Lambert-Eaton Myasthenic Syndrome (LES)

Investigational Drug, Firdapse, Available at No Cost to U.S. Patients with LEMS Coral Gables drug company to make new medicine available at no cost April 23, 2014 Evan S. Benn Coral Gables-based Catalyst Pharmaceutical Partners soon will make its investigational drug Firdapse available at no cost to patients with a certain autoimmune disorder. The drug, which is supposed to relieve muscle weakness in people with Lambert-Eaton Myasthenic Syndrome, is entering the final phase of its clinical trials. Catalyst said it expects to submit an application for Firdapse’s approval with the U.S. Food and Drug Administration next year. Catalyst has been leading the development of Firdapse in the United States since 2012; the drug already is government-approved and commercially available in Europe. During the U.S. approval process, Catalyst said it will establish an expanded-access program to make Firdapse available for free to patients with LEMS. “As part of our ongoing commitment t

www.netag.nhs.uk/files/appraisal-reports/Firdapse - NETAG appraisal report - Nov 2011 -WEB VERSION.pdf

Firdapse ®  (currently approved in the EU) is the first and only approved drug for the symptomatic treatment of Lambert-Eaton Myasthenic Syndrome in adults, a rare autoimmune disease with the primary symptoms of muscle weakness. Firdapse at a Glance Approved in the European Union Manufactured and commercialized by BioMarin in the European Union Designated as an orphan drug in the United States, the European Union and and Switzerland Regulatory Status In December 2009, Firdapse received marketing approval from the European Commission making it the first approved therapy for the symptomatic treatment of LEMS. Firdapse has been designated an orphan drug in the EU, which grants market exclusivity for ten years. Commercialization Plan Firdapse is commercialized by BioMarin in the European Union. In October 2012, BioMarin out-licensed North American rights of Firdapse to Catalyst Pharmaceutical Partners. For more information on Firdapse development plans in the U.S., please r

Catalyst Pharmaceutical Partners Receives Breakthrough Therapy Designation From FDA for Firdapse(TM) for the Treatment of LEMS -  CORAL GABLES, Fla., Aug. 27, 2013 (GLOBE NEWSWIRE) -- Catalyst Pharmaceutical Partners, Inc. (Nasdaq: CPRX ), a specialty pharmaceutical company focused on the development and commercialization of novel prescription drugs targeting rare (orphan) neuromuscular and neurological diseases, today announced that its investigational product Firdapse™ (amifampridine phosphate) has received "Breakthrough Therapy Designation" by the U.S. Food and Drug Administration (FDA) for the symptomatic treatment of patients with Lambert-Eaton Myasthenic Syndrome (LEMS). Firdapse™ is Catalyst's investigational therapy that is being evaluated for the treatment of the debilitating symptoms associated with LEMS, including muscle weakness. "We are very pleased to have received Breakthrough Therapy Designation for Firdapse™ and we are excited by the FDA's d

Lambert–Eaton myasthenic syndrome From Wikipedia, the free encyclopedia   (Redirected from  Lambert-Eaton myasthenic syndrome ) Lambert–Eaton myasthenic syndrome Classification and external resources Neuromuscular junction. Lambert–Eaton myasthenic syndrome is caused by autoantibodies to the presynaptic membrane. Myasthenia gravis is caused by autoantibodies to the postsynaptic acetylcholine receptors. 1.  Presynaptic  terminal 2.  Sarcolemma 3.  Synaptic vesicle 4.  Nicotinic acetylcholine receptor 5.  Mitochondrion ICD - 10 G 73.1 ICD - 9 358.1 DiseasesDB 4030 MedlinePlus 000710 MeSH D015624 Lambert–Eaton myasthenic syndrome  ( LEMS , also  Lambert–Eaton syndrome , or  Eaton–Lambert syndrome ) is a rare  autoimmune  disorder that is characterized by  muscle weakness  of the limbs. It is the result of an  autoimmune reaction  in which  antibodies  are formed against presynaptic  voltage-gated calcium channels , and likely other nerve terminal proteins, in the 

Lambert Eaton Myasthenic Syndrome (LEMS): An Autoimmune Syndrome Causing Muscle Weakness Lambert Eaton Myasthenic Syndrome belongs to the class of disorders referred to as neuromuscular transmission disorders. That is to say, that this group of diseases affects the ability of the nerves to communicate with the muscles and thus leads to muscle weakness. Lambert Eaton Myasthenic Syndrome (LEMS) is also an autoimmune disorder because it is a product of the body that is acting to cause the disorder. In other words, the body is acting against itself to cause disease. Finally, LEMS is often a paraneoplastic disorder. The term paraneoplastic refers to a disease that is indirectly caused by a neoplasm or cancer. While we will discuss LEMS in detail, it is important to know that a variety of paraneoplastic syndromes exist and can be the first signs of a malignancy. PATHOPHYSIOLOGY In order to understand LEMS, it is necessary to first understand how muscles are made to work. We will ta

Catalyst Pharmaceutical Partners Announces Recommendation of Data Monitoring Committee to Continue Pivotal Phase lll Clinical Trial for Firdapse(TM) CORAL GABLES, Fla., Oct. 15, 2013 (GLOBE NEWSWIRE) -- Catalyst Pharmaceutical Partners, Inc. (Nasdaq:CPRX), a specialty pharmaceutical company focused on the development and commercialization of novel prescription drugs targeting rare (orphan) neuromuscular and neurological diseases and disorders, announced today that, at a recently held meeting, the independent Data Monitoring Committee (DMC) overseeing the Company's ongoing pivotal Phase III clinical trial in the United States and Europe evaluating Firdapse(TM) for the treatment of Lambert-Eaton Myasthenic Syndrome (LEMS) recommended that the Company continue the trial as planned based on the committee's review of safety and clinical data from the trial. The DMC is a group of experts responsible for the independent review of accumulated, unblinded clinical safety and effica