Opfolda is intended to be used as part of a two-part therapy, which includes Pombiliti (cipaglucosidase alfa). Image credit: Shutterstock/Sonia Bonet

Amicus Therapeutics announced that the Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for Opfolda (miglustat) intended for treatment of Pompe Disease.

The CHMP used evidence from a Phase III trial (NCT03729362) involving 123 patients with late-onset Pompe Disease (LOPD) to adopt a positive opinion for Opfolda. A decision from the European Commission (EC) is expected in Q3 2023. Yesterday, the EC released broad reforms, which included a stipulation that the EC will award approval decisions in 46 days after a CHMP opinion, instead of 67 days.

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Pompe Disease, also known as glycogen storage type II, is a rare inherited lysosomal disorder caused by a deficiency of enzyme acid alpha-glucosidase (GAA) leading to the accumulation of glycogen in skeletal and cardiac muscles.

Opfolda is intended to be used as part of a two-part therapy, which includes Pombiliti (cipaglucosidase alfa), the biologic component, which is an enzyme replacement therapy (ERT) to help break down glycogen. Opfolda is a pharmacokinetic enzyme stabilizer that minimises the loss of cipaglucosidase alfa’s activity. If approved, this will become the first two-component therapy approved in the European Union for late-onset Pompe Disease (LOPD).

The recommendation follows Pombilit’s EC approval in March 2023. The treatment will go up against Sanofi’s Nexviazyme (avalglucosidase alfa-ngpt) which received an EC approval in June 2022, following a US Food and Drug Administration (FDA) approval in August 2021. Sanofi markets avalglucosidase alfa as Lumizyme in the US to treat LOPD since its approval in 2010.

Sanofi’s Nexviazyme generated €196m ($215m) in global revenues in 2022, while Lumizyme was associated with €958m ($1.05bn) according to the company’s full-year report. The market for Pompe disease is expected to grow as new therapies get approved.

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“Based on the strength of the label and our launch readiness, once fully approved we believe there is significant opportunity to bring Pombiliti and Opfolda as the first two-component therapy for adult LOPD patients in Europe, and to establish this novel treatment combination as a potential new standard of care in Pompe disease,” said Bradley Campbell, president and CEO of Amicus Therapeutics.