Medullary Thyroid Cancer (MTC) is a form of cancer that originates from the parafollicular cells (C cells), which produce the hormone calciton. There are majorly two forms of MTC, one is called familial MTC caused due to genetic susceptibility and another one is called sporadic, caused by itself. AstraZeneca got approval from FDA for its orphan drug called Vandetanib for treatment of advanced MTC. The drug is approved for people, whose MTC cannot be removed by surgery or has spread to other parts of the body.
What is AstraZeneca’s Vandetanib is indicated for?
The FDA approved Vandetanib for
- the treatment of symptomatic or progressive medullary thyroid cancer in patients with non-operable locally advanced or metastatic disease.
- If patient has indolent or slowly progressing disease, vandetanib is required to be evaluated because of the treatment-related risks.
How do clinical results of Vandetanib look like?
AstraZeneca has been working on this drug from quite a while and the phase III trail named ZETA showed that:
- Risk for MTC progression reduced by 65% for patients on Vandetanib in comparison with those who received placebo.
- Median progression-free survival...
or PFS was at least 22.6 months in the vandetanib arm, compared with 16.4 months in the placebo arm.
- QT prolongation was reported in 14% of patients in the vandetanib arm and 1% of patients in the placebo arm.
FDA is still not sure if Vandetanib will increase overall survival rate as no significant overall survival difference was noted.
What side effects have been reported for Vandetanib?
Side effects include diarrhea, rash and nausea. Vandetanib also can affect electrical activity of the heart and cause irregular heartbeats that can lead to death. It has been approved under restrictions which require doctors and patients to be informed of the risks.
AstraZeneca acknowledged that Vandetanib would be dispensed exclusively through the pharmacy business unit of Biologics, Inc.