17 Oncology Times
FDA Priority Review Status for Defibrotide and Halaven
he U.S. Food and Drug the Prescription Drug User Fee Act Approval for defibrotide will be Halaven has previously been TAdministration has given pri- (PDUFA). based on the safety and efficacy approved for the treatment of pa- ority review designations to the data from three clinical studies of tients with metastatic breast can- following: patients with hepatic VOD with cer who have received at least two
• Defibrotide for the treatment Defibrotide multi-organ dysfunction following chemotherapeutic regimens for the •
of patients with hepatic veno-occlu- Defibrotide, marketed by Jazz HSCT, as well as a retrospective re- treatment of metastatic disease, October 25, 2015 sive disease (VOD), also known as Pharmaceuticals, is an oligonucle- view of registry data from the Center and whose prior therapy included sinusoidal obstruction syndrome or otide with a mechanism of action for International Blood and Marrow an anthracycline and a taxane in SOS) with evidence of multi-organ that encompasses both restoration of Transplant Research. The safety data- either the adjuvant or metastatic dysfunction following hematopoi- the thrombo-fibrinolytic balance and base includes more than 900 patients setting. etic stem-cell transplantation (HSCT) endothelial cell protection. The FDA exposed to deibrotide in the clinical Halaven’s approval for this in- and; action date for defibrotide for this in- development program for the treat- dication in sarcoma will be based • Halaven (eribulin mesylate; also dication is March 31. ment of hepatic VOD. on data from a Phase III clinical
known as eribulin) for the treatment The prod- trial of 452 patients with high- or • oncology-times.com of patients with inoperable soft tis- uct had pre- medium-grade liposarcoma or sue sarcoma (leiomyosarcoma and viously been Halaven leiomyosarcoma who had pre- liposarcoma) who have received prior granted both Halaven, mar- viously undergone at least two chemotherapy for advanced or meta- Orphan Drug keted by Eisai, anti-cancer regimens who were as- static disease. and Fast Track is a microtu- signed to receive either Halaven or status. In ad- bule dynamics dacarbazine. The FDA’s priority review designa- dition, defib- inhibitor that The data—which were presented tion shortens the time to complete rotide is being exerts its ef- at this year’s American Society of a drug’s review and aims to deliver made available as an investigational fects via a tubulin-based antimitotic Clinical Oncology Annual Meeting— a decision on marketing approval new drug (IND) free of charge through mechanism ultimately leading to showed that patients receiving designation for drugs that may offer an expanded access Treatment apoptotic cell death after prolonged Halaven had a median overall survival major advances in treatment or pro- Protocol, which is currently enrolling and irreversible mitotic blockage. The of 13.5 months compared with 11.5 vide a treatment where no adequate patients diagnosed with VOD in the FDA action date for Halaven for this months for patients receiving dacar- O therapy exists within six months under United States. indication is January 29. bazine (OT 6/25/15 issue). T
AACR Survey Rally for Medical Continued from page 16 Research In conjunction with the re- • The epigenome-modifying lease of its report, AACR held agent panobinostat; a Congressional briefing and • The immunotherapies reception to celebrate the blinatumomab, dinutuximab, contributions of medical re- nivolumab, and pembrolizumab; search and highlight the need • The imaging agent techne- for Congress to support a tium 99m tilmanocept (for lym- strong, sustained funding phatic mapping in solid tumors); stream for cancer research. • The cancer screening test The Senate NIH Caucus, led Cologuard (for colorectal cancer); by Senators Dick Durbin and (D-IL) and Lindsey Graham • The preventive vaccine hu- (R-SC), was an honorary man papillomavirus 9-valent vac- sponsor. cine (types 6, 11, 16,18, 31,33,45, AACR also sponsored 52 and 58) for cervical, vulvar, a breakfast and Rally for vaginal, and anal cancers. Medical Research Hill Day featuring members of Congress, AACR CEO Foti, Four Immunotherapies and Sue Peschin, CEO of the The four immunotherapies approved precision medicine, noting that increas- Alliance for Aging Research, among by the FDA during the same period ingly, anticancer therapies are precisely many others. During the Hill Day, represent the largest number of im- targeted against factors responsible for hundreds converged on Capitol Hill munotherapeutic agents approved the cancer, thus reducing harm to nor- for meetings with House and Senate in a 12-month period since the first mal cells. staffers to make the case for “robust, AACR Cancer Progress Report was “Although precision medicine is sustained, and predictable” funding in- published in 2011, the new report not unique to the practice of oncology, creases for cancer research for 2016 and states—“highlighting how this pow- oncology is leading such efforts largely beyond (read more about it on page 40). erful form of cancer treatment has because of our immense knowledge AACR, the founding organizer, part- emerged as a key pillar of cancer care.” of the role of genetic mutations in the nered with some 300 organizations development and progression of can- across the country to spread the mes- cer,” the AACR document says. “As we sage about stronger support for bio- Precision Medicine look to the future, the pace of progress medical research; this was the third The report, which features the stories of in precision medicine will continue to such event that has been held in O 13 cancer patients, hails the promise of accelerate.” Washington. T