GenSight Biologics, a biopharma company focused on neurology, has said today that the U.S. Food and Drug Administration (FDA) has designated its candidate GS030 as orphan drug for the treatment of retinitis pigmentosa.
Bernard Gilly, Chief Executive Officer of GenSight Biologics said that 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.
GS030 is currently undergoing a Good Laboratory Practices (GLP) regulatory toxicity study, and expects to begin with a Phase I/II clinical study in retinitis pigmentosa patients in in the third quarter this year, the company said in its press release.
Furthermore, with this designation, GenSight gets benefits in the US, which includes marketing GS030 for 7 years, if it gets approval for the treatment of retinitis pigmentosa.
In addition, the company added that GS030 had received both Orphan Drug Designation and Advanced Therapy Medicinal Product classification in Europe in September 2016.