Catalyst Pharmaceuticals Announces Its Support of Spinal Muscular Atrophy Awareness Month
August 01 2019 - 8:03AM
Catalyst Pharmaceuticals, Inc. (Catalyst) (Nasdaq: CPRX), a
biopharmaceutical company focused on developing and commercializing
innovative therapies for people with rare debilitating, chronic
neuromuscular and neurological diseases, today announced its
support of Spinal Muscular Atrophy (SMA) Awareness Month. Taking
place in August, SMA Awareness Month involves activities worldwide
to spotlight the work the SMA community does year-round to raise
awareness of the debilitating and potentially deadly disease.
“We are proud to be part of the global movement
to raise awareness of SMA and recognize the importance of
developing new evidence-based medicines required to improve the
lives of patients, families and caregivers afflicted with SMA,”
said Patrick J. McEnany, Chairman and CEO of Catalyst.
“We are pleased to be leading the Catalyst
sponsored pilot study of Firdapse® as a possible treatment option
for SMA Type 3,” stated Dr. Lorenzo Maggi at the Carlo Besta
Neurological Institute in Milan, Italy. “Defects in the
neuromuscular junction appear to be concomitant to degeneration of
motor neurons. The literature suggests that amifampridine may have
clinical utility in these patients through its action on the
impaired neuromuscular junction transmission.”
Out of every 10,000 births, one baby is born
with SMA. SMA is a genetic disease that affects the part of the
nervous system that controls voluntary muscle movement. It is
caused by malfunctioning genes that affect how nerve cells and
muscle cells communicate across the neuromuscular junction—the
place where nerve cells meet muscle cells. Patients with SMA can
have severe muscle weakness in the upper arms, legs, and shoulders.
In some instances, SMA may lead to paralysis or death.
About Catalyst Pharmaceuticals
Catalyst Pharmaceuticals is a biopharmaceutical
company focused on developing and commercializing innovative
therapies for people with rare debilitating, chronic neuromuscular
and neurological diseases, including Lambert-Eaton myasthenic
syndrome (LEMS), anti-MuSK antibody positive myasthenia gravis
(MuSK-MG), congenital myasthenic syndromes (CMS), and spinal
muscular atrophy (SMA) Type 3. Catalyst's new drug application for
Firdapse® (amifampridine) 10 mg tablets for the treatment of adults
with LEMS was approved in November 2018 by the U.S. Food &
Drug Administration ("FDA"), and Firdapse is now commercially
available in the United States. Prior to its approval,
Firdapse for LEMS had received breakthrough therapy designation and
orphan drug designation from the FDA.
Firdapse is being evaluated in clinical trials
for the treatment of MuSK-MG, CMS, and SMA Type 3 and has received
Orphan Drug Designation from the FDA for myasthenia
gravis and CMS. Firdapse (amifampridine) 10 mg tablets is the first
and only approved drug in Europe for the symptomatic
treatment in adults with LEMS.
Forward-Looking Statements
This press release contains forward-looking
statements. Forward-looking statements involve known and unknown
risks and uncertainties, which may cause Catalyst's actual results
in future periods to differ materially from forecasted results. A
number of factors, including (i) whether Catalyst’s
proof-of-concept study evaluating Firdapse for the treatment of SMA
Type 3 will be successful, and (ii) those factors described in
Catalyst's Annual Report on Form 10-K for the fiscal year 2018 and
its other filings with the U.S. Securities and Exchange Commission
(SEC), could adversely affect Catalyst. Copies of Catalyst's
filings with the SEC are available from the SEC, may be found on
Catalyst's website, or may be obtained upon request from Catalyst.
Catalyst does not undertake any obligation to update the
information contained herein, which speaks only as of this
date.
Investor Contact
Brian Korb
Solebury Trout
(646) 378-2923
bkorb@troutgroup.com
Company Contact
Patrick J. McEnany
Catalyst Pharmaceuticals
Chief Executive Officer
(305) 420-3200
pmcenany@catalystpharma.com
Media Contact
David Schull
Russo Partners
(212) 845-4271
david.schull@russopartnersllc.com
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