Selumetinib granted US orphan drug designation for adjuvant treatment of differentiated thyroid cancer

12 May 2016

Anglo-Swedish pharma major AstraZeneca (LSE: AZN) received orphan drug designation from the US Food and Drug Administration for its MEK 1/2 inhibitor, selumetinib (AZD6244, ARRY-142886) for adjuvant treatment of patients with stage III or IV differentiated thyroid cancer (DTC).

DTC is diagnosed in around 60,000 Americans annually, and radioactive iodine (RAI) is recommended for those with known or suspected metastases at diagnosis and those at high risk of recurrence.

A small number of patients do not benefit from currently available treatment with RAI because they do not express sufficient sodium/iodine symporter (NIS), which is important for RAI uptake into thyroid cells.

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