Catalyst Pharmaceuticals, Inc.'s Firdapse met two primary endpoints in a phase 3 trial studying the drug for the symptomatic treatment of a muscle weakness disease, Lambert-Eaton myasthenic syndrome, or LEMS.
The U.S. Food and Drug Administration granted the medicine a breakthrough therapy designation. The company conducted the trial under an advanced declaration by the U.S. FDA through the Special Protocol Assessment.
Firdapse demonstrated statistically significant improvements in arm strength, leg strength, face and neck muscle performance and other metrics that were used to calculate the quantitative myasthenia gravis score. Patients in the trial also reported improvement of their symptoms over the course of treatment with Firdapse.
Firdapse was safe and well tolerated in the trial.
The company intends to submit the therapy for approval to the U.S. FDA in the first quarter of 2018. Firdapse is currently approved in Europe for symptomatic treatment in adults with LEMS.
In March, the company reported positive top-line results from the investigator-sponsored trial evaluating Firdapse as a treatment for myasthenia gravis patients with anti-MuSK antibodies, or MuSK-MG.