FDA declares Firdapse as orphan drug for treatment of Myasthenia Gravis
Release Date: 14-Oct-2016
Catalyst Pharmaceuticals, a company dedicated to drug discovering and developing for patients with rare diseases, announces that orphan drug designation has been granted to Firdapse to treat patients suffering from myasthenia gravis, by Food and Drug Association (FDA).
Antibodies to the muscle-specific kinase (MuSK-MG), results in a rare Myasthenia Gravis disease, found to be dominant in females and characterized by prominent bulbar involvement, more severe clinical condition and resistance to treatment. Many patients with Myasthenia Gravis are recorded to be unresponsive to the current treatment with anticholinesterase inhibitors or immunosuppressant.
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. The benefits apply across all stages of drug development and include an accelerated approval process; seven years of market exclusivity following marketing approval; tax credits on U.S. clinical trials; eligibility for Orphan Drug grants; and waiver of Prescription Drug User Fee Act (PDUFA) and certain other administrative fees.
For Orphan Drug Clinical Insight Reports Contact: firstname.lastname@example.org
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