Amicus Drug Scores FDA Approval For Rare Inherited Pompe Disease

 The FDA has approved Amicus Therapeutics' FOLD Pombiliti (cipaglucosidase alfa-atga)+Opfolda (miglustat) 65mg capsules for Pompe disease.

It's a disease in which the body can't make a protein that breaks down a complex sugar called glycogen for energy. Too much sugar builds up and damages muscles and organs.

This two-component therapy is indicated for adults living with late-onset Pompe disease (LOPD) weighing ≥40 kg and who are not improving on their current enzyme replacement therapy (ERT).

Pombiliti is a recombinant human GAA enzyme (rhGAA) naturally expressed with high levels of bis-M6P (Mannose 6-Phosphate), designed for increased uptake into muscle cells. Once in the cell, Pombiliti can be properly processed into its most active and mature form to break down glycogen. 

Opfolda is an enzyme stabilizer designed to stabilize the enzyme in the blood.

The FDA approval was based on clinical data observed from the Phase 3 pivotal study (PROPEL), the only trial in LOPD to study ERT-experienced participants in a controlled setting.

https://www.benzinga.com/general/biotech/23/09/34979628/amicus-therapeutics-drug-scores-fda-approval-for-rare-inherited-pompe-disease