FDA Again Extends Review for Pompe Disease Therapy
The FDA cited COVID-19-related travel restrictions that have impacted the agency’s inspection of a manufacturing site in China.
The FDA has deferred action on Amicus Therapeutics’ biologics license application (BLA) for cipaglucosidase alfa, the biologic component of AT-GAA. Due to restrictions on travel related to COVID-19, the FDA was unable to conduct the required inspection of the WuXi Biologics manufacturing site in China during the review cycle.
AT-GAA is a therapeutic to treat patients with Pompe Disease, a genetic lysosomal disorder caused by deficiency of the enzyme acid alpha-glucosidase (GAA) that leads to severe muscle weakness that worsens over time. AT-GAA contains two components: cipaglucosidase alfa, a recombinant human acid alpha-glucosidase (rhGAA) enzyme, and miglustat, a stabilizer of cipaglucosidase alfa.
Amicus officials expect the FDA to approve the two components of AT-GAA, including the BLA for cipaglucosidase alfa and a new drug application for miglustat, together.
Related: FDA Extends Review Time for Pompe Disease Therapy
This is second time action this therapeutic has been delay. In May 2022, the FDA had extended the review of this BLA by 90 days to allow additional time to review information submitted by the company as part of its ongoing reviews.
In this episode of the "Meet the Board" podcast series, Briana Contreras, Managed Healthcare Executive editor, speaks with Ateev Mehrotra, a member of the MHE editorial advisory board and a professor of healthcare policy and medicine at Harvard Medical School. Mehtrotra is also a hospitalist at the Beth Israel Deaconess Medical Center in Boston. In the discussion, Contreras gets to know Mehrotra more on a personal level and picks his brain on some of his research interests including telehealth, alternative payment models and price transparency.
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