Catalyst Pharmaceuticals Inc (NASDAQ:CPRX), a leading biotech stocks has licensed BioMarin Pharmaceutical Inc. (NASDAQ:BMRN), another top biotech stocks to develop, market and commercialize Firdapse in the U.S.
Firdapse is currently licensed in the European Union for treatment of LEMS in adults. The drug has Orphan Drug and Breakthrough Therapy designation in the United States for treatment of LEMS. However the drug is yet to be approved in the United States. In 2016, the company was issued with a refusal to file notice in relation to the New Drug Application for Firdapse. In its determination, FDA said Catalyst’s New Drug Application was not sufficient enough after preliminary review. As a result, and as per FDA’s requirements, Catalyst is required to carry out additional trials on the drug.
Catalyst is currently carrying out Phase III trial on Firdapse for treatment of LEMS so as to get the approval in the U.S. the company managed to strike a deal with FDA under a Special Protocol Assessment (SPA) for clinical endpoint, protocol design and statistical analysis in Oct 2016. Results from the study are expected in the second quarter of 2017. A positive result from the study will allow Catalyst to resubmit its New Drug Application for Firdapse in the second half of 2017.
Additionally, the company is developing Firdapse for more indications. Catalyst is also carrying out a sponsored phase II and Phase II trial of Firdapse for treatment of MuSK-antibody positive MG. the company in May 2017 reported the Phase II/III results which showed a lot of improvement which according to investigators, is an indication of big clinical benefits to patients. The company seems to be making impressive steps in the development of Firdapse given the tremendous commercial potential in the market.
Apart from Firdapse, the company is also developing CPP-115 which is still in the early stages of development. Addionally, Catalyst is developing a generic type of Sabril for which it has licensed Lundbeck Inc.in to market in the U.S. market. Sabril is used in the treatment of complex partial seizures and infantile spasms.