Aldagen, Inc announced that it has received orphan drug designation for ALD-101 from the US Food and Drug Administration (FDA). The designation was granted for the use of ALD-101 to improve patient outcomes by decreasing time to platelet and neutrophil engraftment in patients with inherited metabolic disorders undergoing umbilical cord blood transplantation.
Aldagen is currently conducting a phase-3 trial infusing ALD-101 in patients with inherited metabolic disorders undergoing umbilical cord blood transplantation. Cord blood transplantation is commonly used to treat children with inherited metabolic diseases, including Krabbe syndrome, metachromatic leukodystrophy, Hurler syndrome and adrenoleukodystrophy.
These diseases are progressive, degenerative and often fatal. In many cases, the only treatment available to these patients is a transplant of blood-forming stem cells found in cord blood. Umbilical cord stem cells are located in the umbilical cord of a newborn at birth. Umbilical cord stem cells are not embryonic stem cells. The FDA orphan drug designation, administered by the Office of Orphan Products Development, provides potential incentives such as funding for clinical studies, study design assistance, waiver of FDA user fees, tax credits and, importantly, up to seven years of market exclusivity upon marketing approval.
"FDA orphan drug designation for ALD-101 is an important asset in Aldagen's development of ALD-101," said Tom Amick, chairman and chief executive officer of Aldagen. "We are encouraged by the reductions in engraftment times seen in the phase-1 ALD-101 trial and look forward to continuing our on-going phase-3 trial of ALD-101."
ALD-101 is the population of stem cells that Aldagen produces from a portion of an umbilical cord blood unit using our proprietary stem cell isolation technology.
Aldagen is a biopharmaceutical company developing proprietary regenerative cell therapies that target significant unmet medical needs.