GenSight Biologics receives Orphan Drug Designation for GS030 in Retinitis Pigmentosa
Thursday, September 01, 2016
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 European Commission, based on a favorable recommendation from the European Medicines Agency (EMA), has granted orphan drug designation (ODD) to the Company’s product candidate GS030 for the treatment of retinitis pigmentosa. The EMA also granted Advanced Therapy Medicinal Product (ATMP) classification to GS030.
“We are very excited that GS030 has received both Orphan Drug Designation and Advanced Therapy Medicinal Product classification in Europe, recognizing the urgent and unmet medical need for a safe and effective treatment for retinitis pigmentosa patients, and the potential of GS030 to address it. This will allow us to optimize future steps to further advance GS030 toward regulatory approval,” commented Jean-Philippe Combal, Chief Operating Officer of GenSight Biologics.
GS030 is currently undergoing preclinical development, and is expected to initiate in September 2016 a Good Laboratory Practices (GLP) regulatory toxicity study in non-human primates, prior to entering 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 European Commission 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 five in 10,000 persons in the European Union (EU). The orphan drug designation provides GenSight with incentives and benefits in the EU, including a period of market exclusivity if GS030 is approved for the treatment of retinitis pigmentosa patients.
Medicines classified as ATMP are deemed to offer groundbreaking innovative opportunities for the treatment of certain conditions with high unmet needs. The classification provides GenSight with scientific regulatory guidance from the EMA, notably on the applicable regulatory framework and pathway, as well as a centralized approval procedure in Europe.
Source : finance.yahoo.com
