This article is more than 5 years old. Information may no longer be current.
FDA grants priority review to defibrotide for hepatic veno-occlusive disease
Click Here to Manage Email Alerts
The FDA granted priority review to defibrotide, an investigational agent in development for treatment of patients with hepatic veno-occlusive disease who have evidence of multiorgan dysfunction after hematopoietic stem cell transplantation.
The FDA previously granted orphan drug status and fast track designation to defibrotide (Jazz Pharmaceuticals).
Hepatic veno-occlusive disease is a rare but life-threatening complication of HSCT, and the mortality rate for veno-occlusive disease with multiorgan dysfunction exceeds 80%.
“The FDA’s acceptance for filing and priority review status of the new drug application for defibrotide is an important milestone for Jazz and reflects our commitment to bringing meaningful medicines to patients who have significant unmet needs,” Karen Smith, MD, PhD, global head of research and development and chief medical officer of Jazz Pharmaceuticals, said in a press release. “We look forward to continuing to work closely with the FDA to obtain approval for defibrotide for patients with hepatic veno-occlusive disease with evidence of multiorgan dysfunction in the U.S. as quickly as possible, as there are no other approved therapies for treating this rare, often fatal complication of HSCT.”
The new drug application is based on safety and efficacy data from three studies that evaluated defibrotide as a treatment for veno-occlusive disease with multiorgan dysfunction after HSCT, as well as a retrospective review of registry data from the Center for International Blood and Marrow Transplant Research.
The FDA is expected to make a decision on the new drug application by March 31, 2016.