Applied Genetic Technologies Corp. (AGTC) today announced the European Commission's (EC) approval of orphan medicinal product status for its gene therapy product.
The investigational product will be used to treat X-linked retinitis pigmentosa (XLRP), which causes progression vision loss and eventually blindness.
"Receiving orphan medicinal product designation from the EC for our XLRP gene therapy candidate is an important milestone for AGTC, and a positive step forward in our ongoing efforts to develop effective treatments for rare inherited retinal diseases," AGTC President and CEO Sue Washer said. "With many of our programs in or nearing clinical trials, this is an exciting time for the company and patients affected with ocular diseases with no currently available treatments."
XLRP is a genetic condition, caused by mutations in the RPGR gene. The progressive vision loss begins with night blindness and on the average, blindness by age 45. Testing on dogs with XLRP showed that gene therapy prevented and in some cases cured the animals' vision loss.
AGTC is also working on gene therapy products to treat X-linked retinoschisis and achromatopsia. It also received orphan drug designation from the EC and the U.S. Food and Drug Administration for those products. The designation provides incentives from the EU to develop treatments for rare diseases, including lower fees and protection from competitors.