Fate Therapeutics, Inc., a biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for cancer and immune disorders, announced that the US Food and Drug Administration (FDA) has granted Orphan Drug designation for ProTmune, the company's lead product candidate that is currently undergoing phase 1/2 clinical investigation. The FDA designation is for "prevention of graft-versus-host disease (GVHD) in patients undergoing allogeneic hematopoietic cell transplantation" and broadly covers diseases, including blood cancers and genetic disorders, for which the procedure is performed.
"The granting of both orphan drug and Fast Track designations for ProTmune validates the product candidate's unique therapeutic potential to address life-threatening complications and improve the curative potential of allogeneic HCT," said Scott Wolchko, president and chief executive officer of Fate Therapeutics. "Graft-versus-host disease is a significant cause of morbidity and mortality in patients undergoing allogeneic HCT and there are no FDA-approved therapies to prevent its occurrence. Through our development of ProTmune, we seek to transform the allogeneic HCT paradigm by providing immunocompromised patients a therapeutically-optimized donor graft containing immune cells with reduced alloreactivity and enhanced infection-fighting and anti-tumor properties."
Graft-versus-host disease (GvHD) is a severe immunological complication that arises when newly-transplanted donor immune cells attack the patient's tissues and organs, resulting in a potentially fatal immune system reaction. Despite the use of protocols to prevent its occurrence, up to 50 percent of patients experience GvHD. Additionally, GvHD is treated systemically with immunosuppressive agents, with only about half of patients responding to treatment. The systemic use of these agents increases a patient's risk of severe infection from bacteria, viruses and fungi as well as cancer relapse, further compromising the curative potential of allogeneic HCT.
The FDA provides orphan designation to drugs and biologics which are intended for the safe and effective treatment, diagnosis or prevention of rare diseases and disorders that affect fewer than 200,000 people in the United States. Orphan drug designation would provide seven years of market exclusivity in the United States, with certain exceptions, if market approval is granted for ProTmune. Orphan designation also qualifies a company for various development incentives, including tax credits for qualified clinical testing and a waiver of PDUFA filing fees.
ProTmune is an investigational programmed cellular immunotherapy undergoing clinical development for the prevention of acute GvHD and cytomegalovirus (CMV) infection in patients undergoing allogeneic HCT. The cell therapy is produced by modulating a donor-sourced, mobilized peripheral blood graft ex vivo with two small molecules (FT1050 and FT4145) to enhance the biological properties and therapeutic function of the graft's immune cells. The programmed mobilized peripheral blood graft is administered to a patient as a one-time intravenous infusion.