Catalyst Pharmaceuticals Announces Submission of New Drug Application for Firdapse® for Treatment of Lambert-Eaton Myastheni...
March 29 2018 - 7:19AM
Catalyst Pharmaceuticals, Inc. (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 submission of a New
Drug Application (NDA) to the U.S. Food and Drug Administration
(FDA) for Firdapse® (amifampridine) for the symptomatic treatment
of Lambert-Eaton myasthenic syndrome (LEMS).
Last month, Catalyst announced the results of its recent
positive Type C meeting with the FDA and reported that the minutes
of the meeting received from the Agency reflect the FDA’s advice to
Catalyst that its proposed filing package would be sufficient for
submission of an NDA for Firdapse. Last year, Catalyst reported
positive top-line results from its second Phase 3 clinical trial of
Firdapse for the treatment of LEMS, which was conducted under a
protocol agreed to by the FDA through the Special Protocol
Assessment process.
“We are pleased to reach this regulatory milestone and believe
that our NDA submission contains all of the necessary information
to satisfy the FDA requirements,” said Patrick J. McEnany, Chairman
and Chief Executive Officer of Catalyst Pharmaceuticals, Inc. “This
important milestone is the culmination of a strong collaboration
and commitment among the patients, physicians and Catalyst
employees who have worked diligently to advance Firdapse and to
further expand access to an FDA-approved product to all LEMS
patients. We look forward to continuing to work with the FDA during
the review process and to a potential future launch of Firdapse, if
it is approved.”
Catalyst also reported on its decision not to include in this
FDA submission those limited types of congenital myasthenic
syndromes (CMS) that are considered mechanistically similar to
LEMS. Catalyst reported that while it considered adding these
indications to this NDA submission, after discussion with the FDA
at its Type C meeting and further consideration, it decided not to
overcomplicate the review of its NDA submission for LEMS with this
second indication. Catalyst is currently conducting a Phase 3 trial
evaluating Firdapse for the treatment of CMS, and expects to
complete enrollment in this trial before the end of the year (and
to report top-line results for its trial in the first quarter of
2019). Assuming the trial is successful, Catalyst plans to seek to
add CMS to its label for Firdapse for a much broader population of
CMS patients.
The NDA submission announced today addressed the two issues
raised in the 2016 Refusal to File letter for the previous Firdapse
NDA submission and included all additional information requested by
the FDA. Once an NDA is submitted, the FDA has a 60-day filing
review period to determine whether the NDA is complete and
acceptable for filing, after which time the Agency will notify the
company submitting the NDA of its decision. Catalyst plans to
communicate the Agency’s decision once Catalyst has been
notified.
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 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),
congenital myasthenic syndromes (CMS), MuSK antibody positive
myasthenia gravis, spinal muscular atrophy (SMA) type 3 and
infantile spasms. Firdapse® has received Breakthrough Therapy
Designation from the U.S. Food and Drug Administration (FDA) for
the treatment of LEMS and Orphan Drug Designation for LEMS, CMS and
myasthenia gravis. Firdapse is the first and only approved drug in
Europe for the symptomatic treatment in adults with LEMS.
Catalyst is also developing CPP-115 to treat refractory
infantile spasms. CPP-115 has been granted U.S. Orphan Drug
Designation for the treatment of infantile spasms by the FDA and
has been granted E.U. Orphan Medicinal Product Designation for the
treatment of West syndrome by the European Commission. In
addition, Catalyst is developing a generic version of Sabril®
(vigabatrin).
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 the results of the LMS-003 trial,
combined with the results of the Company's previous Phase 3 trial,
will be acceptable to the FDA as support for an approval of
Firdapse for the treatment of LEMS, (ii) whether the results of the
abuse liability studies undertaken by Catalyst will be acceptable
to the FDA as support for an approval of Firdapse, (iii) whether
the NDA submitted for Firdapse will be accepted by the FDA, and the
timing of any such acceptance, (iv) whether the receipt of
breakthrough therapy designation for Firdapse will expedite the
development and review of Firdapse by the FDA or the likelihood
that the product will be found to be safe and effective, (v)
whether, if an NDA for Firdapse is accepted for filing, such NDA
will be given a priority review by the FDA, (vi) whether Firdapse
will ever be approved for commercialization, (vii) whether Catalyst
will be the first company to receive an approval for amifampridine
(3,4-DAP), giving it 5-year marketing exclusivity for its product,
and (viii) those other factors described in Catalyst's Annual
Report on Form 10-K for the fiscal year 2017 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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