BioMarin five-year data show durable motor function in late-onset Pompe disease

BioMarin announced Friday that five-year Phase 3 trial data show its combination therapy Pombiliti and Opfolda helped adults with late-onset Pompe disease maintain walking distance and stabilize respiratory function, with no new safety signals. The analysis followed 82 patients, including both those new to enzyme replacement therapy and those previously treated with it. BioMarin acquired Amicus Therapeutics in December, expanding its presence in rare metabolic diseases. Late-onset Pompe disease is a rare, inherited lysosomal disorder causing progressive muscle weakness, often impacting breathing muscles. The results were presented at the World Muscle Society congress in Japan.