India, Oct. 2 -- On Friday, BioMarin Pharmaceutical (BMRN) reported five-year Phase 3 data for POMBILITI + OPFOLDA in adults with late-onset Pompe disease (LOPD) at the World Muscle Society Congress in Hiroshima, Japan.
The PROPEL open-label extension included 82 patients continuously treated from the Phase 3 baseline through Year 5. Among ERT-experienced patients, mean changes in six-minute walk distance and predicted forced vital capacity were 0.7% and -2.8%, respectively, at Year 5.
No new safety signals were reported. BioMarin also presented Phase 1/2 data for investigational nivudirsen (BMN 351) in Duchenne muscular dystrophy, showing generally favorable tolerability and near full-length dystrophin expression.
Currently, BMRN is tra...