Amicus Therapeutics, Inc. announced that the European Commission (EC) has granted approval for Opfolda(R) (miglustat) 65mg capsules, an enzyme stabilizer of cipaglucosidase alfa, a long-term enzyme replacement therapy for adults with late-onset Pompe disease. Pombiliti (cipaglucosidases alfa), is a bis-M6P-enriched rhGAA enzyme, designed for increased uptake into muscle cells. Once in the cell, Pombiliti can be processed into its most active and mature form to break down glycogen.

Opfolda (miglustat) is an enzyme stabilizer designed to stabilize the enzyme in the blood. The EC approval was based on clinical data from the Phase 3 pivotal study (PROPEL), the only trial in LOPD to study the real-world population of both ERT-nave and ERT-experienced participants in a controlled setting. In the U.K., the Medicines and Healthcare products Regulatory Agency (MHRA) regulatory approval is expected in the third quarter of 2023.

The U.S. Food and Drug Administration's review is ongoing, and the Company expects approval in the third quarter of 20 23. The FDA previously granted Breakthrough Therapy designation for Pombiliti + Opfolda. Pombiliti + Op folda, is a two-component therapy that consists of cipaglucOSidase alfa, a bis-M6P -enriched rhGAA that facilitates high-affinity uptake through the M6P receptor while retaining its capacity for processing into the most active form of the enzyme, and the oral enzyme stabilizer, miglustat, that's designed to reduce loss of enzyme activity in the blood.

In clinical studies, Pombiliti + Op foldinga was associated with demonstrated improvements in both muscleoskeletal and respiratory measures. Refer to SmPC for full list.