Builds on FDA’s Granting of First-Ever Orphan Status for ACT’s Treatment Using Embryonic Stem Cells
MARLBOROUGH, Mass.--(BUSINESS WIRE)-- Advanced Cell Technology, Inc. (“ACT”; OTCBB: ACTC), a leader in the field of regenerative medicine, announced today that, working with the European regulatory consulting firm TMC Pharma Services, it has filed for Orphan Drug Designation with the European Medicines Agency (EMA) for its Retinal Pigment Epithelial (RPE) Cells for use in treatment of Stargardt’s Macular Dystrophy (SMD), also known as Stargardt’s Disease. Orphan drug designation may provide ACT with several advantages, including ten years of marketing exclusivity for embryonic stem cell derived RPE cells upon market authorization for the indication, financial incentives such as fee reductions or waivers, provide free scientific advice during the drug development process, and access to a centralized review process for all 27 member states.
“In the past month, we announced significant milestones in our macular degeneration program,” said Gary Rabin, ACT’s Interim Chairman and CEO. “We have been approved to begin our first clinical trial in the United States for the treatment of SMD using stem-cell derived RPE cells. We have also filed an investigational new drug application with the FDA for treating dry age-related macular degeneration, or Dry AMD, which represents a $25-30 billion market in the US and Europe alone, with no treatments currently available. Earlier this year, we received orphan drug designation from the U.S. Food and Drug (FDA) Administration for our SMD program, which grants the Company seven years of marketing exclusivity in the U.S., among other benefits. Building on this momentum, ACT has now taken the next important step in expanding our global efforts by filing for Orphan drug status in Europe, identifying clinical trial sites and establishing our European operations.”
“The sheer numbers of people suffering from these diseases are truly astounding,” stated Robert Lanza, MD, ACT’s Chief Scientific Officer. “Although the initial studies are designed to evaluate the safety and tolerability of this therapy, in picking Stargardt’s as our leading indication for macular degenerations, we made the determination that based on the patient population to be enrolled during the Phase I and II studies, Stargardt’s was the disease with the highest likelihood for the clinicians to see incorporation of RPE and early signals of efficacy. Degenerative diseases of the retina, which includes Stargardt’s Disease, are among the most common causes of untreatable blindness in the world. As many as thirty million people in the United States and Europe suffer from macular degeneration. Through a culmination of years of innovation and hard work by ACT’s scientific team, we believe we are finally in a position to offer treatment.”
Dr. Lanza added, “While everyone at ACT is profoundly saddened by the tragic loss of our close friend and leader Bill Caldwell, we are, to a person, committed to realizing his dream for ACT and the transforming role that he saw for stem cell therapies in potentially improving the lives of millions of people. Not too long ago, in accepting the responsibility for the hopes of macular degeneration patients, Bill boldly asserted, ‘We do not intend to let them down.’ We will do everything in our power to honor that promise. ACT is uniquely positioned within the regenerative medicine field. We have the technology and a wonderful scientific and development team. We have a number of very promising programs in our pipeline, and I have no doubt that these will result in the legacy to regenerative medicine, and to medicine as a whole, that Bill Caldwell envisioned.”
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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