BioMarin reports five-year Pompe therapy data and early Duchenne findings
BioMarin presented five-year extension results for Pombiliti plus Opfolda in late-onset Pompe disease at the World Muscle Society Congress. Among 82 continuously treated participants, the ERT-experienced cohort recorded a 0.7% mean change in percent-predicted six-minute walk distance and a -2.8% change in forced vital capacity at year 5. The company also reported that its Phase 1/2 nivudirsen study in Duchenne muscular dystrophy showed generally favorable tolerability and near full-length dystrophin expression.
Why it matters
The update provides longer-duration clinical and safety evidence for BioMarin's marketed Pompe regimen. Nivudirsen remains investigational, so its Phase 1/2 findings add early development evidence rather than a regulatory decision.
Key facts
- 1The PROPEL extension analysis included 82 people who stayed on Pombiliti plus Opfolda from the original study baseline through year five. prnewswire.com
- 2For the ERT-experienced cohort, mean prior ERT exposure at baseline was 7.6 years. prnewswire.com
- 3Treatment-related adverse events occurred in 51.2% of participants, and five people stopped treatment for safety reasons. prnewswire.com
- 4BioMarin said no new safety signals emerged in the Pombiliti plus Opfolda analysis. prnewswire.com
- 5The ongoing 351-201 trial is a Phase 1/2 study of nivudirsen in boys aged 4 to 10 with exon 51 skip-amenable Duchenne muscular dystrophy. prnewswire.com
Summary written by AlphAI from 3 of 3 sources. Not investment advice. Figures are as stated by the linked sources.