Advanced Cell Technology, Inc. (ACT), a leader in the field of regenerative medicine, announced that the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA) has announced a positive opinion of ACT’s human Embryonic Stem Cell (hESC)-derived Retinal Pigment Epithelial (RPE) cells for designation as an orphan medicinal product for the treatment of Stargardt’s disease.
These opinions will now be forwarded by the EMA to the European Commission for their adoption, which should follow in coming weeks. This follows on the US Food & Drug Administration’s granting of Orphan Drug Designation for the same programme from ACT, last year.
“We are very pleased to receive this positive opinion for Orphan Drug Designation from the EMA for our hESC-derived RPE cells for the treatment of Stargardt’s disease,” said Gary Rabin, ACT’s interim chairman and CEO. “There is currently no approved treatment for Stargardt’s disease, and we are working diligently to move this European trial forward as quickly as possible.”
Orphan Drug Designation by the European Commission 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 five in 10,000 persons in the European Union (EU).
In addition to a 10-year period of marketing exclusivity in the EU after product approval, Orphan Drug Designation 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.
Stargardt’s disease or Stargardt’s Macular Dystrophy is a genetic disease that causes progressive vision loss, usually starting in children between 10 to 20 years of age. Eventually, blindness results from photoreceptor loss associated with degeneration in the pigmented layer of the retina, called the Retinal Pigment Epithelium (RPE).
Advanced Cell Technology, Inc. is a biotechnology company applying cellular technology in the field of regenerative medicine.