Pharma Focus Europe

Amicus Therapeutics Unveils Newly Approved Pompe Disease Therapy in the European Union

Wednesday, June 28, 2023

Amicus Therapeutics, a biotechnology company dedicated to developing medicines for rare diseases, has received approval from the European Commission (EC) for Opfolda® (miglustat) 65mg capsules. Opfolda is an enzyme stabilizer used in the treatment of late-onset Pompe disease, a rare neuromuscular disorder caused by a deficiency of the enzyme acid α-glucosidase (GAA). The EC's approval follows their previous approval of Pombiliti® (cipaglucosidase alfa) in March 2023. Amicus plans to launch Pombiliti + Opfolda in Germany and begin reimbursement processes with healthcare authorities in other European countries.

Late-onset Pompe disease is a rare genetic disorder that can have severe consequences for patients and their families. The approval of Pombiliti and Opfolda by the EC is the result of the dedicated work of many individuals and teams focused on improving the lives of people with Pompe disease. Amicus is eager to provide this much-needed treatment to all adults with late-onset Pompe disease in the European Union.

Pombiliti + Opfolda is a two-component therapy consisting of Pombiliti, a bis-M6P-enriched rhGAA enzyme designed to improve uptake into muscle cells and break down glycogen, and Opfolda, 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 participants with late-onset Pompe disease who were either treatment-naïve or had previous enzyme replacement therapy (ERT) experience.

The approval of Pombiliti and Opfolda marks an important milestone in providing broad access to these treatments for the late-onset Pompe disease community in Europe, where there is a significant medical need for new therapeutic options. Clinical studies have shown that Pombiliti and Opfolda can lead to clinically meaningful improvements in mobility and respiratory function, offering hope for individuals living with Pompe disease.

Amicus anticipates regulatory approval for Pombiliti + Opfolda in the UK in the third quarter of 2023. The therapy is currently under review by the U.S. Food and Drug Administration (FDA), with expected approval also in the third quarter of 2023. The FDA had previously granted Breakthrough Therapy designation for Pombiliti + Opfolda.

Pombiliti + Opfolda represents a potential advancement in the treatment of Pompe disease, addressing the unmet needs of patients. Clinical studies have demonstrated improvements in musculoskeletal and respiratory measures, highlighting the potential of this innovative therapy.

Pompe disease is an inherited lysosomal disorder characterized by a deficiency of the enzyme acid alpha-glucosidase (GAA), resulting in the accumulation of glycogen in cells. The disease can manifest as an infantile form with rapid deterioration and significant impact on heart function, or as a late-onset form that primarily affects skeletal muscles and respiratory function, leading to progressive muscle weakness throughout the body.

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