Paris, France, July 10, 2017 – Horama (www.horama.fr), a French biotech company based in Paris and specialized in gene therapy for the treatment of rare inherited retinal diseases, today announces that the U.S. Food and Drug Administration (FDA) has granted O rphan D rug D esignation (ODD) to HORA – RLBP1 , the company’s product for the treatment of retinitis punctata albescens , a r etinitis pigmentosa caused by mutation s in the RLBP1 gene .
✓ HORA – RBLP1 had already been granted ODD status by the European Commission , in October 2016, for the treatment of retinitis pigmentosa
✓ The FDA had also granted ODD status, in July 2016 , to another of Horama’s drug candidates, HORA – PDE6B, for the treatment of retinitis pigmentosa , which is caused by mutation s in the PDE6B gene
In the United States , the orphan drug designation is granted to products dedicated to the t reatment or prevention of diseases with unmet medical need, affecting fewer than 200,000 people in the United States. This status comes with significant incentives for companies, such as a seven – year marketing exclusivity in the United States after the pro duct is approved by the FDA for a specific disease .
In Europe , the orphan drug designation is granted to foster the development of new drugs for the treatment, prevention or diagnosis of lethal, disabling or chronic diseases or of conditions with unmet medical need affecting u p to five in every 10,000 people in the European Union. This status guarantees ten years of exclusive marketing rights, after being granted a marketing authorization , in all EU member states.
There are current ly more than 5 million people in the world suffering from retinal dystrophies , which are rare inherited retinal diseases leading to a complete loss of vision . To date, more than 200 genes have been identified as responsible for retinal dystrophies.
Horama focuses on gene therapy for the treatment of inherited r etinitis pigmentosa , which are considered orphan retinal diseases . The company develops treatments , based on adeno – associated virus (AAV) vectors, which slow down and even stop the loss of visu al function associated to retinal dystrophies.
“ Being granted an orphan drug status in Europe and the United States , for HORA – RLBP1 and HORA – PDE6B , marks a key step in the development of our two drug candidates, which will benefit from significant incentives in both territories . This status is granted after an evaluation of the rarity and severity of the condition , as well as the potential benefits o f the products in the target ed disease ,” says Christine Placet, CEO Horama.
Horama has a diversified product portfolio resulting from its gene therapy platforms, whose two main programs, HORA – PDE6B (retinitis pigmentosa) and HORA – RLPB1 ( retinitis punctata albescens ) , have recently been granted ODD status and should be clinical ly tested in the coming months.
Horama is a clinical – stage biotech company that develops gene – therapy treatments, based on recombinant adeno – associated virus (rAAV) vectors, targeting rare inherited retinal diseases. The company’s portfolio especially includes two products scheduled to enter into clinical trials in the coming months. Horama was founded in 2014 in Paris with the support of a group of scientists who ha d carried out one of the very first gene – therapy trials in ophthalmology, in 2011.
These world – renowned cofounders all have complementary skills .