Acceleron Receives FDA Fast Track Designation for ACE-083 in Facioscapulohumeral Muscular Dystrophy (FSHD)
01 Mayo 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 U.S.
Food and Drug Administration (FDA) has granted Fast Track
designation to ACE-083, a locally-acting “Myostatin+” muscle agent,
for the treatment of patients with facioscapulohumeral muscular
dystrophy (FSHD).
“This is an important milestone in the development of
ACE-083—our lead program within our neuromuscular franchise,” said
Matthew Sherman, Chief Medical Officer of Acceleron. “FSHD is a
serious and rare neuromuscular disorder for which there are
currently no approved therapies available. With this designation,
we will be able to expedite the FDA review process of ACE-083, and
if successful, deliver the first locally-acting, ‘Myostatin+’
muscle agent as a meaningful treatment option for the thousands of
patients impacted by FSHD.”
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. The
purpose is to get important new drugs to the patient earlier. Fast
Track addresses a broad range of serious conditions. 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 FSHD and one in Charcot-Marie-Tooth (CMT) disease.
About ACE-083
ACE-083 is a locally-acting therapeutic candidate, based on the
naturally-occurring protein follistatin, which utilizes the
"Myostatin+" approach to inhibit multiple TGF-beta 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
diseases such as facioscapulohumeral muscular dystrophy (FSHD) and
Charcot-Marie-Tooth (CMT) disease, in which improved muscle
strength in target muscles may provide a clinical benefit and
enhance quality of life.
About Facioscapulohumeral Muscular Dystrophy
(FSHD)
FSHD is a rare genetic muscle disorder affecting approximately
20,000 people in the United States for which there are
currently no approved treatments. The primary clinical presentation
of FSHD is debilitating skeletal muscle weakness and loss. The
symptoms of FSHD develop in a descending pattern, beginning with
the face and upper body and progressing to the lower body in a
"muscle by muscle" fashion. The disease is typically diagnosed by a
characteristic pattern of muscle weakness and other clinical
symptoms, as well as through genetic testing.
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 pulmonary
program with a Phase 2 trial of sotatercept planned in pulmonary
arterial hypertension.
For more information, please visit www.acceleronpharma.com.
Follow Acceleron on Social
Media: @AcceleronPharma and LinkedIn.
Cautionary Note on 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 or
its collaboration partner, Celgene, will be unable to successfully
complete the clinical development of the Company's compounds, that
the Company or Celgene 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.
Source: Acceleron Pharma
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version on businesswire.com: https://www.businesswire.com/news/home/20180501005351/en/
Acceleron Pharma Inc.Todd James, IRC, 617-649-9393Vice
President, Investor Relations and Corporate CommunicationsorCandice
Ellis, 617-649-9226Manager, Investor Relations and Corporate
CommunicationsorMedia:BMC CommunicationsBrad Miles,
646-513-3125
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