CORAL GABLES, Fla., Feb. 28, 2018 (GLOBE NEWSWIRE) — 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 company’s support of the 11th annual Rare Disease Day on February 28, 2018. Rare Disease Day emphasizes the importance of rare disease research toward the development of diagnostic tools, treatments and cures, as well as improved health and social care for patients and their families.
Catalyst continues to support patient organizations that provide disease awareness for Lambert-Eaton Myasthenic Syndrome, Congenital Myasthenic Syndromes and anti-body positive MuSK myasthenia gravis. These are rare diseases in which patients are often misdiagnosed with other diseases prior to receiving a definitive diagnosis. Therefore, educating the patient and physicians at medical congresses is an important outreach to the rare disease community to facilitate proper and prompt diagnoses.
“Catalyst is proud to support Rare Disease Day and once again be part of this global movement to raise awareness for rare diseases,” said Patrick J. McEnany, Chairman and CEO of Catalyst. “With the upcoming resubmission of our NDA for Firdapse®, we are one step closer to hopefully being able to provide a treatment for those afflicted by the rare disease Lambert-Eaton Myasthenic Syndrome.”
Rare Disease Day takes place every year on the last day of February (February 28 or February 29 in a leap year) to underscore the nature of rare diseases and the challenges such patients face on a day-to-day basis. 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 more patients with rare diseases to take an active role in raising awareness about those diseases.
For more information about Rare Disease Day in the U.S., go to 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/wrdd2018/.
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).
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.
Source: Catalyst Pharmaceuticals, Inc.
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