The European Commission (EC) has approved Amicus Therapeutics’ Pompe disease therapy, Opfolda (miglustat), for adults with late-onset disease.
The authorisation allows the company to offer the enzyme stabiliser together with its previously-approved long-term enzyme replacement therapy, Pombiliti (cipaglucosidase alfa).
Pompe disease is a rare, progressive and debilitating muscle disorder caused by a deficiency of the enzyme acid alpha-glucosidase (GAA), which leads to a build-up of glycogen in cells.
The disease ranges from a rapidly deteriorating infantile form with significant impact on heart function to a more slowly progressive, late-onset form primarily affecting skeletal muscle and progressive respiratory involvement.
Amicus describes Pombiliti plus Opfolda as a “unique two-component therapy”. Pombiliti is a bis-M6P-enriched rhGAA that facilitates high uptake into muscle cells while retaining its capacity for processing into the most active form of the enzyme, while Opfolda is designed to reduce loss of enzyme activity in the blood.
Bradley Campbell, president and chief executive officer of Amicus, said: “We are extremely pleased with the EC approval of Pombiliti and Opfolda, and are grateful to the Pompe community around the world who have helped advance this therapy.
“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.”
The EC’s decision was based on positive results from the phase 3 PROPEL study, in which the combination exhibited clinically meaningful and positive changes in the key mobility and respiratory manifestations of the disease.
The company outlined that regulatory decisions for use of the therapy in the UK and US are expected in the third quarter of this year, adding that the US Food and Drug Administration has previously granted Breakthrough Therapy designation for the combination.
Tiffany House, president of the International Pompe Association, said: “The Pompe community is greatly appreciative of Amicus’ long-standing commitment to develop a treatment option to address the continuing unmet needs of people living with late-onset disease. Each person deserves alternatives to help them best manage their condition.”