BioMarin reports five-year Pompe disease data showing durable motor function
BioMarin shared five-year data showing a combination therapy helped adults with late-onset Pompe disease maintain motor function.
TL;DR
- Five-year Phase 3 trial data for Pombiliti and Opfolda in late-onset Pompe disease (LOPD) was announced by BioMarin.
- The combination therapy helped adults maintain walking distance and respiratory function over five years.
- No new safety signals were identified.
- BioMarin acquired the combination therapy from Amicus Therapeutics in December.
- The trial analyzed 82 patients, including those new to enzyme replacement therapy and those previously treated.
- LOPD is a rare inherited lysosomal disorder causing progressive muscle weakness.
- Results were presented at the 31st annual Congress of the World Muscle Society in Japan.