FDA approves firdapse for rare autoimmune disorder

FDA approves firdapse for rare autoimmune disorder
(HealthDay)—Firdapse (amifampridine) tablets have been approved by the U.S. Food and Drug Administration for adults with Lambert-Eaton myasthenic syndrome (LEMS).

The drug is the first agency-sanctioned treatment for the autoimmune disease that affects the connections between nerves and muscles. LEMS affects about three people per one million worldwide, the FDA said. The disorder most commonly occurs among people with cancer, such as small cell lung cancer.

The drug's safety and effectiveness were evaluated in involving 64 adults. Using a 13-item physician-rated categorical scale assessing and a seven-point scale on which patients rated their overall impression of the effects of the study treatment on their physical well-being, researchers found that those treated with Firdapse benefited more than those who took a placebo, the agency said. The drug's most common side effects included paresthesia, upper respiratory tract infection, abdominal pain, nausea, diarrhea, headache, and elevated liver enzymes. Patients who have a hypersensitivity reaction such as rash, hives, itching, fever, swelling, or trouble breathing should immediately contact their health care provider.

"Patients with LEMS have significant weakness and fatigue that can often cause great difficulties with ," Billy Dunn, M.D., director of the FDA's Division of Neurology Products, said in an agency news release. "There has been a long-standing need for a treatment for this rare disorder."

Firdapse is produced by Catalyst Pharmaceuticals, based in Coral Gables, Florida.


Explore further

FDA approves drug for treatment of travelers' diarrhea

More information: More Information

Copyright © 2018 HealthDay. All rights reserved.

Citation: FDA approves firdapse for rare autoimmune disorder (2018, November 30) retrieved 16 June 2019 from https://medicalxpress.com/news/2018-11-fda-firdapse-rare-autoimmune-disorder.html
This document is subject to copyright. Apart from any fair dealing for the purpose of private study or research, no part may be reproduced without the written permission. The content is provided for information purposes only.
 shares

Feedback to editors

User comments

Please sign in to add a comment. Registration is free, and takes less than a minute. Read more