Acceleron Receives FDA Fast Track Designation for ACE-083 in Charcot-Marie-Tooth Disease
28 Noviembre 2018 - 6:00AM
Business Wire
Acceleron Pharma Inc. (NASDAQ:XLRN), a leading biopharmaceutical
company in the discovery and development of TGF-beta therapeutics
to treat serious and rare diseases, today announced that the United
States Food and Drug Administration (FDA) has granted Fast Track
designation to ACE-083, the Company’s locally-acting “Myostatin+”
muscle agent, for the treatment of patients with
Charcot-Marie-Tooth disease (CMT).
“We’re pleased that the FDA has granted this designation for
ACE-083,” said Robert K. Zeldin, M.D., Chief Medical Officer of
Acceleron. “Patients with CMT currently have no approved therapies.
To date, results from our Phase 2 trials have shown that patients
treated with ACE-083 experience robust increases in muscle volume.
If our ongoing clinical studies show that ACE-083 also improves
functional outcomes and confirm the favorable safety profile
observed thus far, the Fast Track process could help us work with
the FDA to deliver it to patients as quickly as possible.”
The FDA’s Fast Track designation is a process designed to
facilitate the development and expedite the review of drugs to
treat serious conditions and fill an unmet medical need. Once a
drug receives Fast Track designation, early and frequent
communication between the FDA and a drug company is encouraged
throughout the entire drug development and review process. The
frequency of communication assures that questions and issues are
resolved quickly, often leading to earlier drug approval and access
by patients.
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. Earlier
this year, the FDA granted 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.
About CMT
CMT is the most common inherited neurologic disease, 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 FDA-approved therapies for
CMT.
About Acceleron
Acceleron is a Cambridge-based, 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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Acceleron Pharma Inc.Todd James, IRC, 617-649-9393Vice
President, Investor Relations and Corporate CommunicationsorCandice
Ellis, 617-649-9226Manager, Investor Relations and Corporate
CommunicationsorMedia:Matt Fearer, 617-301-9557Director, Corporate
Communications
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