We issued an updated report on Catalyst Pharmaceuticals Inc. CPRX on Jul 4. 

In November 2018, the company’s Firdapse (amifampridine) tablets obtained FDA approval for the treatment of Lambert-Eaton Myasthenic Syndrome (LEMS) in adults. The drug became the first FDA-approved treatment for LEMS and also the first approved product in Catalyst’s portfolio. 

The drug was launched in January 2019. In the first quarter of 2019, revenues came entirely from Firdapse. The initial uptake of the drug was encouraging. The company is optimistic about the drug and expects it to drive revenues in the upcoming quarters. 

Catalyst Pharma’s shares have skyrocketed 111% year to date compared with the industry’s growth of 11.8%.

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The company is also working on developing Firdapse for additional indications. It is currently conducting phase III studies on the drug for the treatment of congenital myasthenic syndromes (“CMS”) and anti-MuSK -antibody positive myasthenia gravis (MuSK-MG). The company expects to report top-line results from the phase III studies in the second half of 2019. 

Catalyst is also conducting a proof-of-concept study, evaluating Firdapse as a symptomatic treatment for patients with SMA type III. The company expects to report top-line results from the study in the first half of 2020. A potential approval of the drug for additional indications will further drive sales for the company. 

Apart from these, the company is in early stages of developing a longer-acting formulation of Firdapse to provide a better treatment alternative that will integrate well with patients' daily activities. It is working to develop a formulation that provides optimum level of efficacies through day and night. Hence, the formulation will be more convenient and patient-friendly. 

However, all the other candidates in Catalyst’s portfolio are in early stages of development. Thus, the successful development and commercialization of drug candidates are highly crucial for the company. 

Recently, Catalyst Pharmafiled a suit against the FDA and several related parties, challenging the recent approval of Jacobus Pharmaceutical Co's drug, Ruzurgi, for the treatment of LEMS in pediatric patients. 

According to the lawsuit, the approval of Ruzurgi, in May 2019, violated provisions of the FDA regulations regarding labeling, resulting in misbranding in violation of the Federal Food, Drug, and Cosmetic Act (FDCA). The lawsuit also alleges that the approval also violated Catalyst's rights to exclusivity for its drug, Firdapse. Among other remedies, the suit seeks an order vacating the FDA's approval of Ruzurgi.