Catalyst Pharmaceuticals has reported positive top-line results from the MuSK-MG clinical trial of Firdapse to treat patients with myasthenia gravis (MG).
Firdapse is formulated with the firm's amifampridine phosphate designed to treat Lambert-Eaton myasthenic and congenital myasthenic syndromes.
The results showed that the trial has met both the co-primary efficacy endpoints of change from baseline (CFB) in total quantitative myasthenia gravis (QMG) score and CFB in total myasthenia gravis activities of daily living (MG-ADL) score.
The randomised, double blind, placebo-controlled, single site, outpatient, investigator-sponsored MuSK-MG proof-of-concept trial evaluated the safety, tolerability and efficacy of amifampridine phosphate in patients with a positive serological test for the anti-MuSK antibodies (MuSK-MG).
Catalyst chief medical officer Gary Ingenito said: "If the significant clinical effect observed in this trial is reproduced in a multicentre trial, amifampridine phosphate would, upon approval, likely become the first line standard of care for MuSK-MG.
"Based on these results, we intend to discuss with FDA conducting a registration trial in the US, evaluating amifampridine phosphate for the symptomatic treatment of patients with MuSK-MG."
The trial involved a cross-over design with three treatment periods to determine the effect of the treatment, as well as correct any subjective carryover from earlier treatment periods into later treatment periods.
The trial's secondary efficacy measures also achieved statistical significance and the drug was found to be well-tolerated.
Firdapse had previously secured orphan medicinal product designation from the European Union (EU) for the treatment of Lambert-Eaton myasthenic syndrome.