Last modified
12/04/2018 - 08:22

FDA approves first treatment for Lambert-Eaton myasthenic syndrome, a rare autoimmune disorder

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 affects the connection between nerves and muscles and causes weakness and other symptoms in afflicted patients. This can be the first FDA approval of any 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 Goods 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 activities. ”

That individuals with LEMS, the body’s own immune system attacks the neuromuscular junction (the connection between nerves and muscles) and disrupts 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 its onset precedes or coincides with the diagnosis of cancer. The prevalence of LEMS is approximated to be three each million individuals worldwide.

The efficacy of Firdapse was studied in two scientific trials that together included 64 adult patients who received Firdapse or placebo. The studies measured the Quantitative Myasthenia Gravis report (a 13-item physician-rated particular scale assessing muscle weakness) and the Subject Global Impression (a seven-point size on which patients rated their overall impression of the effects of the analysis treatment on their physical well-being). For both measures, the patients acquiring 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), upper respiratory system infection, stomach pain, nausea, diarrhea, headache, elevated lean meats enzymes, back pain, hypertonie and muscle spasms. Seizures have been observed in patients with out a history of seizures. Patients should notify their health care provider immediately if they have signs of hypersensitivity responses such as rash, urticaria, itching, fever, swelling or trouble breathing.

The FOOD AND DRUG ADMINISTRATION (FDA) granted this application Top priority Review and Breakthrough Therapy designations. Firdapse also received Orphan Drug designation, which gives incentives to assist and encourage the development of drugs for rare diseases.

The FDA granted the approval of Firdapse to Catalyst Pharmaceuticals, Inc.

The particular FDA, an agency within the U. S. Section of Health and Human being Services, protects the general public health by assuring the safety, effectiveness, and security of human and veterinarian drugs, vaccines and other biological products for individual use, and medical devices. The agency also is in charge of the safety and security of our nation’s food supply, cosmetics, vitamin supplements, products that give off electronic radiation, and for managing tobacco products.

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