European Union Grants Approval for Amicus’s Therapy for Adults with Late-onset Pompe Disease

European Union Grants Approval for Amicus’s Therapy for Adults with Late-onset Pompe Disease

Rare Daily Staff

The European Commission has granted approval to Amicus Therapeutics Opfolda, a long-term enzyme replacement therapy for adults with late-onset Pompe disease.

Pompe disease is an inherited lysosomal disorder caused by deficiency of the enzyme acid alpha-glucosidase (GAA). Reduced or absent levels of GAA lead to accumulation of glycogen in cells, which is believed to result in the clinical manifestations of Pompe disease. Pompe disease ranges from a rapidly deteriorating infantile form with significant impact to heart function, to a more slowly progressive, late-onset form primarily affecting skeletal muscle and progressive respiratory involvement. Late-onset Pompe disease can be severe and debilitating with progressive muscle weakness throughout the body that worsens over time, particularly skeletal muscles and muscles controlling breathing.

Opfolda is an enzyme stabilizer of Amicus’ Pombiliti, which was previously approved by the EC in March 2023. Pompe disease is an inherited lysosomal disorder caused by deficiency of the enzyme acid α-glucosidase (GAA). Amicus plans to immediately launch Pombiliti + Opfolda in Germany and is commencing reimbursement processes with healthcare authorities in other European countries.

“Given the strength of the label and our launch readiness, we believe Pombiliti and Opfolda has the potential to become the next standard of care in this devastating condition by showing that improvement is possible for people living with late-onset Pompe disease,” said Bradley Campbell, president and CEO of Amicus Therapeutics.

Pombiliti + Opfolda is a two-component therapy. Pombiliti, 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 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-naïve 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 2023. The FDA previously granted Breakthrough Therapy designation for Pombiliti + Opfolda.

Photo: Bradley Campbell, president and CEO of Amicus Therapeutics