After Amicus scores FDA nod for Pompe disease combo, it's game on with Sanofi

Slated for an FDA decision last October, Amicus Therapeutics’ Pompe disease bid was foiled by COVID-related travel restrictions.

Nearly a year later, the Philadelphia company has gained its long-awaited green light, as the FDA has signed off on Pombiliti and Opfolda as a two-component treatment for the rare genetic disorder.

The endorsement covers adults living with late-onset Pompe disease who are not improving on their current enzyme replacement therapy (ERT). While Pombiliti is an infused long-term ERT, Opfolda is an oral stabilizer, which reduces the loss of enzyme activity in the blood during the infusion.

With the nod for Pombiliti and Opfolda—on top of approvals this summer in Europe and the U.K.—Amicus is taking dead aim at Sanofi, which has cornered the Pompe disease market with two treatments that combined for sales of 620 million euros ($654 million) in the first half of this year.

“We ultimately see this becoming the therapy of choice for adults who are not improving on their current therapy,” Jeff Castelli, Amicus’ chief development officer, said in a Thursday conference call.

To Amicus, that means blockbuster potential. The company sees the Pompe disease market reaching $1.8 billion by 2027 and earmarks peak sales of its combo at $1.2 billion. The company is set to launch “immediately” in the U.S., where it has priced the treatment at $650,000 annually for a patient of median weight.  

“We believe we should price our medicine at no additional cost to payers, compared to similar competitor products,” Bradley Campbell, Amicus’ CEO, said during the call. “We will never raise the annual price of our products more than (the consumer price index).”

The key for Amicus to compete with Sanofi will be to convince ERT-experienced patients to switch to the combo, according to the company’s chief business officer Sebastien Martel, who estimates there are between 3,500 to 4,000 treated Pompe disease patients globally, with just 100 to 200 treatment-naive patients joining the group each year.

Sanofi’s longtime Pompe disease ERTs are Lumizyme—which is known as Myozyme outside the U.S. and was approved in 2006—followed by Nexviazyme, which was endorsed in 2021 for patients age 1 and older.

Pompe disease is a progressive muscle disorder caused by a deficiency of the enzyme acid alpha-glucosidase, which leads to a buildup of glycogen in cells. The disease ranges from a rapidly deteriorating infantile form, with significant impact on heart function, to a more slowly progressing, late-onset form which primarily affects skeletal muscle and respiratory function.

Amicus’ approval is based on results of the phase 3 PROPEL study, which compared its treatment to Sanofi’s Lumizyme using a six-minute walk test. At the 52-week mark, patients on Amicus’ combo could walk 21 meters farther than at baseline, compared to seven meters for those in the Lumizyme (alglucosidase alfa) arm.

While the result missed the bar for statistical significance, it will help Amicus push its product.

Amicus also is “working from a position of strength,” said the company’s head of global marketing, Mike Keavany, citing its successful launch of Fabry disease treatment Galafold, which was approved in 2018, and is expected to generate sales of between $340 million and $360 million this year.

Amicus was on track for the FDA approval almost a year ago. But FDA officials were unable to make the trip to China to inspect the plant of the company's contract manufacturer, WuXi Biologics.