Lambert-Eaton Myasthenic Syndrome (LEMS) is an autoimmune disorder. It’s a rare condition characterized by muscle weakness in the limbs. When the muscle weakness includes the respiratory muscles, the disease can be life threatening. In addition, half of those with a LEMS diagnosis also have an underlying malignancy such as small cell lung cancer, and for some LEMS is the first cancerous sign.
In the United States, the condition affects one in every 100,000 people. But we have good news for this community-
Firdapse, created by Catalyst Pharmaceuticals, has just been approved by the FDA as a new treatment for LEMS!
How does the drug work?
Firdapse is administered orally by 10mg tablets. It’s a channel blocker that slows repolarization of the presynaptic membrane. It allows for a greater release of ACh into the synaptic cleft which improves muscle function.
Firdapse already had Orphan Drug Designation as well as Breakthrough Therapy Designation. Additionally, it was given Priority Review by the FDA. However, it had not received approval yet for LEMS. Now, Firdapse can officially be prescribed for adults with the condition. Its been approved for use in both the United States and the European Union and it should be readily available by early 2019.
“The FDA’s approval of Firdapse is a potentially transformative milestone in the lives of patients suffering with LEMS.”
The efficacy of Firdapse was determined through two phase three clinical trials. Patients in the trials placed on Firdapse showed significant improvements in muscle function. It also reduced muscle weakness. These improvements were rapid and sustained.
The approval of Firdapse for use in both the United States and the European Union is certainly noteworthy and looking forward, we’re excited to seeing what other diseases Firdapse may prove efficacious for.
You can tune in to a conference call, held by Catlayst Pharmaceuticals, to discuss the cost and commercialization plan for the drug on December 13th. Anyone may listen in by clicking here. The call will be held at 8:30am ET.
In the meantime, you can read more about Firdapse and its approval here.