FOLD - Amicus Opfolda combo for Pompe disease on track for EU approval after EMA nod

2023-04-28 05:30:40 ET

A committee of the European Medicines Agency (EMA) recommended the approval of Amicus Therapeutics' ( NASDAQ: FOLD ) oral therapy Opfolda (miglustat) in combination with cipaglucosidase alfa to treat glycogen storage disease type 2, or Pompe disease.

The EMA's Committee for Medicinal Products for Human Use (CHMP) said that the benefit of Opfolda is its ability to stabilize cipaglucosidase alfa, a recombinant human acid ??glucosidase, when given together in patients with late-onset Pompe disease.

Opfolda is a hybrid drug of Zavesca which has been approved in the EU since 2002. Opfolda contains the same active substance as Zavesca but in a lower strength, the CHMP added.

The CHMP noted that Opfolda is an enzyme stabilizer of cipaglucosidase alfa long-term enzyme replacement therapy in adults with late-onset Pompe disease (acid ??glucosidase (GAA) deficiency).

European Commission (EC) will now make a final decision on the treatment's approval.

In March, the EC approved Amicus's Pombiliti (cipaglucosidase alfa), a long-term enzyme replacement therapy (ERT), used in combination with miglustat for adults with late-onset Pompe disease.

The company had noted in its March 27 release that it was looking forward to CHMP's opinion on the analytical testing for miglustat.

Pompe disease is an inherited lysosomal disorder caused by deficiency of the enzyme GAA.

For further details see: