Marlborough’s Advanced Cell Technology Inc. has taken a step toward receiving orphan drug designation in Europe for a treatment for Stargardt’s disease.
The European Medicines Agency gave the human embryonic stem cell-based treatment a positive opinion. The opinion now goes to the European Commission, which will determine whether to grant the orphan drug status. The U.S. FDA approved an orphan drug designation for the same product last year.
If the European designation receives final approval it would offer the company regulatory and financial benefits as it develops and markets the treatment.
Stargardt’s disease, also called Stargardt’s Macular Dystrophy, is a genetic disease that causes progressive vision loss. Orphan drug designations provide incentives for companies to develop treatments for serious diseases affecting a small percentage of the population.
