Pharma Focus America

Amicus Therapeutics Secures Approval and Launches New Therapy for Pompe Disease in the European Union

Amicus Therapeutics, a global biotechnology company focused on rare diseases, has announced the approval of Opfolda® (miglustat) 65mg capsules by the European Commission (EC). Opfolda is an enzyme stabilizer used in combination with Pombiliti® (cipaglucosidase alfa), an enzyme replacement therapy for adults with late-onset Pompe disease. This approval marks a significant advancement in addressing the unmet needs of patients with this rare neuromuscular disorder.

Late-onset Pompe disease is caused by a deficiency of the enzyme acid α-glucosidase (GAA) and can have severe consequences for patients and their families. Amicus Therapeutics plans to launch Pombiliti + Opfolda in Germany immediately and is initiating reimbursement processes with healthcare authorities in other European countries. The approval of Pombiliti and Opfolda represents a collaborative effort aimed at improving the lives of those affected by Pompe disease.

Pombiliti + Opfolda is a unique two-component therapy. Pombiliti, also known as cipaglucosidase alfa, is an enzyme that is designed to enhance uptake into muscle cells and facilitate the breakdown of glycogen. Opfolda, on the other hand, is an enzyme stabilizer that helps maintain enzyme activity in the blood.

The EC approval was based on data from the Phase 3 pivotal study (PROPEL), which included both treatment-naïve and previously treated individuals with late-onset Pompe disease. Clinical studies have demonstrated that Pombiliti + Opfolda leads to improvements in musculoskeletal and respiratory measures.

Amicus Therapeutics' commitment to developing treatment options for late-onset Pompe disease has been well-received by the Pompe community. The approval of Pombiliti + Opfolda provides hope and alternative options for individuals living with this condition, offering them improved management of their disease.

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