Advanced Cell Technology (ACTC.OB) Orphan Drug Designation from EMA for hESC-Derived RPE Cells
This positive opinion of ACTC.OB’s human embryonic stem cell (hESC) derived retinal pigment epithelial (RPE) cells designates an orphan medicinal product for the treatment of Stargardt’s disease.
The Bottom Line: This opinion will now be forwarded by the EMA to the European Commission for their adoption. This follows on the US FDA’s granting of Orphan Drug Designation (ODD) and by the European Commission which provides regulatory and financial incentives for companies to develop and market therapies that treat a life-threatening or chronically debilitating condition affecting no more than 5 in 10,000 persons in the EU. In addition to a 10-year period of marketing exclusivity in the EU after product approval, ODD provides companies with scientific advice and regulatory assistance from the EMA during the product development phase, direct access to centralized marketing authorization, as well as reductions in certain fees associated with the application and approval process.