The U.S. Food and Drug Administration has given priority review designations to the following:
* Defibrotide for the treatment of patients with hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome or SOS) with evidence of multi-organ dysfunction following hematopoietic stem-cell transplantation (HSCT) and;
* Halaven (eribulin mesylate; also known as eribulin) for the treatment of patients with inoperable soft tissue sarcoma (leiomyosarcoma and liposarcoma) who have received prior chemotherapy for advanced or metastatic disease.
The FDA's priority review designation shortens the time to complete a drug's review and aims to deliver a decision on marketing approval designation for drugs that may offer major advances in treatment or provide a treatment where no adequate therapy exists within six months under the Prescription Drug User Fee Act (PDUFA).
Defibrotide
Defibrotide, marketed by Jazz Pharmaceuticals, is an oligonucleotide with a mechanism of action that encompasses both restoration of the thrombo-fibrinolytic balance and endothelial cell protection. The FDA action date for defibrotide for this indication is March 31.
The product had previously been granted both Orphan Drug and Fast Track status. In addition, defibrotide is being made available as an investigational new drug (IND) free of charge through an expanded access Treatment Protocol, which is currently enrolling patients diagnosed with VOD in the United States.
Approval for defibrotide will be based on the safety and efficacy data from three clinical studies of patients with hepatic VOD with multi-organ dysfunction following HSCT, as well as a retrospective review of registry data from the Center for International Blood and Marrow Transplant Research. The safety database includes more than 900 patients exposed to deibrotide in the clinical development program for the treatment of hepatic VOD.
Halaven
Halaven, marketed by Eisai, is a microtubule dynamics inhibitor that exerts its effects via a tubulin-based antimitotic mechanism ultimately leading to apoptotic cell death after prolonged and irreversible mitotic blockage. The FDA action date for Halaven for this indication is January 29.
Halaven has previously been approved for the treatment of patients with metastatic breast cancer who have received at least two chemotherapeutic regimens for the treatment of metastatic disease, and whose prior therapy included an anthracycline and a taxane in either the adjuvant or metastatic setting.
Halaven's approval for this indication in sarcoma will be based on data from a Phase III clinical trial of 452 patients with high- or medium-grade liposarcoma or leiomyosarcoma who had previously undergone at least two anti-cancer regimens who were assigned to receive either Halaven or dacarbazine.
The data-which were presented at this year's American Society of Clinical Oncology Annual Meeting-showed that patients receiving Halaven had a median overall survival of 13.5 months compared with 11.5 months for patients receiving dacarbazine (OT 6/25/15 issue).