Agios Receives Positive Opinion on Orphan Drug Designation from the European Medicines Agency for Mitapivat in Pyruvate Kinas...
March 30 2020 - 4:01PM
Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field
of cellular metabolism to treat cancer and rare genetic diseases,
today announced that the European Medicines Agency (EMA) Committee
for Orphan Medicinal Products issued a positive opinion on the
company’s application for orphan drug designation for its
investigational medicine mitapivat as a potential treatment for
pyruvate kinase (PK) deficiency, a rare, debilitating, hemolytic
anemia. Mitapivat was previously granted orphan drug designation by
the United States Food and Drug Administration. Mitapivat is an
investigational, first-in-class, oral, small molecule allosteric
activator of wild-type and a variety of mutated pyruvate kinase-R
(PKR) enzymes that directly targets the underlying metabolic defect
in PK deficiency.
“We are pleased to receive a positive opinion from EMA for
orphan drug designation for mitapivat as a recognition of the need
for innovative treatments for patients with PK deficiency, a
serious lifelong disease characterized by hemolytic anemia that can
lead to severe symptoms including iron overload, splenomegaly and
heart failure,” said Chris Bowden, M.D., chief medical officer at
Agios. “Both of our pivotal trials are fully enrolled, and we look
forward to the completion of these trials intended to support
global regulatory filings. Mitapivat is the first and only PKR
activator to receive this important designation in Europe and the
first potential disease-modifying treatment for PK deficiency
patients.”
Orphan drug designation in the European Union (EU) is granted by
the European Commission based on a positive opinion issued by the
EMA Committee for Orphan Medicinal Products. To qualify, an
investigational medicine must be intended to treat a seriously
debilitating or life-threatening condition that affects fewer than
five in 10,000 people in the EU, and there must be sufficient
non-clinical or clinical data to suggest the investigational
medicine may produce clinically relevant outcomes. EMA orphan drug
designation provides companies with certain benefits and
incentives, including clinical protocol assistance, differentiated
evaluation procedures for Health Technology Assessments in certain
countries, access to a centralized marketing authorization
procedure valid in all EU member states, reduced regulatory fees
and 10 years of market exclusivity.
Mitapivat Clinical Development Agios has two
ongoing global, pivotal trials in adults with PK deficiency that
are fully enrolled. Learn more at activatetrials.com.
- ACTIVATE: A placebo-controlled trial with a 1:1 randomization
evaluating patients who do not receive regular transfusions. The
primary endpoint of the trial is the proportion of patients who
achieve a sustained hemoglobin increase of ≥1.5 g/dL.
- ACTIVATE-T: A single arm trial of regularly transfused patients
with a primary endpoint of reduction in transfusion burden over six
months compared to individual historical transfusion burden over
prior 12 months.
In addition, Agios is conducting a Phase 2 study evaluating the
efficacy, safety, pharmacokinetics and pharmacodynamics of
treatment with mitapivat in adults with non-transfusion-dependent
β- and α-thalassemia (NTDT). The trial is fully enrolled, and the
primary endpoint is hemoglobin response. Preliminary Phase 2 data
establishing proof-of-concept for mitapivat in thalassemia were
disclosed at the end of 2019. Mitapivat is also being studied in
sickle cell disease under a Cooperative Research and
Development Agreement (CRADA) with the U.S. National
Institutes of Health.
Mitapivat is not approved for use by any regulatory
authority.
About AgiosAgios is focused on discovering and
developing novel investigational medicines to treat malignant
hematology, solid tumors and rare genetic diseases through
scientific leadership in the field of cellular metabolism. In
addition to an active research and discovery pipeline across these
three therapeutic areas, Agios has two approved oncology precision
medicines and multiple first-in-class investigational therapies in
clinical and/or 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 mitapivat; Agios’ plans
regarding future data presentations; and the benefit of Agios’
strategic plans and focus. The words "expects," "anticipates,"
"believes," "intends," "estimates," "plans," "will," "outlook,"
"goal", "potential" 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, a positive opinion on Agios’ application for orphan drug
designation for mitapivat 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: the results of Agios’ 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 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 and conduct its current and future
clinical trials; unplanned cash requirements and expenditures;
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 maintain key
collaborations; and general economic, market and global health
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. Any
forward-looking statements contained in this press release speak
only as of the date hereof, and Agios expressly disclaims any
obligation to update any forward-looking statements, whether as a
result of new information, future events or otherwise, except as
required by law.
Contacts
Investors:Holly Manning, 617-844-6630Director,
Investor RelationsHolly.Manning@agios.com
Media:Jessica Rennekamp, 857-209-3286Associate
Director, Corporate CommunicationsJessica.Rennekamp@agios.com
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