Catalyst Pharmaceuticals Gets Positive FDA Advise On Firdapse NDA Refiling

Shares of Catalyst Pharmaceuticals, Inc. CPRX increased almost 4% on Feb 13 after the company announced positive results from a recent Type C meeting with the FDA on the resubmission of a new drug application (NDA) for its marketed drug, Firdapse (amifampridine phosphate). Catalyst Pharma is looking to get Firdapse approved in the United States for treatment of Lambert-Eaton myasthenic syndrome (LEMS).

The company expects to resubmit the NDA to the FDA for Firdapse in the first quarter of 2018.

Notably, Firdapse has already been approved in the EU for the symptomatic treatment of LEMS in adults.

The positive feedback from the FDA came on the back of preliminary data package submitted by Catalyst Pharma for the proposed NDA resubmission. The data package includes positive top-line findings from a second confirmatory phase III trial (LMS-003) on Firdapse for the aforementioned indication reported in November 2017 and positive outcomes from abuse liability studies showing no abuse potential for Firdapse.

We remind investors that in February 2016, Catalyst Pharma received a refusal-to-file letter from the FDA in connection with the NDA submitted for Firdapse to the regulatory body in December 2015. The FDA determined that the application was not sufficiently complete after a preliminary review and hence, the agency requires additional supporting information to review the application.

Thereafter, the company conducted the LMS-003 study on the candidate with the analysis achieving a statistical significance for both its co-primary endpoints — quantitative myasthenia gravis score (QMG) and subject global impression — and secondary endpoint. For the QMG endpoint, the data showed a clinically significant difference of 6.4 points between the Firdapse and placebo groups.

The top-line results showed that Firdapse has a major magnitude of effect for treating the symptoms of LEMS.  Additionally, it was well tolerated in the program and showed a similar safety profile like the earlier studies.

Catalyst Pharma's shares have significantly outperformed the industry in a year's time. The stock has skyrocketed 199.1% versus the industry's decrease of 0.8%.

Notably, Firdapse enjoys an Orphan Drug and Breakthrough Therapy status in the United States for the treatment of LEMS.

Catalyst Pharma had in-licensed development and commercialization rights to Firdapse in the United States from BioMarin Pharmaceutical Inc. BMRN.

Also, the company is working on developing Firdapse for additional indications. It is evaluating the drug in another phase III study for treatment of congenital myasthenic syndromes CMS. Top-line data from the study is expected in the second half of 2018. Positive results would allow Catalyst Pharma to add the CMS indication to Firdapse's label.

The company's efforts to develop Firdapse appear encouraging, given its significant commercial potential. An approval for the same is likely to be a huge growth driver for Catalyst Pharma.

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