Amicus Therapeutics’ Pombiliti (cipaglucosidase alfa) Receives EC’s Approval for the Treatment of Late-Onset Pompe Disease
Shots:
- The EC has approved Pombiliti (cipaglucosidase alfa) for the treatment of adults with late-onset Pompe disease (LOPD) used in combination with miglustat. The EMA’s CHMP opinion for miglustat is expected in Q2’23
- The approval was based on the results from the P-III (PROPEL) evaluating the efficacy, safety, and tolerability of Pombiliti
- Additionally, AT-GAA showed improvements in both musculoskeletal and respiratory measures in clinical studies. AT-GAA is a two-component investigational therapy that combines cipaglucosidase alfa to enable high-affinity uptake through the M6P receptor
Ref: Globenewswire | Image: Amicus Therapeutics
Related News:- Amicus Therapeutics Acquires Celenex for its Ten Gene Therapy Programs
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Neha is a Senior Editor at PharmaShots. She is passionate and very enthusiastic about recent updates and developments in the life sciences and pharma industry. She covers Biopharma, MedTech, and Digital health segments along with different reports at PharmaShots. She can be contacted at connect@pharmashots.com.
