Catalyst Pharmaceuticals Supports Rare Disease Day® and Joins Global Movement to Raise Important Awareness for Rare Diseases...
February 27 2017 - 8:03AM
Catalyst Pharmaceuticals, Inc. (Nasdaq:CPRX), (Catalyst) a
biopharmaceutical company focused on developing
and commercializing innovative therapies
for people with rare debilitating diseases,
today announced that it has joined
forces with 30 million Americans
and health care advocates around the world for
Rare Disease Day® on February 28. Rare Disease Day is an annual
awareness day dedicated to elevating public understanding of rare
diseases and calling attention to the special challenges they
present to patients and families.
Catalyst continues to support patient
organizations that provide disease awareness for Lambert-Eaton
Myasthenic Syndrome (LEMS) and Congenital Myasthenic Syndromes
(CMS). These are rare diseases in which patients are often
misdiagnosed with other diseases prior to getting a definitive
diagnosis, thus educating the patient and physicians at medical
congresses is an important outreach to the rare disease community
to facilitate proper and prompt diagnoses.
"Completing one phase 3 clinical study and
enrolling a second phase 3 clinical study for LEMS indicates our
commitment to the LEMS community for patients to have access to an
FDA approved treatment" says Patrick J. McEnany, Chairman and CEO
of Catalyst. "In addition, we have expanded our CMS phase 3
clinical study to include adults in recognition of the need to
provide access to a potential treatment across the entire CMS
population. We are pleased to continue to support the efforts of
the rare disease community to find ways to broaden the awareness of
rare diseases and improve access to treatments.”
According to the National Institutes of Health
(NIH), a disease is rare if it affects fewer than 200,000 people.
Nearly 1 in 10 Americans live with a rare disease—affecting 30
million people—and nearly half of these patients are children.
There are more than 7,000 rare diseases and only approximately 450
FDA-approved medical treatments.
Rare Disease Day takes place every year on the
last day of February (February 28 or February 29 in a leap
year)—the rarest date on the calendar—to underscore the nature of
rare diseases and what patients face. It was established in Europe
in 2008 by EURORDIS, the organization representing rare disease
patients in Europe, and is now observed in more than 80 nations.
Rare Disease Day is sponsored in the U.S. by the National
Organization for Rare Disorders (NORD)®, the largest and leading
independent, nonprofit organization committed to the
identification, treatment, and cure of rare diseases and one which
Catalyst actively supports. Additionally, Catalyst supports
Global Genes in their efforts to continue to get patients with rare
diseases to take an active role in raising awareness about their
disease.
For more information about Rare Disease Day in
the U.S., go to http://www.rarediseaseday.us. For information
about global activities, go to www.rarediseaseday.org. To search
for information about rare diseases, visit NORD’s website
www.rarediseases.org and Global Gene’s website
https://globalgenes.org/world-rare-disease-day.
About Catalyst
Pharmaceuticals
Catalyst Pharmaceuticals is a biopharmaceutical
company focused on developing and commercializing innovative
therapies for people with rare debilitating diseases, including
Lambert-Eaton myasthenic syndrome (LEMS), congenital myasthenic
syndromes (CMS), infantile spasms, and Tourette's Disorder.
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
infantile spasms, epilepsy and other neurological conditions
associated with reduced GABAergic signaling, like post-traumatic
stress disorder and Tourette's Disorder. 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 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, the timing on Catalyst's
second trial evaluating Firdapse for the treatment of LEMS and
whether the trial will be successful, whether Catalyst's
assumptions in its updated business plan will be accurate and the
impact of unanticipated events or delays in projected activities on
Catalyst's cash requirements and on Catalyst's ability to get to an
accepted NDA submission for Firdapse without the need for
additional funding, what clinical trials and studies will be
required before Catalyst can resubmit an NDA for Firdapse for the
treatment of CMS and whether any such required clinical trials and
studies will be successful, whether the investigator-sponsored
study evaluating Firdapse for the treatment of MuSK-MG will be
successful, whether any NDA for Firdapse resubmitted to the FDA
will ever be accepted for filing, the timing of any such NDA
filing or acceptance, whether, if an NDA for Firdapse is accepted
for filing, such NDA will be given a priority review by the FDA,
whether Firdapse will ever be approved for commercialization,
whether Catalyst will be the first company to receive approval for
amifampridine (3,4-DAP), giving it 7-year marketing exclusivity for
its product, whether CPP-115 will be determined to be safe for
humans, what additional testing will be required before CPP-115 is
"Phase 2 ready", whether CPP-115 will be determined to be effective
for the treatment of infantile spasms, post-traumatic stress
disorder, Tourette's Disorder or any other indications, whether
Catalyst can successfully design and complete a bioequivalence
study of its version of vigabatrin compared to Sabril that is
acceptable to the FDA, whether any such bioequivalence study the
design of which is acceptable to the FDA will be successful,
whether any ANDA that Catalyst submits for a generic version of
Sabril will be accepted for filing, whether any ANDA for Sabril
accepted for filing by the FDA will be approved (and the timing of
any such approval), whether any of Catalyst's product candidates
will ever be approved for commercialization or successfully
commercialized, and those other factors described in Catalyst's
Annual Report on Form 10-K for the fiscal year 2015 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
The Trout Group LLC
(646) 378-2923
bkorb@troutgroup.com
Company Contact
Patrick J. McEnany
Catalyst Pharmaceuticals
Chief Executive Officer
(305) 420-3200
pmcenany@catalystpharma.com
Media Contacts
David Schull
Matt Middleman, M.D.
Russo Partners
(212) 845-4271
(212) 845-4272
david.schull@russopartnersllc.com
matt.middleman@russopartnersllc.com
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