PARIS--(BUSINESS WIRE)--SparingVision, a biotechnology company specializing in the research and development of innovative therapies for treatment of hereditary retinal degenerative diseases such as retinitis pigmentosa, announces today the decision of the European Commission to grant Orphan Drug Designation for SPVN06 for the treatment of inherited retinal dystrophies. The decision is based on a positive opinion from the European Medicine Agency’s (EMA) Committee for Orphan Medicinal Products (COMP).
SPVN06 is a breakthrough treatment for retinitis pigmentosa, an orphan inherited retinal disease which leads to blindness and affects nearly two million worldwide. SPVN06 uses a gene therapy-based approach independent of mutated genes and by a single subretinal injection of proprietary neurotrophic factors, aims at stopping and preventing the degeneration of photoreceptors leading to blindness.
“This orphan designation is very encouraging news both for SparingVision and our innovative mutation-independent approach of gene therapy, and for patients suffering from the debilitation condition of retinitis pigmentosa,” says Florence Allouche, SparingVision President and Chief Executive Officer. “As planned, we are continuing our pre-clinical development and look forward to starting our Phase 1 trial in patients with retinitis pigmentosa by 2021 in France and the United States”.
In Europe, orphan status is obtained from European Medicine Agency’s (EMA) for a drug in a condition affecting less than 5/10,000 people in order to encourage the development of “orphan drug” for a small number of patients. This status confers a number of advantages to the further clinical development, allowing faster registration and additional protection with a 10-year market exclusivity after the Market Authorization.
SparingVision is a biotechnology company focused on the discovery and development of innovative therapies for the treatment of blinding inherited retinal diseases. SparingVision is developing SPVN06, a gene-independent treatment for retinitis pigmentosa, the most common inherited retinal degeneration. There is currently no treatment to treat all genetic forms of this rare retinal disease that leads to blindness and affects nearly 2 million worldwide. SparingVision is a spin-off of the Paris Vision Institute. Bpifrance, Foundation Fighting Blindness (US) and Fondation Voir & Entendre invested €15.5 million in the company. SparingVision was laureate and Grand Prize of i-Lab 2017, the French National Innovative Companies Competition. SparingVision has been awarded from the EIC Accelerator program (H2020 SME instrument Phase 2), securing non-dilutive funding of €2.5 million.