CORAL GABLES, Fla., Feb. 28, 2014 (GLOBE NEWSWIRE) -- Catalyst Pharmaceutical Partners, Inc. (Nasdaq:CPRX), a specialty pharmaceutical company focused on developing safe and effective, approved medicines targeting orphan neuromuscular and neurological diseases, joins with the National Organization for Rare Disorders (NORD) in the observance of Rare Disease Day.
"Catalyst is focused on the development of innovative treatments for rare diseases," said Patrick McEnany, Chief Executive Officer, Catalyst Pharmaceutical Partners. "Recognizing Rare Disease Day is an opportunity to support the important work of advocacy groups for rare diseases such as NORD, which support patients with these rare afflictions. We have a pipeline of product candidates for the treatment of rare conditions, led by our phase 3 program for Firdapse™ (3,4-DAP) to treat Lambert Eaton Myasthenic Syndrome (LEMS). Firdapse™ may also be an effective treatment for other rare neuromuscular diseases, such as congenital myasthenic syndrome. We are also developing CPP-115 as a potential treatment of rare neurological conditions, such as Tourette Syndrome and infantile spasms."
In the United States, a rare disease is defined as one that affects fewer than 200,000 persons. According to the National Institutes of Health (NIH), there are nearly 7,000 rare diseases affecting nearly 30 million Americans. In the U.S., the coalition supporting Rare Disease Day includes patient organizations, NIH and other government entities, medical researchers, hospitals and academic institutions, and pharmaceutical companies developing treatments for rare diseases. Rare Disease Day was launched in Europe in 2008 by EURORDIS, the organization representing rare disease patients in Europe. It is now observed in more than 65 nations, and is sponsored in the U.S. by NORD. For more information about Rare Disease Day activities in the U.S., go to www.rarediseaseday.us.
About Catalyst Pharmaceutical Partners
Catalyst Pharmaceutical Partners, Inc. is a specialty pharmaceutical company focused on the development and commercialization of prescription drugs targeting rare (orphan) neuromuscular and neurological diseases, including Lambert-Eaton Myasthenic Syndrome (LEMS), infantile spasms, and Tourette Syndrome. Catalyst's lead candidate, Firdapse™ for the treatment of LEMS, is currently undergoing testing in a global, multi-center, pivotal phase 3 trial and has received Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA). Catalyst is also developing a potentially safer and more potent vigabatrin analog (designated CPP-115) to treat infantile spasms, and epilepsy, as well as other neurological conditions associated with reduced GABAergic signaling, like post-traumatic stress disorder and Tourette Syndrome. 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.
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 the timing of completion of Catalyst's currently ongoing Phase 3 trial of Firdapse™, whether the Phase 3 trial will be successful, whether the product will be found to be safe and effective, 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 2012 and Catalyst's 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.