According to a story from statnews.com, the recent FDA approval of a new treatment for Lambert-Eaton myasthenic syndrome could actually be bad news for patients. As it turns out, the syndrome has been treatable for years with an unapproved chemical compound called 3,4-DAP. The newly approved drug is effectively identical to the chemical, which has been used off-label since the 1980s.
About Lambert-Eaton Myasthenic Syndrome
Lambert-Eaton myasthenic syndrome (LEMS) is a rare autoimmune disease which is characterized by weakness of the limb muscles. In this autoimmune disease, the immune system develops antibodies against an ion channel that plays a vital role in neuromuscular function. Lambert-Eaton myasthenic syndrome commonly appears as the result of cancer, often small cell lung cancer. Genetic variants are often the cause when cancer is not involved. The disease presents with muscle weakness of the upper arms and legs, with the legs usually being most affected. Other symptoms include difficulty swallowing, double vision, drooping eyelids, orthostatic hypotension, ataxia, and reduced reflexes. Weakness may be temporarily relieved after exercise; hot temperatures seem to worsen symptoms. Treatment of comorbid cancer can resolve the disease; other treatments include immune system suppressors (IVIg and prednisone) and drugs that improve neuromuscular function. To learn more about Lambert-Eaton myasthenic syndrome, click here.
Getting Treatment Off-Label
Lambert-Eaton myasthenic syndrome patients have been fortunate in that obtaining 3,4-DAP has not been too difficult despite the fact that it was never approved for treating the disorder. Patients could pay $300 to $500 a month from certain pharmacies to get regular access. Jacobus Pharmaceutical Co., the small drug company that developed the compound, has also been supplying some patients with the drug free of charge.
No Cause for Celebration?
Unfortunately, Catalyst Pharmaceuticals’ new product Firdapse is expected to run about $100,000 in price. The drug costs almost nothing to make and the approval could also allow the company to halt the distribution of off label 3,4-DAP even though Catalyst is not the original creator.
The new approval was hailed by Catalyst and the FDA as a major victory for Lambert-Eaton myasthenic syndrome patients, who will now have access to an FDA approved treatment for the first time. However, patients who have been able to manage the condition cheaply with the off label version see things differently. Now it appears that they will be paying hundreds of thousands for a drug that they were previously getting for far less.
While the end result is not clear and primarily will depend on the actions of Catalyst and insurance companies, the situation could very possibly create a new and preventable problem for Lambert-Eaton myasthenic syndrome patients where there was none before.