Osiris Therapeutics Inc has received orphan drug designation from the US Food and Drug Administration for Prochymal, an adult stem cell product under investigation for the treatment of acute Graft vs. Host Disease (GVHD). Orphan drug status provides market exclusivity to the company for up to 7 years.
Prochymal is a formulation of a specific type of adult stem cell that has the ability to modulate the immune system in a way that may benefit patients suffering from a variety of immunological disorders.
Osiris has two ongoing clinical trials of Prochymal for the treatment of GVHD, a complication of bone marrow transplantation. In January of this year, Prochymal became the first stem cell drug to be granted fast track status from FDA. FDA established the fast track programme to expedite the development of promising treatments for life-threatening conditions, states a company release.
"Having both orphan drug designation and fast track status clearly adds support to the development of Prochymal for patients suffering from life-threatening GVHD," said C. Randal Mills, president and CEO of Osiris Therapeutics.
GVHD is a life threatening immune disorder that affects approximately 50% of patients who receive donated bone marrow transplants for the treatment of cancers such as leukaemia. GVHD is a form of immune rejection between the newly transplanted bone marrow cells and the patient's own cells, leading to a cascade of syndromes often resulting in death.
Osiris Therapeutics, Inc. is the one of the leading companies in adult stem cell therapy.