Amicus Puts Positive Spin on PDUFA Delay for Pompe Treatment

FDA_Sarah Silbiger/Getty Images

Sarah Silbiger/Getty Images

Tuesday brought frustrating news for Amicus Therapeutics as the U.S. Food and Drug Administration pushed back the target action dates for the company’s Biologics License Application (BLA) for cipaglucosidase alfa and New Drug Application (NDA) for miglustat by 90 days. Together they are being developed as a treatment for Pompe disease.

In tandem, these drugs make up a two-component therapy called AT-GAA. Cipaglucosidase alfa is a unique recombinant human acid alpha-glucosidase (rhGAA) enzyme that optimizes carbohydrate structures, especially bis-phosphorylated mannose-6 phosphate (bis-M6P) glycans, to improve uptake into cells. Miglustat is a stabilizer of cipaglucosidase alfa.

The revised PDUFA action date for miglustat is August 29, 2022, while the new date for cipaglucosidase alfa is October 29, 2022. Amicus expects the agency to approve the applications together.

The FDA indicated it extended the target action dates to allow more time to review the information submitted by the company. It was not related to any request for more information by the regulator. Amicus said the additional time will allow more time to complete the pre-license approval inspections required at the WuXi Biologics manufacturing site in China.

“We continue to work collaboratively with the FDA as it completes its review of the AT-GAA applications,” stated John F. Crowley, chairman and chief executive officer at Amicus. “We want to thank the FDA for its continued diligence during the review process. We remain deeply committed to bring AT-GAA to as many people living with Pompe disease as quickly as possible and delivering on our promise to become the potential new standard of care.”

Pompe disease is an inherited lysosomal disease resulting from a deficiency of the enzyme acid alpha-glucosidase (GAA). This drop in GAA leads to the accumulation of glycogen in cells. The disease can be debilitating and is marked by severe muscle weakness that grows worse over time. It ranges from a rapidly fatal infantile form with significant effects on heart function to a slowly progressive, late-onset type primarily affecting skeletal muscle. It affects about 5,000 to 10,000 people globally.

Amicus, based in Philadelphia, announced its first-quarter financial results on Monday. It reported first-quarter revenue of Fabry disease drug Galafold (migalastat) of $78.8 million, an increase year-over-year of 18.5% from the first quarter of 2021. That represented total revenue for the quarter.

The company reported cash, cash equivalents and marketable securities of $411.2 million as of March 31, 2022. Total GAAP operating expenses for the quarter were $146.5 million. Amicus reported a net loss of $85.3 million, or $0.30 per share.

Amicus projects full-year Galafold revenue of $350 to $365 million. Fabry disease patients don’t have the enzymes that break down lipids or fat. They then collect in blood vessels and tissue, increasing the risk of heart attack, stroke and kidney failure. Specifically, they don’t produce normal versions of alpha-galactosidase a (alpha-GAL), which prevent sphingolipids from collecting in the body. It is an inherited condition, sometimes called Anderson-Fabry disease.

Crowley said, “Against a tumultuous market, we at Amicus have a deeply sharpened focus. We continue to ensure that Galafold gets to as many amenable people living with Fabry disease around the world, as seen by the significant first-quarter performance of this precision medicine. We are committed to the regulatory approvals of AT-GAA for people living with Pompe disease, first in the United States and then in Europe and beyond. We are well on track with these anticipated approvals and the associated launch preparations, furthering our belief in the potential for this treatment regimen to become the new global standard of care in Pompe disease.”

In the European Union, the company expects the Committee for Medicinal Products for Human Use (CHMP) will produce a positive opinion for its submission for AT-GAA in late 2022.

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