Agios Submits Marketing Authorisation Application to European Medicines Agency for Mitapivat for Treatment of Adults with Pyr...
June 28 2021 - 7:00AM
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
it has submitted a Marketing Authorisation Application (MAA) for
mitapivat to the European Medicines Agency (EMA) for the treatment
of adults with pyruvate kinase (PK) deficiency in the European
Union. This submission follows the company’s recent New Drug
Application (NDA) submission of mitapivat to the U.S. Food and Drug
Administration (FDA) for treatment of adults with PK deficiency in
the U.S.
“With both our NDA and MAA filings, we are poised to 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 Chris
Bowden, M.D., chief medical officer at Agios. “There are no
approved therapies for PK deficiency, and the current management
strategies of blood transfusions and splenectomy are associated
with both short- and long-term risks, including iron overload,
blood clots and increased risk for infections. PK deficiency can
lead to chronic fatigue, hemolytic crisis, gallstones,
splenomegaly, liver cirrhosis, pulmonary hypertension and
osteoporosis, and the burden of disease can take a toll on
patients’ ability to navigate work and other day-to-day activities,
as well as on their mental health. We are looking forward to
working with both the FDA and EMA to provide a potential new
treatment option for this community that may help address the
significant unmet needs of PK deficiency patients.”
Like the NDA, the MAA submission is 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 approved for use by any regulatory
authority.
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.
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 the potential benefits of Agios’ products and product
candidates; Agios’ plans for future regulatory submissions; and the
potential benefits of Agios’ strategic plans and focus. The words
“anticipate,” “expect,” “goal,” “hope,” “milestone,” “plan,”
“potential,” “possible,” “strategy,” “will,” “vision,” 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. 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, without limitation, risks and
uncertainties related to: the impact of the COVID-19 pandemic to
Agios’ business, operations, strategy, goals and anticipated
milestones, including its ongoing and planned research activities,
ability to conduct ongoing and planned clinical trials, clinical
supply of current or future drug candidates, commercial supply of
future approved products, and launching, marketing and selling
future approved products; Agios’ results of clinical trials and
preclinical studies, including subsequent analysis of existing data
and new data received from ongoing and future studies; the content
and timing of decisions made by the U.S. FDA, the EMA or
other regulatory authorities, investigational review boards at
clinical trial sites and publication review bodies; Agios’ ability
to obtain and maintain requisite regulatory approvals and to enroll
patients in its planned clinical trials; unplanned cash
requirements and expenditures and competitive factors; Agios’
ability to obtain, maintain and enforce patent and other
intellectual property protection for any product candidates it is
developing; Agios’ ability to establish and maintain
collaborations; and general economic and market conditions. 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, including
the risks and uncertainties set forth under the heading Risk
Factors in our filings with the 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: 1AB Josie Butlerjosie@1abmedia.com
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