RARE Daily

FDA Expands Approval of Catalyst’s Firdapse to Include LEMS Patients 6 and Older

September 30, 2022

The U.S. Food and Drug Administration granted expanded approval to Catalyst Pharmaceuticals’ Firdpase to include the treatment of Lambert-Eaton myasthenic syndrome patients 6 and older.

Firdapse is currently approved in the United States and Canada for the treatment of LEMS in adult patients.

LEMS is a rare autoimmune disease that affects about one in every 100,000 people worldwide. In LEMS, the body attacks nerve cells that control a chemical called acetylcholine (ACh). ACh is an important chemical because it helps send messages between the nerve cells and the muscle cells. In LEMS, the nerve cells lose the ability to release ACh and that effects how muscles work. LEMS causes muscles to feel weak because it affects the neuromuscular junction—the place where nerve cells meet muscle cells.

LEMS can occur at any age, but is commonly diagnosed in people who are over 40 years of age. Approximately 50 percent of people with LEMS also develop cancer—the most common form is a type of lung cancer called small cell lung cancer (SCLC), this is also known as paraneoplastic LEMS. Paraneoplastic LEMS typically occurs in people with a history of smoking and who are 50 years of age or older. Cancer is an important diagnostic criteria in people with LEMS, so cancer testing is recommended.

Firdapse is the only FDA-approved, evidence-based treatment for adults diagnosed with Lambert-Eaton myasthenic syndrome (LEMS). LEMS reduces the ability of nerves to release ACh, so the muscles don’t receive the signal they need to move and function. Firdapse improves the ability of nerves to communicate with muscles by helping to restore the release of ACh.

“While the U.S. LEMS pediatric population is an exceptionally small number of patients, this positive outcome helps ensure that all eligible LEMS patients have access to Firdapse for the treatment of this rare disease,” stated Patrick McEnany, chairman and CEO of Catalyst.

As part of Catalyst’s efforts to help ensure those patients who can benefit from Firdapse are able to obtain access, Catalyst offers a comprehensive patient access support program and patient assistance for qualifying LEMS patients through its Catalyst Pathways program.

Author: Rare Daily Staff

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