Firdapse gets orphan drug status for treatment of Myasthenia Gravis

Release Date: 12-Nov-2016

Catalyst pharmaceuticals Inc., a biopharmaceutical company, declare its novel therapy Firdapse (amifampridine phosphate) as orphan drug for the treatment of patients with Myasthenia Gravis, as approved by Food and Drug Association (FDA). The company focuses on developing and commercializing new therapies for the treatment of rare debilitating diseases. 


Myasthenia Gravis is the rare neuromuscular disease antibodies to the muscle-specific kinase (MuSK-MG) which inflicts approximately 5-8% of Myasthenia Gravis patients which is equal to 4,500 patients in the US. The characteristics of myasthenia gravis are predominance in women, prominent bulbar involvement, sever clinical conditions and resistant to treatment. Presently, there are anticholinesterase inhibitors or immunosuppressant available for the treatment of the disease.


FDA provides a number of benefits to the drug developers or pharmaceutical companies through development and commercialization. Orphan Drug designation is granted by the FDA's Office of Orphan Products Development for drugs that are expected to provide significant therapeutic advantage over existing treatments and that target conditions affecting 200,000 or fewer U.S. patients annually. Orphan Drug designation qualifies a company for several benefits under the Orphan Drug Act 1983. These benefits include assistance with clinical study design in drug development, tax credits for qualified clinical trials costs, exemptions from certain FDA application fees, and seven years of market exclusivity upon regulatory product approval.



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