FDA Issues Deferred Action Letter on AT-GAA Regulatory Filing Due to the Inability to Conduct Required Manufacturing Site Inspection Prior to the PDUFA Action Date
Company is Now Actively Engaged with the Agency to Develop Plans and Logistics for a Pre-Approval Inspection
PHILADELPHIA, Oct. 28, 2022 (GLOBE NEWSWIRE) — Amicus Therapeutics (Nasdaq: FOLD) right this moment introduced that the U.S. Food and Drug Administration (FDA) has deferred motion on the Biologics License Application (BLA) for cipaglucosidase alfa, the biologic element of AT-GAA. Due to restrictions on journey associated to COVID-19, the FDA was unable to conduct the required inspection of the WuXi Biologics manufacturing web site in China in the course of the evaluation cycle. As a consequence, the FDA is deferring motion on the applying till the manufacturing web site inspection is full. The Company continues to count on the FDA to approve the 2 elements of AT-GAA, together with the BLA and New Drug Application (NDA) for miglustat, collectively.
The sole cause cited in the FDA-issued letter for the deferred motion was the Agency’s incapacity to finish the manufacturing facility inspection. While each purposes stay below evaluation, the FDA has not supplied anticipated motion date(s) as they proceed to observe the general public well being scenario and journey restrictions in China. However, the Company is now actively engaged with the FDA on creating plans and logistics for a pre-approval inspection plan.
Under FDA steering referring to pre-approval inspections in the course of the COVID-19 pandemic, the Agency could defer motion on a pending utility when a facility inspection is important however can’t be accomplished by the PDUFA objective date attributable to journey restrictions, supplied that no deficiencies have been recognized and the applying in any other case satisfies the necessities for approval.1
“We are now one step away from the necessary approvals for AT-GAA in the U.S. We continue to believe this is a question of ‘when’ not ‘if’ AT-GAA will be approved and we will continue to work with great urgency to support the FDA’s completion of the final plant inspection necessary for approval so that this important new treatment option is made available for people living with Pompe disease in the United States,” stated Bradley Campbell, President and Chief Executive Officer at Amicus Therapeutics, Inc. “We are also very pleased with the progress of the regulatory review in the EU and look forward to a Committee for Medicinal Products for Human Use (“CHMP”) opinion by the top of the yr. We stay dedicated to bringing AT-GAA to as many individuals dwelling with Pompe illness all over the world as shortly as doable.”
Previously, the FDA granted Breakthrough Therapy Designation to AT-GAA for the remedy of late-onset Pompe illness primarily based on scientific efficacy outcomes from the Phase 1/2 scientific research. In the European Union, the place a pre-approval inspection isn’t required, the regulatory evaluation is on observe and the Committee for Medicinal Products for Human Use (CHMP) opinion is predicted earlier than yr finish.
About AT-GAA
AT-GAA is an investigational two-component remedy that consists of cipaglucosidase alfa (ATB200), a singular recombinant human acid alpha-glucosidase (rhGAA) enzyme with optimized carbohydrate constructions, significantly bis-phosphorylated mannose-6 phosphate (bis-M6P) glycans, to reinforce uptake into cells, administered in conjunction with miglustat (AT2221), a stabilizer of cipaglucosidase alfa.
About Pompe Disease
Pompe illness is an inherited lysosomal dysfunction attributable to deficiency of the enzyme acid alpha-glucosidase (GAA). Reduced or absent ranges of GAA ranges result in accumulation of glycogen in cells, which is believed to consequence in the scientific manifestations of Pompe illness. The illness could be debilitating and is characterised by extreme muscle weak spot that worsens over time. Pompe illness ranges from a quickly deadly childish kind with important impacts to coronary heart operate to a extra slowly progressive, late-onset kind primarily affecting skeletal muscle. It is estimated that Pompe illness impacts roughly 5,000 to 10,000 individuals worldwide.
About Amicus Therapeutics
Amicus Therapeutics (Nasdaq: FOLD) is a world, patient-dedicated biotechnology firm centered on discovering, creating and delivering novel high-quality medicines for individuals dwelling with uncommon illnesses. With extraordinary affected person focus, Amicus Therapeutics is dedicated to advancing and increasing a strong pipeline of cutting-edge, first- or best-in-class medicines for uncommon illnesses. For extra data please go to the corporate’s web site at www.amicusrx.com, and comply with on Twitter and LinkedIn.
Forward-Looking Statements
This press launch incorporates “forward-looking statements” throughout the that means of the Private Securities Litigation Reform Act of 1995, together with statements referring to expectations relating to the FDA regulatory course of, and the end result of the FDA’s evaluation and FDA’s capability to conduct a producing web site inspection. There could be no assurance that the FDA will grant approval for AT-GAA or the timing of any such approval. Words comparable to, however not restricted to, “look forward to,” “believe,” “expect,” “anticipate,” “estimate,” “intend,” “confidence,” “encouraged,” “potential,” “plan,” “targets,” “likely,” “may,” “will,” “would,” “should” and “could,” and comparable expressions or phrases determine forward-looking statements. The forward-looking statements included in this press launch are primarily based on administration’s present expectations and perception’s that are topic to a lot of dangers, uncertainties and components, together with that the Company will be unable to efficiently full the event of, receive regulatory approval for, or efficiently manufacture and commercialize AT-GAA. In addition, all ahead wanting statements are topic to the opposite dangers and uncertainties detailed in our Annual Report on Form 10-Okay for the yr ended December 31, 2021 and Quarterly Report 10-Q for the quarter ended June 30, 2022. As a consequence, precise outcomes could differ materially from these set forth in this press launch. You are cautioned to not place undue reliance on these forward-looking statements, which communicate solely of the date hereof. All ahead wanting statements are certified in their entirety by this cautionary assertion and we undertake no obligation to revise this press launch to replicate occasions or circumstances after the date hereof.
References
- U.S. Food and Drug Administration. Manufacturing, Supply Chain, and Drug Inspections – COVID-19. Available at: https://www.fda.gov/drugs/coronavirus-covid-19-drugs/manufacturing-supply-chain-and-drug-inspections-covid-19 Last accessed: October 2022.
CONTACTS:
Investors:
Amicus Therapeutics
Andrew Faughnan
Executive Director, Investor Relations
[email protected]
(609) 662-3809
Media:
Amicus Therapeutics
Diana Moore
Head of Global Corporate Communications
[email protected]
(609) 662-5079
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