Amicus Therapeutics

New Capabilities

As of October 2026: Acquired by BioMarin in December 2025

Adults with late-onset Pompe disease who had already spent an average of 7.6 years on enzyme therapy kept their walking distance nearly unchanged over five more years on BioMarin's Pombiliti and Opfolda, while lung function slipped slightly. BioMarin presented the results October 2 at the World Muscle Society congress in Hiroshima.

Updated Oct 4