AstraZeneca gets Zactima Orphan Drug designation

AstraZeneca’s ZD6474 (Zactima) cancer drug has been granted Orphan Drug designation by the US Food and Drug Administration (FDA).

Also known as Zactima, ZD6474 trials will now begin in the treatment of patients with follicular, medullary, anaplastic, and locally advanced and metastatic papillary thyroid cancer.

The Orphan Drug Act designation was created in 1982 and provides incentives for sponsors to develop products for rare diseases, specifically, those that affect less than 200,000 people in the United States.

Medullary thyroid cancer comprises two-to-three per cent of the 25,000 new cases of thyroid cancer in the US each year.

ZD6474 must still go through the FDA marketing approval process, like any other drug or biologic, that evaluates for safety and efficacy.

ZD6474 is currently being developed in a Phase II clinical trial in medullary thyroid cancer and Phase III clinical trials in advanced non-small cell lung cancer are scheduled to start in the next few months.

The drug works by selectively targeting key cell signalling pathways involved in tumour growth and spread including VEGF (vascular endothelial growth factor) receptor signalling and EGF (epidermal growth factor) receptor signalling.

ZD6474 also inhibits RET kinase, which drives the growth and survival of certain tumours, and is believed to be an important pathway in medullary thyroid cancer.

In addition to the US, AstraZeneca has applied for Orphan Drug designation for ZD6474 in medullary thyroid cancer in Europe.

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