Catalyst Pharmaceuticals, Inc. CPRX announced that the FDA has granted orphan drug designation to Firdapse (amifampridine phosphate) for the treatment of myasthenia gravis.
Orphan drug designation is given to drugs being developed for rare diseases/disorders that affect less than 200,000 people in the U.S. Incentives that come with orphan drug status include close guidance from the FDA that may accelerate the time to marketing approval, orphan drug grants, tax credits and a period of market exclusivity upon marketing approval.
As per the information provided by the company, myasthenia gravis caused by antibodies to the muscle-specific kinase (MuSK-MG) is a rare disease, estimated to affect 5–8% of all myasthenia gravis patients, which means approximately 4,500 patients in the U.S.
The company is currently supporting an investigator-sponsored, randomized, double-blinded, placebo-controlled study evaluating Firdapse for the treatment of patients with MuSK-MG. Top-line results from this study are expected in early 2017.
Assuming positive results from the trial and availability of funds, Catalyst Pharma plans to initiate a registration quality trial in the U.S. to evaluate Firdapse in the treatment of MuSK-MG.
We remind investors that the FDA has granted Breakthrough Therapy Designation to Firdapse for the treatment of Lambert-Eaton myasthenic syndrome (LEMS). Firdapse also enjoys orphan drug status for LEMS and congenital myasthenic syndromes (CMS) in the U.S. Moreover, the drug is approved in Europe for the symptomatic treatment of adults with LEMS.
Firdapse’s approval in the U.S. is critical to the company’s success. Earlier this year, the company received a refusal-to-file letter from the FDA in connection with its NDA for Firdapse. The agency had determined that the company’s NDA was insufficient after a preliminary review. Consequently, the company was required to conduct additional studies as per the agency’s requirements, which translated into additional costs.
Currently, Catalyst Pharma carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the health care sector include Pacira Pharmaceuticals, Inc. PCRX, Corcept Therapeutics Incorporated CORT and ANI Pharmaceuticals, Inc. ANIP. All of these stocks sport a Zacks Rank #1 (Strong Buy).
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