The FDA has approved the orphan drug vandetanib (AstraZeneca), a kinase inhibitor, for the treatment of symptomatic or progressive medullary thyroid cancer in patients with unresectable locally advanced or metastatic disease. This approval was based on data from the ZETA study, a Phase 3, randomized, double-blind, placebo-controlled international study of 331 patients with unresectable locally advanced or metastatic medullary thyroid cancer. Patients who received vandetanib showed a statistically significant improvement in progression-free survival (PFS) when compared to those randomized to placebo (HR=0.35 [0.24–0.53]; p<0.0001). This difference reflects a 65% reduction in risk for disease progression. Median progression-free survival was at least 22.6 months in the vandetanib arm compared with 16.4 months in the placebo arm.
No trade name has been established for vandetanib at this time.
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