SparingVision Receives European Orphan Designation for its Drug Candidate SPVN06 Dedicated to Inherited Retinal Dystrophies

SparingVision announced 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 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,” Florence Allouche, SparingVision President and Chief Executive Officer, said in a company news release. “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 the European Medicine Agency (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.