Amicus Therapeutics announced Prescription Drug User Fee Act action dates of May 29, 2022, for the New Drug Application and July 29, 2022, for the Biologic License Application regarding the 2-component cipaglucosidase alfa/miglustat (AT-GAA) therapy for Pompe disease (PD).
The company noted that the US Food and Drug Administration and European Union regulatory reviews of AT-GAA are progressing. Launch preparations for AT-GAA continue, and these activities include adding to personnel and inventory in the expectation that AT-GAA will become the new standard of care treatment for patients with PD.
“We are committed to the regulatory approvals of AT-GAA for people living with [PD], first in the United States and then in Europe and beyond,” said John F. Crowley, chairman, and chief executive officer of Amicus.
“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 [PD].”
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The announcement highlighted the encouraging data from the long-term phase 1/2 clinical trials of AT-GAA showing persistent and durable effects of the treatment on the 6-Minute Walk Test scores, stability, and increased forced vital capacity, as well as reduced biomarkers of disease substrate and muscle damage in patients with PD for up to 36 months.
The Committee for Medicinal Products for Human Use is expected to issue an opinion on AT-GAA in late 2022. In addition, under the Early Access to Medicines Scheme in the United Kingdom, physicians in all the leading PD centers have requested access to the new treatment.
Amicus Therapeutics announces first quarter 2022 financial results. News release. Amicus Therapeutics; May 9, 2022.