Acceleron Pharma Inc. (NASDAQ:XLRN), a leading biopharmaceutical
company in the discovery and development of TGF-beta superfamily
therapeutics to treat serious and rare diseases, today announced
that the United States Food and Drug Administration (FDA) has
granted Orphan Drug designation to ACE-083, the Company’s
locally-acting “Myostatin+” muscle agent, for the treatment of
patients with Charcot-Marie-Tooth disease (CMT).
“This formal designation aligns with our mission to develop
therapies for patients with rare diseases of high unmet medical
need,” said Robert K. Zeldin, M.D., Chief Medical Officer of
Acceleron. “In clinical trials to date, treatment with ACE-083 has
resulted in substantial increases in muscle volume in target
muscles. We believe that if our ongoing studies demonstrate that
such increases lead to improved functional outcomes, ACE-083 has
the potential to become an important new therapy for patients with
CMT.”
Orphan designation is granted by the FDA Office of Orphan
Products Development to advance the evaluation and development
of safe and effective therapies for the treatment of rare diseases
or conditions affecting fewer than 200,000 people in the U.S. Under
the Orphan Drug Act, the FDA may provide grant funding
toward clinical trial costs, tax advantages, FDA user-fee
benefits, and seven years of market exclusivity in the United
States following marketing approval by the FDA. The
granting of an orphan designation request does not alter the
standard regulatory requirements and process for obtaining
marketing approval. For more information about orphan designation,
please visit the FDA website at www.fda.gov.
ACE-083 is currently being evaluated in two Phase 2 trials: one
in CMT and one in facioscapulohumeral muscular dystrophy (FSHD).
Preliminary results from Part 2 of the trials are expected by year
end 2019 for CMT and in the second half of 2019 for FSHD. During
2018, the FDA granted Fast Track designation for ACE-083 in CMT as
well as Fast Track and Orphan Drug designations for ACE-083 for the
treatment of patients with FSHD.
About ACE-083
ACE-083, a locally-acting therapeutic candidate based on the
naturally-occurring protein follistatin, utilizes the Myostatin+
approach to inhibit multiple TGF-beta superfamily ligands. It is
designed to have a concentrated effect along targeted muscles to
maximize growth and strength selectively in the muscles into which
the drug is administered. Acceleron is developing ACE-083 for
disorders such as CMT and FSHD, in which improved muscle strength
in target muscles may provide a clinical benefit and enhance
quality of life.
ACE-083 is an investigational therapy that is not approved for
any use in any country.
About CMT
CMT is one of the most common inherited neurologic diseases,
estimated to affect more than 125,000 people in the United
States. The primary clinical manifestations of CMT include muscle
weakness in the lower legs and hands. The lower leg muscle weakness
can result in foot drop leading to frequent trips and falls. The
disease is typically diagnosed by a characteristic pattern of
muscle weakness and sensory deficits, family history, nerve
conduction studies, and genetic testing. There are
no regulatory authority-approved therapies for CMT.
About Acceleron
Acceleron is a clinical-stage biopharmaceutical company
dedicated to the discovery, development, and commercialization of
therapeutics to treat serious and rare diseases. The Company's
leadership in the understanding of TGF-beta 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, neuromuscular, and pulmonary diseases. In hematology,
the Company and its global collaboration partner, Celgene, are
developing luspatercept for the treatment of chronic anemia in
myelodysplastic syndromes, beta-thalassemia, and myelofibrosis.
Acceleron is also advancing its neuromuscular franchise with two
distinct Myostatin+ agents, ACE-083 and ACE-2494, and a Phase 2
pulmonary program with sotatercept in pulmonary arterial
hypertension.
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 the
Company's strategy, future plans and prospects, including
statements regarding the development of the Company's compounds,
the timeline for clinical development and regulatory approval of
the Company’s compounds and the expected timing for reporting of
data from ongoing clinical trials. The words "anticipate,"
"believe," "could," "estimate," "expect," "goal," "intend," "may,"
"plan," "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 risks and
uncertainties, including, but not limited to, that preclinical
testing of the Company's compounds and data from clinical trials
may not be predictive of the results or success of ongoing or later
clinical trials, that the development of the Company's compounds
will take longer and/or cost more than planned, that the Company
will be unable to successfully complete the clinical development of
the Company’s compounds, that the Company may be delayed in
initiating, enrolling or completing any clinical trials, and that
the Company's compounds will not receive regulatory approval or
become commercially successful products. These and other risks and
uncertainties are identified under the heading "Risk Factors"
included in the Company's most recent Annual Report on Form 10-K,
and other filings that the Company 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 the
Company 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/20190305005184/en/
Acceleron Pharma Inc.Investors:Todd James, IRC, 617-649-9393Vice
President, Investor Relations and Corporate Communications
Media:Matt Fearer, 617-301-9557Director, Corporate
Communications
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