The orphan drug designation is important to Athersys because its benefits to the company include funding for certain clinical studies, study-design assistance, tax incentives and seven years of market exclusivity for the product upon regulatory approval, according to a statement from Athersys.
The FDA grants the designation to incentive companies that are working on drugs to treat rare diseases that fill unmet medical needs.
Graft-versus-host disease occurs after a stem cell or bone marrow transplant in which the newly transplanted material attacks the transplant recipient’s body.
Athersys’ stem cell therapy, called MultiStem, is currently in a Phase 1 clinical trial to evaluate its safety in support of bone marrow transplantation for the treatment of certain cancers of the blood and immune system, where GvHD is a common complication, according to the statement.
Preclinical data have shown that MultiStem suppresses immune responses that are factors in causing GvHD. Athersys has exclusive development rights for oncology applications of MultiStem. The company has partnered with Pfizer for developing treatments for inflammatory bowel disease and Angiotech for cardiovascular disease treatments.
MultiStem is an off-the-shelf stem cell treatment derived from the bone marrow of adults or other non-embryonic sources. The cells have a drug-like effect, reducing inflammation, protecting damaged tissue and forming new blood vessels, and then are cleared from the body.
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