BioMarin Reports Positive Five-Year Data for Pompe Disease Combination Therapy
On Friday, BioMarin announced five-year data from a Phase 3 trial of its combination therapy Pombiliti and Opfolda used to treat late-onset Pompe disease (LOPD). The results showed that the treatment helped patients maintain walking distance over five years while keeping respiratory function stable. No new safety concerns were identified during this long-term analysis, which included 82 participants—both enzyme replacement therapy newcomers and those previously treated. Late-onset Pompe disease is a rare inherited disorder causing progressive muscle weakness affecting breathing muscles in many cases. The data was presented at the World Muscle Society Congress in Japan. BioMarin acquired Amicus Therapeutics last December to expand its portfolio of therapies for rare metabolic diseases, including this combination therapy.
Topics: enzyme_replacement_therapy Phase_3_trials rare_disease