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Catalyst Pharmaceuticals Announces Support of Rare Disease Day 2019

1216 Days ago

CORAL GABLES, Fla., Feb. 28, 2019 (GLOBE NEWSWIRE) -- Catalyst Pharmaceuticals, Inc. (Catalyst) (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 support of Rare Disease Day 2019. Taking place today, Rare Disease Day involves activities worldwide to raise awareness of rare diseases and their impact on patients’ lives among the general public and decision-makers in industry and government.

“We are proud to support Rare Disease Day and recognize the importance of investment in medical research that is needed to improve the lives of patients who are afflicted with rare and ultra-rare diseases,” said Patrick J. McEnany, Chairman and CEO of Catalyst. “We want to put the patient first in everything we do and continue to collaborate with advocacy organizations focused on Lambert-Eaton Myasthenic Syndrome (LEMS) and other diseases, for the development of our research and educational programs. Furthermore, we believe that the pharmaceutical industry has an obligation to bring to market new evidence-based medicines that can benefit all patients with a specific disease. This is critical to enabling us to make a difference, and we applaud the work of fellow industry members who work closely with the patient organizations and put patients’ needs at the forefront.”

“To be able to dance with my daughter at her wedding was life-changing for me. With the recent approval of Firdapse I’m energized by the increased awareness, education and support that is now available for the entire LEMS community”, said Bill Ronayne, a LEMS patient. “On Rare Disease Day, it is important to recognize and thank all the people and organizations, like Catalyst, who are dedicated to the rare disease community and work to give patients like me hope for the future.”

For information about Rare Disease Day activities, visit https://rarediseases.org/rare-disease-day/.

About Lambert-Eaton Myasthenic Syndrome (LEMS)

LEMS, is a rare autoimmune disorder, most often characterized by fatigable limb muscle weakness. The disease is caused by autoantibodies against voltage-gated calcium channels located in the nerve-muscle junction, resulting in improper nerve-muscle communication, leading to progressive muscle weakness, when left untreated.  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.  In early course of the disease, muscles closest to the trunk are affected, which are manifested as difficulties with climbing stairs or rising from a sitting position.  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, with symptoms of dry mouth, dry skin, constipation, blurred vision, impaired sweating, and/or inappropriate blood pressure variations.

About Catalyst Pharmaceuticals

Catalyst Pharmaceutical 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 (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 recently approved 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 CMS, MuSK-MG and SMA Type 3 and has received Orphan Drug Designation from the FDA for CMS and myasthenia gravis. Firdapse (amifampridine) 10 mg tablets is the first and only approved drug in Europe for the symptomatic treatment in adults with LEMS.

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 will be successful in commercializing Firdapse, (ii) whether, even if Catalyst is successful in commercializing Firdapse, Catalyst will become profitable, (iii) whether Firdapse will ever be approved for the treatment of CMS, MuSK-MG, SMA Type 3, or any other disease, and (iv) 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

Company Contact
Patrick J. McEnany
Catalyst Pharmaceuticals
Chief Executive Officer
(305) 420-3200

Media Contact
David Schull
Russo Partners
(212) 845-4271

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