Amicus Therapeutics Receives EMA’s CHMP Positive Opinion Recommending Marketing Authorization of Opfolda (miglustat) for Late-Onset Pompe Disease
Shots:
- The EMA’s CHMP has adopted a positive opinion recommending marketing authorization of miglustat. The EC’s decision & commercial launch of Pombiliti + Opfolda is expected in Q3’23
- The opinion was based on the P-III trial (PROPEL) evaluating the efficacy, safety & tolerability of Pombiliti. AT-GAA showed improvements in musculoskeletal & respiratory measures in clinical studies
- If Pombiliti + Opfolda is approved, it will be the first two-component therapy available in the EU for the treatment of adults with LOPD. Pombiliti + Opfolda also known as AT-GAA, the 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’ Pombiliti (cipaglucosidase alfa) Receives EC’s Approval for the Treatment of Late-Onset Pompe Disease
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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.
