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Catalyst Pharmaceuticals’ neuromuscular drug succeeds in seven-patient trial

PBR Staff Writer Published 16 March 2017

Catalyst Pharmaceuticals said its experimental drug to treat patients with a severe form of myasthenia gravis (MG), a debilitating neuromuscular disease, met the main goals of a study.

The drug, Firdapse, was being tested on seven patients with Musk-MG, a rare subpopulation of MG patients.

The seven-patient trial assessed the safety, tolerability and efficacy of Firdapse (amifampridine phosphate). The drug met both of the co-primary efficacy endpoints.

The achieved results were statistically and clinically significant on the given parameters of change from baseline in total Quantitative Myasthenia Gravis score and CFB in total Myasthenia Gravis Activities of Daily Living score.

Catalyst said various secondary efficacy measures also achieved statistical significance and amifampridine phosphate was well tolerated in this population of patients.

Catalyst Pharmaceuticals chief medical officer Gary Ingenito said:  "While several effective treatment options exist for the anti-acetylcholine receptor form of myasthenia gravis (AcHR-MG), MuSK-MG has been particularly refractory to current MG treatment options and represents an unmet medical need in the MG community of patients.

"If the significant clinical effect observed in this trial is reproduced in a multicenter trial, amifampridine phosphate would, upon approval, likely become the first line standard of care for MuSK-MG.”

The company provided funding, study drug, and placebo for the MuSK-MG proof-of-concept trial.

Based on the results, the company plans to discuss with the Food And Drug Administration (FDA) on carrying out a registration trial in the US to assess amifampridine phosphate for the symptomatic treatment of patients with MuSK-MG.

Catalyst noted that even though many patients with MuSK MG are currently treated with anticholinesterase inhibitors or immunosuppressants, such patients do not generally respond adequately to the treatments.

Image: Catalyst Pharmaceuticals’ neuromuscular drug succeeds in seven-patient trial. Photo: courtesy of ponsulak /