Agios Announces FDA Acceptance and Priority Review of New Drug Application for Mitapivat for Treatment of Adults with Pyruvate Kinase Deficiency
August 17 2021 - 4:01PM
Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field
of cellular metabolism developing and delivering innovative
treatments for genetically defined diseases, today announced that
the U.S. Food and Drug Administration (FDA) has accepted the
company’s New Drug Application (NDA) for mitapivat for the
treatment of adults with pyruvate kinase (PK) deficiency. The NDA
was granted a Priority Review designation and has been given a
Prescription Drug User Fee Act (PDUFA) action date of February 17,
2022, accelerating the review time from 10 months to six months
from the day of filing acceptance. The FDA’s Priority Review
designation is given to investigational medicines that treat a
serious condition and offer significant improvements in safety or
effectiveness.
“The acceptance of our NDA for mitapivat with priority review
represents an important milestone on the path to expeditiously
deliver the first potentially disease-modifying therapy for people
with PK deficiency, a chronic, lifelong hemolytic anemia
characterized by serious complications affecting multiple organs,”
said Sarah Gheuens, M.D., Ph.D., senior vice president of clinical
development and incoming chief medical officer at Agios. “We look
forward to working with the FDA during the review process and will
continue to execute on our global strategy to ensure we are well
positioned to rapidly deliver mitapivat to patients and healthcare
providers upon approval.”
Agios also submitted a marketing authorization application (MAA)
to the European Medicines Agency (EMA) in June 2021 for mitapivat
as a potential treatment for adults with PK deficiency. As
announced on the company’s second quarter 2021 earnings call, the
MAA passed validation which triggered the start of the MAA review
procedure.
The NDA and MAA submissions are based on results from two
pivotal studies, ACTIVATE and ACTIVATE-T, conducted in not
regularly transfused and regularly transfused adults with PK
deficiency, respectively. A full analysis of these data –
including patient-reported outcomes (PRO) – was recently presented
at the European Hematology Association (EHA) Virtual Congress.
An extension study for adults with PK deficiency previously
enrolled in ACTIVATE or ACTIVATE-T is ongoing and designed to
evaluate the long-term safety, tolerability and efficacy of
treatment with mitapivat.
Mitapivat is not currently approved for use in any country.
About PK DeficiencyPyruvate kinase (PK)
deficiency is a rare, inherited disease that presents as chronic
hemolytic anemia, which is the accelerated destruction of red blood
cells. The inherited mutations in PKR genes cause a deficit in
energy within the red blood cell, as evidenced by lower PK enzyme
activity, a decline in adenosine triphosphate (ATP) levels and a
build-up of upstream metabolites, including 2,3-DPG
(2,3-diphosphoglycerate).
PK deficiency is associated with serious complications,
including gallstones, pulmonary hypertension, extramedullary
hematopoiesis, osteoporosis and iron overload and its sequelae,
which can occur regardless of the degree of anemia or transfusion
burden. PK deficiency can also cause quality of life problems,
including challenges with work and school activities, social life
and emotional health. Current management strategies for PK
deficiency, including red blood cell transfusions and splenectomy,
are associated with both short- and long-term risks. There are no
currently approved therapies for PK deficiency. For more
information, please visit www.knowpkdeficiency.com.
Agios, in partnership with PerkinElmer Genomics, launched the
Anemia ID program to offer no-cost genetic testing to eligible
patients in the U.S with suspected hereditary anemias,
including PK deficiency. The program was created in response to
feedback from patients, advocates and physicians about the need for
improved diagnosis to inform disease management decisions. To learn
more, please visit www.AnemiaID.com.
Agios also launched the myAgios® patient support services
program for people living with pyruvate kinase (PK) deficiency and
their caregivers. After enrolling in the program, patients and
caregivers are connected with a dedicated Patient Support Manager
(PSM) with a clinical background to provide tailored support,
educational resources and opportunities to connect with other
patients and caregivers in the community. To learn more or enroll,
please visit www.myagios.com.
About AgiosAgios is focused on discovering and
developing novel investigational medicines to treat genetically
defined diseases through scientific leadership in the field of
cellular metabolism. The company’s most advanced drug candidate is
a first-in-class pyruvate kinase R (PKR) activator, mitapivat, that
is currently being evaluated for the treatment of three distinct
hemolytic anemias. In addition to its active late-stage clinical
pipeline, Agios has multiple novel, investigational therapies in
clinical and preclinical development. For more information, please
visit the company’s website at www.agios.com.
Cautionary Note Regarding Forward-Looking
StatementsThis press release contains forward-looking
statements within the meaning of The Private Securities Litigation
Reform Act of 1995. Such forward-looking statements include those
regarding Agios’ expectations for the FDA’s review of its NDA for
mitapivat. The words “expects,” “anticipates,” “believes,”
“intends,” “estimates,” “plans,” “will,” “outlook” and similar
expressions are intended to identify forward-looking statements,
although not all forward-looking statements contain these
identifying words. Such statements are subject to numerous
important factors, risks and uncertainties that may cause actual
events or results to differ materially from Agios’ current
expectations and beliefs. For example, the FDA’s acceptance of
Agios’s NDA for mitapivat does not represent evaluation of the
efficacy and safety of mitapivat, and is not a guarantee of
approval. Management’s expectations and, therefore, any
forward-looking statements in this press release could also be
affected by risks and uncertainties relating to a number of other
important factors, including: risks associated with the regulatory
review process generally; the risk that the FDA may
determine that the data included in the NDA are insufficient for
approval and that the Company must conduct additional clinical
trials, or nonclinical or other studies, before mitapivat can be
approved; the risk that the results of previously conducted studies
involving mitapivat will not be repeated or observed in ongoing or
future studies or following commercial launch, if mitapivat is
approved; and risks associated with the Company’s dependence on
third parties with respect to regulatory matters for mitapivat.
These and other risks are described in greater detail under the
caption “Risk Factors” included in Agios’ public filings with
the Securities and Exchange Commission, or SEC. While the
list of factors presented here is considered representative, this
list should not be considered to be a complete statement of all
potential risks and uncertainties. Any forward-looking statements
contained in this communication are made only as of the date
hereof, and we undertake no obligation to update forward-looking
statements to reflect developments or information obtained after
the date hereof and disclaim any obligation to do so other than as
may be required by law.
Contacts
Investors: 1AB Steve
Klasssteve@1abmedia.com
Media: Jessi Rennekamp, 857-209-3286Director,
Corporate CommunicationsJessica.Rennekamp@agios.com
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