European Medicines Agency Validates Amicus Therapeutics Marketing Authorization Applications for AT-GAA for the Treatment of Pompe Disease
December 03 2021 - 7:00AM
Amicus Therapeutics (Nasdaq: FOLD) today announced that the
European Medicines Agency (EMA) validated the Marketing
Authorization Applications (MAA) for AT-GAA, the Company’s
investigational two-component therapy for the treatment of Pompe
disease. Validation of the application confirms the submission is
accepted, and the EMA’s centralized procedure with Committee for
Medicinal Products for Human Use (CHMP)’s assessment begins.
The MAAs were submitted to the EMA based on the
evaluation of the effects of AT-GAA in adults living with Pompe
disease and its safety profile, which include data from the Phase
1/2 and Phase 3 PROPEL studies, as well as data from the long-term
open-label extension study.
John F. Crowley, Chairman and Chief Executive
Officer of Amicus Therapeutics Inc., stated, “The acceptance of
these filings is an important step forward for people living with
Pompe disease and their families in Europe. Patients need new
medicines as soon as possible. We will work with great urgency with
the EMA as they review the applications over the course of the
coming months. With today’s announcement, we remain confident in
the potential of this medicine to become the next standard of care
in Pompe disease.”
The U.S. Food and Drug Administration (FDA)
previously granted Breakthrough Therapy designation for AT-GAA and
accepted for review the Biologics License Application (BLA) and the
New Drug Application (NDA). The FDA has set a Prescription Drug
User Fee Act action date of May 29, 2022 for the NDA and July 29,
2022 for the BLA.
About AT-GAAAT-GAA is an
investigational two-component therapy that consists of
cipaglucosidase alfa (ATB200), a recombinant human acid
alpha-glucosidase (rhGAA) enzyme with optimized carbohydrate
structures, particularly bis-phosphorylated mannose-6 phosphate
(bis-M6P) glycans, to enhance uptake into cells, administered in
conjunction with miglustat (AT2221), a stabilizer of
cipaglucosidase alfa. In preclinical studies, AT-GAA was associated
with increased levels of the mature lysosomal form of GAA and
reduced glycogen levels in muscle, alleviation of the autophagic
defect and improvements in muscle strength.
About Pompe DiseasePompe
disease is an inherited lysosomal disorder caused by
deficiency of the enzyme acid alpha-glucosidase (GAA). Reduced or
absent levels of GAA levels lead to accumulation of glycogen in
cells, which is believed to result in the clinical manifestations
of Pompe disease. The disease can be debilitating and is
characterized by severe muscle weakness that worsens over time.
Pompe disease ranges from a rapidly fatal infantile form with
significant impacts to heart function to a more slowly progressive,
late-onset form primarily affecting skeletal muscle. It is
estimated that Pompe disease affects approximately 5,000 to 10,000
people worldwide.
About Amicus Therapeutics
Amicus Therapeutics (Nasdaq: FOLD) is a global, patient-dedicated
biotechnology company focused on discovering, developing and
delivering novel high-quality medicines for people living with rare
metabolic diseases. With extraordinary patient focus, Amicus
Therapeutics is committed to advancing and expanding a robust
pipeline of cutting-edge, first- or best-in-class medicines for
rare metabolic diseases. For more information please visit the
company’s website at www.amicusrx.com, and follow us on Twitter and
LinkedIn.
Forward Looking Statement
This press release contains "forward-looking
statements" within the meaning of the Private Securities Litigation
Reform Act of 1995, including statements relating to regulatory
submissions for AT-GAA and the status of those submissions. There
can be no assurance that the FDA or EMA will grant approval for
AT-GAA. Words such as, but not limited to, “look forward to,”
“believe,” “expect,” “anticipate,” “estimate,” “intend,”
“confidence,” “encouraged,” “potential,” “plan,” “targets,”
“likely,” “may,” “will,” “would,” “should” and “could,” and similar
expressions or words identify forward-looking statements. The
forward looking statements included in this press release are based
on management's current expectations and belief's which are subject
to a number of risks, uncertainties and factors, including that the
Company will not be able to successfully complete the development
of, obtain regulatory approval for, or successfully manufacture and
commercialize AT-GAA. In addition, all forward looking statements
are subject to the other risks and uncertainties detailed in our
Annual Report on Form 10-K for the year ended December 31, 2020 and
10-Q for the quarter ending Sept. 30, 2021. As a consequence,
actual results may differ materially from those set forth in this
press release. You are cautioned not to place undue reliance on
these forward-looking statements, which speak only of the date
hereof. All forward looking statements are qualified in their
entirety by this cautionary statement and we undertake no
obligation to revise this press release to reflect events or
circumstances after the date hereof.
CONTACT:
Investors:Andrew
FaughnanExecutive Director, Investor
Relationsafaughnan@amicusrx.com(609) 662-3809
Media:Diana MooreHead of Global
Corporate Affairs & Communicationsdmoore@amicusrx.com(609)
662-5079
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