AstraZeneca today announced that the orphan drug vandetanib is now available to US patients for the treatment of medullary thyroid cancer that cannot be removed by surgery or that has spread to other parts of the body. Vandetanib was approved by the US Food and Drug Administration on April 6, 2011.
Vandetanib is a kinase inhibitor indicated for the treatment of symptomatic or progressive medullary thyroid cancer in patients with unresectable (non-operable) locally advanced or metastatic disease.
The use of vandetanib in patients with indolent, asymptomatic or slowly progressing disease should be carefully considered because of the treatment-related risks.
A Risk Evaluation and Mitigation Strategy (REMS) is required for vandetanib due to the risks of QT prolongation, Torsades de pointes, and sudden death. Only prescribers who are certified through the Vandetanib REMS Program, a restricted distribution program, will be able to prescribe vandetanib.
The FDA approved the kinase inhibitor based on the results of a 331- patient, randomized Phase 3 trial called the ZETA study. The study showed a 65% risk reduction in disease progression in the drug arm compared to placebo. The median progression-free survival on vandetanib was 22.6 months, compared to 16.4 months in the control arm. To date, it is still unknown whether vandetanib affects overall survival of patients with MTD.
“Vandetanib is the only medicine specifically approved for patients with this rare form of cancer,” said Lisa Schoenberg, Vice President of Specialty Care, AstraZeneca. “We believe vandetanib will be important for the community of patients and doctors who are fighting this disease.”
AstraZeneca is working with relevant authorities on a trade name and is currently referring to the treatment by its generic name, vandetanib.