Catalyst Pharmaceuticals’ Firdapse® (amifampridine phosphate) Receives Marketing Approval in Canada for Patients with LEMS...
August 06 2020 - 8:03AM
Catalyst Pharmaceuticals, Inc. (Catalyst) (Nasdaq: CPRX), a
commercial-stage biopharmaceutical company focused on developing
and commercializing innovative therapies for people with rare
debilitating, chronic neuromuscular and neurological diseases,
today announced that Canada’s national healthcare regulatory
agency, Health Canada, has approved the use of Firdapse®
(amifampridine) for the treatment of patients in Canada with
Lambert-Eaton myasthenic syndrome (LEMS), an ultra-rare,
debilitating and potentially life-threatening neurodegenerative
condition.
Firdapse® was approved under Priority Review by
Health Canada. The marketing application submitted to Health Canada
included safety and efficacy data from Catalyst’s two previously
reported multi-national clinical trials evaluating Firdapse® for
the treatment of patients with LEMS, and the data from these trials
was used to support the new drug submission (NDS) to Health Canada.
Firdapse® was previously approved for marketing by the U. S. Food
and Drug Administration and the European Commission in 2018 and
2009, respectively, and is currently being used to treat LEMS
patients in the United States and in more than 15 additional
countries.
“At Catalyst, we are dedicated to changing the
lives of patients suffering from rare neuromuscular diseases, and
we are proud that we have brought forward an approved therapeutic
option for patients in Canada suffering with LEMS. We anticipate
continuing to interact with Health Canada as we try to expand the
Firdapse® label to include other indications,” said Gary Ingenito,
M.D., Ph.D., Catalyst’s Chief Medical and Regulatory Officer.
“We are currently in discussions with a
potential marketing and distribution partner in Canada to provide
access for Firdapse®, patient-by-patient, as rapidly as possible,”
said David Ailinger, Vice President of Business Development at
Catalyst.
About Lambert-Eaton Myasthenic Syndrome
(LEMS)
Lambert-Eaton myasthenic syndrome, or LEMS, is a
rare autoimmune disorder, most often characterized by muscle
weakness of the limbs. The disease is caused by an autoimmune
reaction where antibodies are formed against voltage gated
potassium channels in the connection between nerves and the muscles
they communicate with. In approximately 50% of cases, LEMS is
associated with an underlying malignancy, most commonly small-cell
lung cancer, and in some individuals, LEMS is the first symptom of
such malignancy. LEMS generally affects the extremities,
especially the legs. As the disease most affects the parts of
limbs closest to the trunk, difficulties with climbing stairs or
rising from a sitting position are commonly noted. Physical
exercise and high temperatures tend to worsen the symptoms.
Other symptoms occasionally seen include weakness of the
muscles of the mouth, throat, and eyes. Individuals affected
with LEMS also may have a disruption of the autonomic nervous
system, including dry mouth, constipation, blurred vision, impaired
sweating, and/or hypotension.
About Firdapse®
Firdapse® (amifampridine) 10 mg tablets is an
oral, nonspecific, voltage-dependent, potassium (K+) channel
blocker that causes depolarization of the presynaptic membrane and
slows or inhibits repolarization. This action results in the
opening of slow voltage-dependent calcium (Ca2+) channels, allowing
for a subsequent influx of Ca2+. In turn, it induces the exocytosis
of synaptic vesicles containing Acetylcholine (ACh) to release more
ACh into the synaptic cleft, enhancing neuromuscular transmission,
and providing for improved muscle function.
About Catalyst Pharmaceuticals
Catalyst Pharmaceuticals is a commercial-stage
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), 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
currently being evaluated in clinical trials for the treatment of
MuSK-MG and SMA Type 3 and has received Orphan Drug Designation
from the FDA for myasthenia gravis.
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 can successfully
commercialize its product in Canada on a profitable basis, (ii)
whether Catalyst will reach an agreement with a marketing and
distribution partner to commercialize its product in Canada, (iii)
whether Catalyst will have competition in Canada for its product,
(iv) the impact in the United States if an amifampridine product is
purchased in Canada for use in the United States, and (v) those
factors described in Catalyst's Annual Report on Form 10-K for the
fiscal year 2019 and Catalyst's 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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