On Friday, BioMarin revealed results from a five-year Phase 3 trial for its drugs Pombiliti and Opfolda, aimed at treating late-onset Pompe disease (LOPD). The data demonstrated that the combination therapy effectively helped adult patients maintain their walking distance and stabilize respiratory function over the long term, with no new safety concerns reported.
This trial involved 82 patients, including those new to enzyme replacement therapy and those previously treated. The significance of this data is underscored by BioMarin's recent acquisition of Amicus Therapeutics, which marketed the combination therapy, thereby enhancing BioMarin's footprint in the rare metabolic disease sector.
LOPD is a severe, inherited disorder characterized by progressive muscle weakness, which can severely affect respiratory muscles. The findings were presented at the 31st annual Congress of the World Muscle Society in Japan, indicating a strong commitment to advancing treatment options in this niche area