ACT Granted EU Orphan Medicinal Product Designation for hESC-Derived RPE Cells for Treatment of Stargardt’s Disease
MARLBOROUGH, Mass., June 27, 2011 /PRNewswire/ — Advanced Cell Technology, Inc. (“ACT”; OTCBB: ACTC), a leader in the field of regenerative medicine, announced today that the European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP) has officially granted ACT’s human embryonic stem cell (hESC)-derived retinal pigment epithelial (RPE) cells designation as an orphan medicinal product for the treatment of Stargardt’s Macular Dystrophy (SMD). This follows on the U.S. Food & Drug Administration’s (FDA) granting of Orphan Drug Designation for the same program from the company, last year.
“It is very gratifying to receive approval for orphan drug status 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. “SMD, a juvenile degenerative retinal disease, affects between 80,000 to 100,000 patients in the U.S. and Europe, and there is currently no treatment available for SMD. We are working with European regulatory agencies to gain approval for the trial design we have proposed for the SMD clinical trial in Europe. The initiation of our Phase 1/2 clinical trial for SMD in the U.S. is an important milestone, and we hope to be able to provide more good news to patients and our investors in the near future.”
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 (less than 175,000 patients in total) 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, and reductions in certain fees associated with the application and approval process.
ACT announced recently the enrollment of the first patients in two clinical trials using hESC-derived RPE cells for the treatment of SMD and Dry Age-Related Macular Degeneration.
About Stargardt’s Disease
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).
About Advanced Cell Technology, Inc.
Advanced Cell Technology, Inc. is a biotechnology company applying cellular technology in the field of regenerative medicine. For more information, visit http://www.advancedcell.com.
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