Asterias Biotherapeutics Inc announces that AST-OPC1, its drug in development to help treat spinal cord injury, has received Orphan Drug Designation from the FDA.
The US Food and Drug Administration (FDA) Office of Orphan Products Development grants Orphan Drug Designation to products that treat rare diseases—those that affect fewer than 200,000 people in the United States, explains a media release from Asterias Biotherapeutics Inc.
AST-OPC1, derived from human embryonic stem cells, has three potentially reparative functions at the site of spinal cord injury, the company suggests in the release: produces neurotrophic factors, stimulates vasularization, and induces remyelination of denuded axons—all of which are critical for survival, regrowth, and conduction of nerve impulses through axons at the injury site.
In preclinical animal testing, administration of AST-OPC1 led to remyelination of axons, improved hind limb and forelimb locomotor function, dramatic reductions in injury-related cavitation and significant preservation of myelinated axons traversing the injury site, the release suggests.
Asterias is continuing to test AST-OPC1 on patients with complete cervical spinal cord injuries. Known as the SCiStar trial, it will test three escalating doses of AST-OPC1 administered at 14 to 30 days postinjury in such patients.
For more information, visit Asterias Biotherapeutics Inc.
[Source(s): Asterias Biotherapeutics Inc, PR Newswire]