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Catalyst Pharmaceuticals Observes Rare Disease Day 2016

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2.0 from 8 votes
Monday, February 29, 2016

CORAL GABLES, Fla. - Catalyst Pharmaceuticals, Inc. (Nasdaq:CPRX), 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, today announced its support for Rare Disease Day®, always held today, the last day of February and dedicated to providing education and awareness about rare diseases. This year's theme is "Patient Voice-Join us in making the voice of rare diseases heard." Rare Disease Day is observed around the globe and is led in the U.S. by the National Organization for Rare Disorders (NORD) (http://rarediseases.org), a patient advocacy organization committed to the identification, treatment and cure of rare disorders through programs of education, advocacy and research. Catalyst collaborates with NORD as a corporate member. Global Genes (https://globalgenes.org/), one of the leading rare disease patient advocacy organizations in the world that promotes the needs of the rare disease community and of which Catalyst is also a corporate member, is another organization that supports patient activities to raise rare disease awareness on this special day.

"Providing education and awareness about rare diseases is a major focus for Catalyst," said Patrick J. McEnany, chairman and CEO of Catalyst. "We are pleased to support the efforts of the rare disease community to find ways to broaden the awareness of rare diseases and improve access to treatments."

In support of rare disease awareness, Catalyst has added a patient engagement section to its website (www.catalystpharma.com) that provides stories from patients with Lambert-Eaton myasthenic syndrome (LEMS) and their struggles with diagnosis and living with the disease.

According to the U.S. National Institutes of Health, 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 two-thirds of these patients are children. There are nearly 7,000 identified rare diseases, and with only approximately 450 U.S. Food and Drug Administration medical treatments approved for all of these diseases, many of these patients may receive no treatment for their disorders.

About Lambert-Eaton Myasthenic Syndrome (LEMS)
LEMS is a severely debilitating, rare autoimmune disease that affects approximately 1 in 100,000 people. The most common clinical presentations are proximal muscle weakness, easy fatigability that may lead to difficulty walking and climbing stairs, and cholinergic dysautonomia.

About Congenital Myasthenic Syndromes (CMS)
CMS is a rare autoimmune inherited neuromuscular disorder that is characterized by fatigable weakness of the skeletal muscles with onset at or shortly after birth or early childhood. In some cases, CMS symptoms may include sudden severe exacerbations of weakness or sudden episodes of respiratory insufficiency. Prevalence is estimated at 1-2 in 500,000 people.

About Tourette's Disorder
Tourette's Disorder is a hereditary neurological movement disorder that is characterized by repetitive motor and vocal tics. Symptoms may include involuntary movements of the extremities, shoulders, and face accompanied by uncontrollable sounds and, in some cases, inappropriate words.

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. Catalyst's lead candidate, Firdapse for the treatment of LEMS, has completed testing in a global, multi-center, double-blinded randomized pivotal Phase 3 trial resulting in positive top-line data. Firdapse for the treatment of LEMS has received Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) and Orphan Drug designations for LEMS and CMS. Firdapse is the first and only European approved drug 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).

Contact
Brian Korb
The Trout Group LLC
(646) 378-2923
bkorb@troutgroup.com

Company
Patrick J. McEnany
Catalyst Pharmaceuticals
Chief Executive Officer
(305) 529-2522
pmcenany@catalystpharma.com

Media
David Schull
Matt Middleman, M.D.
Russo Partners
(212) 845-4271
(212) 845-4272
david.schull@russopartnersllc.com
matt.middleman@russopartnersllc.com

Source: Catalyst Pharmaceuticals, Inc.
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2.0 from 8 votes
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