FDA declares BHV-0223 as orphan drug to treat ALS

Release Date: 03-Jan-2017

Biohaven declares that its leading treatment candidate, BHV-0223 received orphan drug designation from US Food and Drug Administration (FDA) for the treatment of Amyotrophic Lateral Sclerosis (ALS).


BHV-0223 is formulation of glutamate modulation agent. It is an orally dissolved table, sublingually absorbed formulation of riluzole. The drug is absorbed through the oral mucosa, the firm is hoping for the drug to bypass first-pass metabolism and offer effective doses of the drug at lower concentrations. Also, the drug edits the any inconvenience for patients such as difficulty in swallowing or the need of three hour fasting twice a day, with the tablet form.



FDA's Orphan Drug Designation program provides certain incentives for companies developing therapeutics to treat rare diseases or conditions, defined as those affecting less than 200,000 individuals in the U.S. A drug candidate and its sponsor must meet several key criteria in order to qualify for, and obtain, orphan drug status. Once a drug has received orphan drug designation, the developer qualifies for a range of benefits, including federal grants, tax credits, reduction in certain regulatory fees, and the potential for seven years of market exclusivity for the drug following FDA marketing approval.


For Orphan Drug Clinical Insight Reports Contact:  neeraj@kuickresearch.com

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