After years of seeking approval for the treatment of Pompe disease, this week, as reported by GlobeNewswire, the FDA granted approval to its developer, Amicus Therapeutics, for the first and only dual compound to treat enzyme replacement therapy patients who have Pompe disease.
The FDA’s decision came about after it extended its review for the second time due to inspection delays caused by various pandemic-related restrictions.
About Pompe Disease
Pompe disease is a rare genetic disorder affecting approximately 3,500 people in the United States. It causes progressive weakness in the skeletal muscles and heart. Mutations in a gene that produces a digestive enzyme called alpha-glucosidase (GAA) have been identified as the cause of Pompe disease. GAA’s normal function is to break down the body’s complex sugars.
In the United States, the therapy was approved for adults weighing forty kg (88 pounds) or more who are changing therapies due to a lack of improvement from their current treatment. The U.S. restriction is somewhat narrower than in the EU, where treatment of Pombiliti™ at any age is permissible. Pombiliti™ is the only dual-compound therapy currently available to treat enzyme replacement therapy patients. The Pombiliti GAA enzyme has been created for improved uptake into the muscle cells.
Opfolda stabilizes blood enzymes.
It’s worth noting that in a late-stage trial, patients were accepted who had not received prior ERT therapy.
Until the recent FDA approval, the French company Sanofi has dominated the market as the standard of care for Pompe disease. Although the Amicus therapy appears to be an improvement to the existing standard of care, according to financial analysts, it will still be a struggle for Amicus to acquire a significant market share.
The treatment involves Pombiliti, a synthetic protein, and Amicus’ oral drug Migalastat with the brand name Opfolda. The combination works together to aid in the breakdown of glycogen.
