FDA Approves Firdapse

Nov 28, 2018 -- Typically the U. S. Food and Drug Administration today approved Firdapse (amifampridine) tablets for the treatment of Lambert-Eaton myasthenic syndrome (LEMS) in adults. LEMS is a rare autoimmune disorder that impacts the connection between nerves and muscles to result in weakness and other symptoms in afflicted patients. This can be the first FDA approval of a treatment for LEMS.

“There has been a long-standing need for a treatment for this rare disorder, ” said Billy Dunn, Mirielle. D., director of the Division of Neurology Items in the FDA’s Centre for Drug Evaluation and Research. “Patients with LEMS have substantial weakness and fatigue that can often cause great difficulties with daily activites. ”

Inside people with LEMS, the body’s own immune system attacks the neuromuscular passageway (the connection between nerve fibres and muscles) and interferes with the ability of nerve tissue to send signals to muscle cells. LEMS may be associated with other autoimmune diseases, but more commonly occurs in patients with cancer such as small cell lung cancer, where their onset precedes or coincides with the diagnosis of cancer. The prevalence of LEMS is estimated to be three per , 000, 000 individuals worldwide.

The efficacy of Firdapse was analyzed in two clinical trials that together included 64 adult patients who received Firdapse or placebo. Typically the studies measured the Quantitative Myasthenia Gravis score (a 13-item physician-rated categorical size assessing muscle weakness) and the Subject Global Effect (a seven-point scale on which patients rated their overall impression of the consequences of the study treatment on their physical well-being). Regarding both measures, the patients obtaining Firdapse experienced a greater benefit than those on placebo.

The most common side effects experienced by patients in the clinical trials were burning or prickling sensation (paresthesia), higher respiratory tract infection, stomach pain, nausea, diarrhea, head ache, elevated liver enzymes, back pain treatments, hypertension and muscle jerks. Seizures have been seen in patients without a history of seizures. Sufferers should inform their physician immediately if they have signs of hypersensitivity responses such as rash, hives, itching, fever, swelling or trouble breathing.

The FDA granted this application Top priority Review and Breakthrough Remedy designations. Firdapse also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for uncommon diseases.