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Catalyst's Firdapse Receives FDA Approval for Rare Disease

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Catalyst Pharmaceuticals Inc.’s (CPRX - Free Report) Firdapse (amifampridine) tablets obtained FDA approval for the treatment of Lambert-Eaton Myasthenic Syndrome (LEMS) in adults. Firdapse becomes the first FDA approved treatment for LEMS.

So far this year, Catalyst’s shares have lost 19.9% compared with the industry’s 9.6% decline.

LEMS is a rare automimmune disorder that affects the connection between nerves and muscles, and causes weakness and other symptoms in affected patients. LEMS is an ultra-rare disease and the prevalence of LEMS is estimated to be three per million individuals worldwide. The market, therefore, represents significant potential for the drug. LEMS may be associated with other autoimmune diseases, but more commonly occurs in patients with cancers like small cell lung cancer, where its onset precedes or coincides with the diagnosis of cancer. 

The efficacy of Firdapse was studied in two studies where the adult patients received Firdapse or placebo. The studies measured the Quantitative Myasthenia Gravis score (a scale assessing muscle weakness) and the Subject Global Impression (a seven-point scale, on which patients rated their overall impression of the effects of the study treatment on their physical well-being). For both measures, patients receiving Firdapse experienced a greater benefit than those on placebo.  

In March 2018, Catalyst submitted a new drug application (NDA) to the FDA, which addressed the two issues raised in the 2016 Refusal to File letter against the previous NDA submission. The letter included all additional information requested by the FDA. The NDA was accepted by the FDA in May 2018 and the agency granted Priority Review. Firdapse also received Orphan Drug status in the United States for the treatment of congenital myasthenic syndromes (CMS) and myasthenia gravis (MG).

Meanwhile, the company is also working on developing Firdapse for additional indications. Catalyst initiated an investigator-sponsored phase II/III study on Firdapse for the symptomatic treatment of MuSK-antibody positive MG in February 2016. In March 2017, the company reported top-line data from the study. In April 2018, the company enrolled the first patient in its phase III study for MuSK-antibody positive myasthenia gravis. The company expects to announce top-line results in the second half of 2019.

Firdapse is the first approved product for Catalyst and thus is a big boost for the company. With the approval, the company will start generating revenues.

Catalyst Pharmaceuticals, Inc. Price

Zacks Rank & Stocks to Consider

Catalyst is a Zacks Rank #3 (Hold) stock.

Some better-ranked stocks worth considering are Bristol-Myers Squibb Company (BMY - Free Report) , Gilead Sciences Inc. (GILD - Free Report) and Merck & Co. (MRK - Free Report) . While Bristol-Myers and Gilead sport a Zacks Rank #1 (Strong Buy), Merck carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

Bristol-Myers’ earnings per share estimates have increased from $3.62 to $3.87 for 2018 and from $3.82 to $4.08 for 2019 over the past 60 days. The company delivered a positive earnings surprise in all of the trailing four quarters with average of 11.99%.

Gilead’s earnings per share estimates have increased from $6.60 to $6.93 for 2018 and from $6.53 to $6.83 for 2019 over the past 60 days. The company delivered a positive earnings surprise in three of the trailing four quarters with average of 6.99%.

Merck’s earnings per share estimates have increased from $4.27 to $4.34 for 2018 and from $4.63 to $4.71 for 2019 over the past 60 days. The company delivered a positive earnings surprise in all of the trailing four quarters with average of 3.96%. Shares of the company have increased 40.5% year to date.

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