US FDA and EMEA accepts AstraZeneca's filing for vandetanib; SOBI gains orphan status for hemophilia A therapy in EU

Anglo-Swedish drug major AstraZeneca (LSE: AZN) announced this morning that the US Food and Drug Administration and the European Medicines Agency (EMEA) have accepted regulatory submissions for review of the investigational drug vandetanib in the treatment of patients with advanced medullary thyroid cancer (MTC). The FDA also granted priority review status for the new drug application and set a Prescription Drug User Fee Act (PDUFA) action date of January 7, 2011, the company noted.

The submissions are supported by the results from the ZETA study evaluating the safety and efficacy of vandetanib compared to placebo in patients with advanced MTC. AstraZeneca is consulting with regulatory authorities on a proposed trade name.

ZETA was a Phase III, double-blind, placebo-controlled study that randomized 331 patients with advanced MTC to oral once-daily vandetanib 300mg or placebo. The study results were presented in June at the American Society of Clinical Oncology annual meeting in Chicago (The Pharma Letter June 8). Results from ZETA showed that treatment with vandetanib significantly extended progression-free survival, the primary endpoint of the study, in patients with advanced MTC. In ZETA, vandetanib demonstrated a 54% reduction in the rate of progression compared to placebo (HR=0.46, p=0.0001). The objective response rate (ORR), a secondary endpoint, was 45% versus 13% across the two groups (p<0.0001).