Advanced Cell Technology, Inc. (OTCBB: ACTC), a biotechnology company
applying cellular technology in the field of regenerative
medicine, announced today that the U.S. Food and Drug Administration
(FDA) has granted orphan drug designation for the company’s MA09-hRPE
cells for use in the treatment of Stargardt’s Macular Dystrophy (SMD).
As a result, the company is eligible to receive a number of benefits,
including tax credits, access to grant funding for clinical trials,
accelerated FDA approval and allowance for marketing exclusivity after
drug approval for a period of as long as seven years.
“We are pleased that the FDA has, for the first time, granted orphan
drug status for the use of an embryonic stem cell derived therapy in
treating an unmet medical need”
“We are pleased that the FDA has, for the first time, granted orphan
drug status for the use of an embryonic stem cell derived therapy in
treating an unmet medical need,” said Edmund Mickunas, Vice President
Regulatory. “We believe that our terminally differentiated RPE cells
represent a promising treatment for patients with SMD and expect to be
in a position to accelerate clinical development and hopefully make RPE
cellular therapy available to the majority of patients sooner.”
US orphan drug designation is granted to companies with products aimed
at treatment of a rare disease or condition that affects fewer than
200,000 Americans. The National Institutes of Health (NIH) recently
proposed broadening the definition of a human embryonic stem cell to
include ACT’s “single blastomere technology platform” which was used to
derive ACT’s MA09-hRPE cells. The Company believes that the SMD program
should be eligible for federal funding once the change is published in
the Federal Register.