LEMS: Firdapse Study Shows Encouraging Results

Catalyst Pharmaceuticals launches Firdapse expanded access program for eligible people living with congenital myasthenic syndromes and Lambert-Eaton myasthenic syndrome

by Quest Staff on October 3, 2014 - 2:00pm

Catalyst Pharmaceuticals, a biopharmaceutical company focused on rare debilitating diseases, announced encouraging results on Sept. 29, 2014, from its phase 3 clinical trial of Firdapse (amifampridine phosphate tablets equivalent to 10mg amifampridine) for the symptomatic treatment of Lambert-Eaton myasthenic syndrome (LEMS).

Firdapse, a potassium channel inhibitor, is a drug that is intended to relieve muscle weakness caused by LEMS and other potential neuromuscular diseases, including certain types of myasthenia gravis (MG) and congenital myasthenic syndromes (CMS).

The drug was primarily assessed based on a physician-rated evaluation consisting of 13 assessments, such as arm and leg strength, face and neck muscle performance, swallowing, grip strength, forced respiration and gaze impairment, as well as patient satisfaction with the effects of the drug. Based on these primary endpoints, patients who received Firdapse showed better results than those who received a placebo. 

Firdapse is the first and only drug approved in Europe for symptomatic treatment in adults with LEMS, and Catalyst intends to submit a new drug application (NDA) to the U.S. Food and Drug Administration (FDA) to get it approved.

LEMS is caused by a misdirected attack of the immune system on nerve endings at the neuromuscular junction, the place where nerve and muscle fibers connect. The disorder causes muscle weakness and sometimes other symptoms, such as dry mouth or constipation.

In the United States, the disease is treated with medications that suppress the immune system and/or medications that slow breakdown of the neurotransmitter acetylcholine.

To learn more about the Firdapse trial, go to ClinicalTrials.gov and enter NCT02189720 in the search box.

New Firdapse expanded access program for people with LEMS and CMS

Catalyst Pharmaceuticals has launched an expanded access program (EAP) in the United States for eligible people living with congenital myasthenic syndromes (CMS) and Lambert-Eaton myasthenic syndrome (LEMS) for treatment with Firdapse. 

An expanded access program can be used to treat seriously ill patients using an investigational drug when no other treatments are available. FDA regulations enable manufacturers to provide these individuals access to the drug under certain situations, known as "expanded access" or "compassionate use," where people may benefit from treatment with the drug. Firdapse is available by prescription and should only be taken under the direction and care of your treating physician.

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