GenSight Biologics, a biopharma company that discovers and develops innovative gene therapies for neurodegenerative retinal diseases and diseases of the central nervous system, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to the Company’s product candidate GS030 for the treatment of retinitis pigmentosa.
“The Orphan Drug Designation both in Europe and in the United States, together with the Advanced Therapy Medicinal Product classification in Europe, fully recognize the urgent and unmet medical need for a safe and effective treatment for retinitis pigmentosa patients, and highlight the potential of optogenetics and GS030 to address it,” commented Bernard Gilly, Chief Executive Officer of GenSight Biologics.
GS030 is currently undergoing a Good Laboratory Practices (GLP) regulatory toxicity study, and is expected to enter the clinic with a Phase I/II clinical trial in retinitis pigmentosa patients in Q3 2017, subject to toxicity results and future regulatory review.
The FDA grants orphan drug designation status to provide incentives to develop medicinal products to treat, prevent or diagnose diseases or conditions that affect no more than 200,000 persons in the United States. The orphan drug designation provides GenSight with incentives and benefits in the US, including a 7-year period of market exclusivity if GS030 is approved for the treatment of retinitis pigmentosa patients.
GS030 had received both Orphan Drug Designation and Advanced Therapy Medicinal Product classification in Europe in September 2016.