US Food and Drug Administration has granted orphan drug status to Marinus Pharma’s ganaxolone.
Release Date: 28-Jan-2017
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: firstname.lastname@example.org
Need custom market research solution? We can help you with that too.
Kuick Research is a market research and analytics company that provides targeted information for critical decisions at business, product and
service levels. We are quick, predictive and known by the recommendations we have made in the past. Our result-oriented research methodology offers
understanding of multiple issues in a short period of time and gives us the capability to keep you full with loads of practical ideas.