GenSight Biologics announces orphan drug status for GS030 for Retinitis Pigmentosa

Release Date: 05-Feb-2017

GenSight Biologics, a biopharma company focused on developing innovative gene therapy for neurodegenerative retinal diseases and diseas for central nervous system, has received Orphan Drug Designation by US Food and Drug Administration (FDA) for leading product candidate, GS030 for the treatment of Retinitis Pigmentosa. The drug is currently undergoing Good Laboratory Practices (GLP) for regulatory toxicity study and is projected to  undergo clinical study in Phase andfrac12; clinical trial in retinitis pigmentosa patients in Q3 2017, subject to toxicity results and future regulatory review.


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.


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