US Food and Drug Administration has granted orphan drug status to Marinus Pharma’s ganaxolone andndash; a CNS-selective GABAA modulator andndash; for the treatment of Fragile X Syndrome, a genetic condition that causes intellectual disability. The pharmaceutical company focuses on developing and commercializing therapeutics to treat epilepsy and neuropsychiatric disorders.
Orphan drug designation is granted by the FDA Office of Orphan Products Development (OOPD) to novel drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S. The OOPD provides incentives for sponsors to develop products for rare diseases, which may include tax credits towards the cost of clinical trials and prescription drug user fee waivers.
For Orphan Drug Clinical Insight Reports Contact: neeraj@kuickresearch.com
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