Amicus Therapeutics’ Pombiliti + Opfolda Receive EC’s Approval for the Treatment of Pompe Disease
Shots:
- The EC has approved Opfolda (65mg capsules) & Pombiliti for adults with LOPD. The company plans to launch Pombiliti + Opfolda in Germany & plans for reimbursement processes with healthcare authorities in other EU countries
- The approval was based on the P-III study (PROPEL) in ERT-naïve & ERT-experienced patients The results showed that Pombiliti + Opfolda exhibited clinically meaningful & positive changes in the key mobility & respiratory manifestations of the disease
- Pombiliti, a bis-M6P-enriched rhGAA enzyme that is designed for increased uptake into muscle cells. Opfolda is an enzyme stabilizer designed to stabilize the enzyme in the blood while Pombiliti + Opfolda received the BTD from the US FDA; MHRA regulatory approval is expected in Q3’23 & US FDA review is ongoing
Ref: Amicus Therapeutics | Image: Amicus Therapeutics
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