Catalyst Pharmaceuticals Announces Plans to Resubmit New Drug Application for Firdapse®
February 12 2018 - 8:03AM
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 the results of its
recent Type C meeting with the U.S. Food and Drug Administration
(FDA). Prior to the meeting, Catalyst had provided the FDA with its
preliminary data package for the proposed NDA resubmission,
including clinical, non-clinical, regulatory and abuse liability
elements. The preliminary data package included the recently
reported positive top-line results from a required second,
confirmatory Phase 3 clinical trial (LMS-003) of Firdapse® for the
symptomatic treatment of LEMS, as well as the recently completed
FDA-required abuse liability studies demonstrating that Firdapse
does not have abuse potential. The minutes of the meeting received
from the FDA reflect the FDA's advice to Catalyst that its proposed
filing package will be sufficient for resubmission of an NDA for
Firdapse, and Catalyst currently anticipates resubmitting its NDA
for Firdapse® for LEMS to the FDA by the end of the first quarter
of 2018.
As previously reported, Catalyst is presently conducting a Phase
3 double-blind placebo-controlled clinical trial evaluating
Firdapse® for the treatment of congenital myasthenic syndromes
(CMS), and Catalyst expects to report top line results from the
trial in the second half of this year. Catalyst is currently
evaluating its options for the most appropriate and efficient path
forward to hopefully include CMS in any approved labeling for
Firdapse.
“We remain on track to resubmit our NDA for Firdapse in the
first quarter of this year,” stated Patrick J. McEnany, President
and CEO of Catalyst Pharmaceuticals. “Based on our discussions with
the FDA, we believe that we have a clear regulatory pathway
forward, and we will continue to work collaboratively with the FDA
as we seek to bring FDA approved therapies forward for the
treatment of LEMS and CMS.”
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 Congenital Myasthenic Syndromes (CMS)
Congenital myasthenic syndromes, or CMS, are rare neuromuscular
disorders comprising a spectrum of genetic defects and is
characterized by fatigable weakness of skeletal muscles with usual
onset at or shortly after birth or early childhood; in rare cases
symptoms may not manifest themselves until later in childhood or
adulthood. The severity and course of the disease are variable,
ranging from minor symptoms to progressive disabling weakness;
symptoms may be mild, but sudden severe exacerbations of weakness
or even sudden episodes of respiratory insufficiency also occur.
Congenital myasthenic syndromes are rare, estimated at one-tenth
that of myasthenia gravis, which in itself is rare. Based on
currently available information, Catalyst estimates that there are
between 1,000 and 1,500 CMS patients in the United States.
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, 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 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
any NDA submitted for Firdapse will be accepted by the FDA, and the
timing of any such submission and 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 2016 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
ContactBrian KorbThe Trout Group LLC(646)
378-2923bkorb@troutgroup.com |
Company
ContactPatrick J. McEnanyCatalyst PharmaceuticalsChief Executive
Officer(305) 420-3200pmcenany@catalystpharma.com |
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Media
ContactDavid SchullRusso Partners(212)
845-4271david.schull@russopartnersllc.com |
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