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Catalyst's Fridapse Gets Priority Review by FDA, Shares Up

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Shares of Catalyst Pharmaceuticals, Inc. (CPRX - Free Report) rose almost 8.2%, after the company announced that the FDA has accepted the new drug application (NDA) for its lead pipeline candidate, Firdapse, and granted Priority Review to the same. The FDA has set an action date of Nov 28, 2018.

Firdapse has been developed for the symptomatic treatment of Lambert-Eaton myasthenic syndrome (“LEMS”). The NDA submission addressed the two issues raised in the 2016 Refusal to File letter during the previous Firdapse NDA submission and included all additional information requested by the FDA.

Priority Review designation from the FDA is generally granted to drugs that have the potential to provide significant improvements in the safety and effectiveness of the treatment, prevention or diagnosis of a serious disease. 

Year to date, Catalyst’s shares have declined 2% against the industry’s increase of 0.8%.

Firdapse is currently approved in the European Union (EU) for the symptomatic treatment of LEMS in adults. However, the drug is not yet approved in the United States.  In January 2017, it held a Type C meeting with the FDA, wherein Catalyst was advised, based on the briefing documents, that the company’s proposed NDA should be sufficient for resubmission. In November 2017, the company announced positive top-line results from its second phase III study of Firdapse in patients with LEMS.

Catalyst also announced its decision of not including in this FDA submission those limited types of congenital myasthenic syndromes (“CMS”) that are considered mechanistically similar to LEMS. Catalyst decided not to overcomplicate the review of its NDA submission for LEMS with this second indication.

The company is currently conducting a phase III study evaluating Firdapse for the treatment of CMS and expects to complete enrollment before the end of 2018 and report top-line results from this study in the first quarter of 2019. If the results of the trial are successful, the company plans to add the CMS indication to its labeling for Firdapse. 

Catalyst had in-licensed rights to Firdapse from BioMarin Pharmaceutical Inc. (BMRN - Free Report) in 2012 for the development and commercialization of the product in the United States. Also, Firdapse enjoys Orphan Drug and Breakthrough Therapy status in the United States for the treatment of LEMS. Firdapse received Orphan Drug status in the United States for the treatment of congenital myasthenic syndromes (“CMS”) and myasthenia gravis (“MG”).

LEMS is an ultra-rare disease even though several drugs and treatment therapies like steroids, azathioprine, other immunosuppressants and intravenous immunoglobulin are currently in use. Guanidine HCl tablets are approved for the treatment of LEMS. However, due to significant side effects, the drug is not viewed as an effective treatment. This, in turn, has created ample space for introducing new therapies for LEMS.

Therefore, approval of Firdapse in these indications will be a huge boost for the company, given its commercial potential in the target markets.

Stocks That Warrant a Look

Catalyst  carries a Zacks Rank #3 (Hold).

Some better-ranked stocks from the same space are Illumina, Inc. (ILMN - Free Report) , Ligand Pharmaceuticals . While Illumina sports a Zacks Rank #1 (Strong Buy), Ligand carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

Illumina’s earnings per share estimates have moved up from $4.83 to $4.86 for 2018 and from $5.57 to $5.60 for 2019 over the past 30 days. The company delivered a positive earnings surprise in all the trailing four quarters with an average beat of 23.17%. The stock has rallied 24.7% so far this year.

Ligand’s earnings per share estimates have been revised upward from $3.91 to $4.37 for 2018 and from $4.61 to $4.98 for 2019 over the past 60 days. The company came up with a positive earnings surprise in all of the trailing four quarters with an average beat of 31.79%. The company’s shares have rallied 39.9% year to date.

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