FDA approves Amicus Therapeutics’ combo therapy for Pompe disease

The FDA on Thursday approved Amicus Therapeutics’ combo therapy for a rare inherited lysosomal disorder called Pompe disease following several regulatory delays.

The drugs are marketed as Pombiliti (cipaglucosidase alfa) and Opfolda (miglustat). Pombiliti is a long-term enzyme replacement therapy and Opfolda is an enzyme stabilizer. The therapy is already approved in the EU, and the UK’s National Institute for Health and Care Excellence recommended it last month as a first-line therapy as well as in later lines of treatment after it received approval there.

The FDA approved it for patients with late-stage disease who are not improving on their current enzyme replacement therapy.

John Crowley

“Today’s FDA approval of Pombiliti and Opfolda is a testament to the power of science, medicine, and our passionate determination to improve the lives of people living with Pompe disease,” Amicus executive chairman John Crowley said in a statement.

The company added that it will launch the combo therapy “immediately” in the US.

Pompe disease causes progressive weakness in the heart and skeletal muscles because of an enzyme deficiency. It’s traditionally treated with other enzyme replacement therapies like Sanofi’s Lumizyme, according to the National Institute of Neurological Disorders and Stroke.

Regulatory roadblocks and questions about efficacy have delayed the therapy’s road to approval, which includes a BLA for cipaglucosidase alfa and an NDA for miglustat.

The FDA’s decision has been delayed several times. Back in May 2022, Amicus said the regulator had pushed back its review period by 90 days because it was still reviewing newly-submitted information.

In October 2022, the FDA again delayed its decision for the therapy, citing its inability to complete a manufacturing inspection in China because of travel restrictions. That inspection has since been completed.

Then came the Phase III data that showed that Amicus’ treatment did not beat out Lumizyme. Pombiliti and Opfolda earned a p-value of p=0.072 in the study’s primary endpoint — the mean change in a six-minute walk distance test — and didn’t hit statistical significance.

Amicus pushed forward with the filing, which included data from its extension PROPEL study. The company said that data showed “durable mean improvements” in the walk distance test, stable lung function and reductions in muscle damage in patients who were on the therapy for up to 104 weeks.

Leerink Partners analysts in August predicted $8.8 million in sales for the therapy this year, assuming there are about 64 patients on commercial therapy by the end of 2023. Leerink is also predicting sales of $10 million in the EU if approximately 72 patients are on the therapy by the end of the year. Amicus has already begun the product’s launch in Germany.

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