Paris, June 30th, 2020 – 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 genomic medicines company, translating pioneering science into vision-saving treatments.
Founded to advance over 20 years of world-leading ophthalmic research from its scientific founders at the Paris Vision Institute, SparingVision is leading a step shift in how ocular diseases are treated, moving beyond single gene correction therapies. At the heart of this is a pipeline of gene independent treatments for rod-cone dystrophies. Lead products, SPVN06 and SPVN20, address mid and late stages of retinitis pigmentosa (RP)respectively. RP is the most common inherited retinal disease affecting two million people worldwide. These novel medicines could form the basis of a suite of new sight-saving treatments with potential applications across many other retinal diseases, regardless of genetic cause.
The Company is supported by a strong, internationally renowned team who aim to harness the potential of genomic medicine to deliver new treatments to all ocular disease patients as quickly as possible.
SparingVision has raised €60 million to date and its investors include 4BIO Capital, Bpifrance, Foundation Fighting Blindness (US), Fondation Voir & Entendre, UPMC Enterprises, Jeito Capital and Ysios Capital. For more information, please visit www.sparingvision.com.