Sotatercept granted first FDA Breakthrough
Therapy designation in pulmonary arterial hypertension since the
Agency established the designation in 2012
Acceleron Pharma Inc. (Nasdaq: XLRN), a biopharmaceutical
company dedicated to the discovery, development, and
commercialization of TGF-beta superfamily therapeutics to treat
serious and rare diseases, today announced that the United States
Food and Drug Administration (FDA) has granted Breakthrough Therapy
designation to sotatercept for the treatment of patients with
pulmonary arterial hypertension (PAH) (World Health Organization
Group 1).
“In January of this year, we reported positive topline results
from our PULSAR Phase 2 placebo-controlled trial of sotatercept in
patients with PAH,” said Habib Dable, President and Chief Executive
Officer of Acceleron. “Based on the results, we believe that
sotatercept has the potential to shift the current treatment
paradigm and provide significant benefit to patients with PAH on
top of currently available therapies. Thus, we’re thrilled that the
FDA has granted this Breakthrough Therapy designation—a first for
an Acceleron-discovered medicine and for a therapeutic candidate in
PAH—as it supports and aligns with our mission to deliver novel
therapeutic options to patients in need as quickly as
possible.”
The FDA’s Breakthrough Therapy designation is intended to
expedite the development and review of drugs for serious or
life-threatening conditions. The criteria for Breakthrough Therapy
designation require preliminary clinical evidence that demonstrates
the drug may provide substantial improvement on at least one
clinically significant endpoint over available therapy. A
Breakthrough Therapy designation conveys more intensive FDA
guidance on an efficient drug development program, an
organizational commitment involving senior managers, and
eligibility for rolling review and priority review. For more
information please visit the FDA website at www.fda.gov.
In 2019, the FDA granted Orphan Drug designation for Sotatercept
in PAH.
About Sotatercept
Sotatercept is an investigational agent designed to be a
selective ligand trap for members of the TGF-beta superfamily to
rebalance BMPR-II signaling, which is a key molecular driver of
PAH. In preclinical studies of PAH, sotatercept reversed pulmonary
vessel muscularization and improved indicators of right heart
failure. Recent topline analysis of the PULSAR Phase 2 trial of
sotatercept in patients with PAH revealed the trial met the primary
as well as key and other secondary endpoints, with adverse events
consistent with previously published data on sotatercept in other
diseases. Sotatercept, which is part of a licensing agreement with
Bristol Myers Squibb, is also being evaluated in the SPECTRA Phase
2 trial in patients with PAH. For more information, please visit
www.clinicaltrials.gov
Sotatercept is an investigational therapy that is not approved
for any use in any country.
About PAH
PAH is a rare and chronic, rapidly progressing disorder
characterized by the constriction of small pulmonary arteries and
elevated blood pressure in the pulmonary circulation. PAH results
in significant strain on the heart, often leading to limited
physical activity, heart failure, and reduced life expectancy. The
5-year survival rate for patients with PAH is approximately 57%.
Available therapies generally act by promoting the dilation of
pulmonary vessels without addressing the underlying cause of the
disease. As a result, PAH often progresses rapidly for many
patients despite standard of care treatment. A growing body of
research has implicated imbalances in BMP and TGF-beta signaling as
a primary driver of PAH in familial, idiopathic, and acquired forms
of the disease.
About Acceleron
Acceleron is a biopharmaceutical company dedicated to the
discovery, development, and commercialization of therapeutics to
treat serious and rare diseases. Acceleron’s leadership in the
understanding of TGF-beta superfamily biology and protein
engineering generates innovative compounds that engage the body's
ability to regulate cellular growth and repair.
Acceleron focuses its research and development efforts in
hematologic and pulmonary diseases. In hematology, Acceleron and
its global collaboration partner, Bristol Myers Squibb, are
co-promoting newly approved REBLOZYL® (luspatercept-aamt), the
first and only approved erythroid maturation agent, in the United
States and are developing luspatercept for the treatment of chronic
anemia in myelofibrosis. Acceleron is developing sotatercept for
the treatment of pulmonary arterial hypertension, having recently
reported positive topline results of the Phase 2 PULSAR trial and
actively enrolling patients in the Phase 2 SPECTRA trial.
For more information, please visit www.acceleronpharma.com.
Follow Acceleron on social media: @AcceleronPharma and
LinkedIn.
Forward-Looking Statements
This press release contains forward-looking statements about
Acceleron’s strategy, future plans and prospects, including
statements regarding the development of sotatercept in PAH, the
timeline for clinical development and regulatory approval of
sotatercept in PAH, the expected timing for reporting of data from
ongoing clinical trials, and the potential of Acceleron’s compounds
as therapeutic drugs. The words "anticipate," "believe," "could,"
"estimate," "expect," "goal," "intend," "may," "plan," “possible,”
"potential," "project," "should," "target," "will," "would," and
similar expressions are intended to identify forward-looking
statements, although not all forward-looking statements contain
these identifying words.
Actual results could differ materially from those included in
the forward-looking statements due to various factors, risks and
uncertainties, including, but not limited to, that preclinical
testing of Acceleron’s compounds and data from clinical trials may
not be predictive of the results or success of ongoing or later
clinical trials, that regulatory approval of Acceleron’s compounds
in one indication or country may not be predictive of approval in
another indication or country, that the development of Acceleron’s
compounds will take longer and/or cost more than planned, that
Acceleron will be unable to successfully complete the clinical
development of Acceleron’s compounds, that Acceleron may be delayed
in initiating, enrolling or completing any clinical trials, that
Acceleron’s compounds will not receive regulatory approval or
become commercially successful products, and that Breakthrough
Therapy designation may not expedite the development or review of
sotatercept. These and other risks and uncertainties are identified
under the heading “Risk Factors” included in Acceleron’s most
recent Annual Report on Form 10-K, and other filings that Acceleron
has made and may make with the SEC in the future.
The forward-looking statements contained in this press release
are based on management's current views, plans, estimates,
assumptions, and projections with respect to future events, and
Acceleron does not undertake and specifically disclaims any
obligation to update any forward-looking statements.
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version on businesswire.com: https://www.businesswire.com/news/home/20200408005276/en/
Acceleron Pharma Inc. Investors: Todd James, 617-649-9393 Senior
Vice President, Corporate Affairs and Investor Relations Media:
Matt Fearer, 617-301-9557 Director, Corporate Communications
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