPD who are not improving on their current enzyme replacement therapy.
Five-Year PROPEL Data
The PROPEL open-label extension included 82 p...