BioMarin said its combination therapy helped adults with late-onset Pompe disease maintain walking distance over five years of continuous treatment. According to Sina Finance, overall respiratory function remained relatively stable, and no new safety signals were identified.

The company presented five-year follow-up data from a Phase III trial of Pombiliti plus Opfolda for late-onset Pompe disease (LOPD). The analysis included 82 participants, including treatment-naive patients who had not previously received enzyme replacement therapy and treated patients who had already used enzyme replacement therapy before enrollment.

Late-onset Pompe disease is a rare genetic lysosomal storage disorder that can cause progressive muscle weakness, including damage to the muscles that control breathing. BioMarin said the results were disclosed at the 31st World Muscle Society Annual Congress in Japan.