AstraZeneca Wins Orphan Status for Thyroid Cancer Drug

FDA has granted AstraZeneca an orphan drug designation for the investigational MEK 1/2 inhibitor, selumetinib, indicated for adjuvant treatment of patients with Stage III or IV differentiated thyroid cancer. The condition is diagnosed in about 60,000 people in the U.S. each year, the company says. Some patients do not benefit from the current available treatment with radioactive iodine (RAI) because they do not express sufficient sodium/iodine symporter (NIS), which is important for the iodine’s uptake into thyroid cells.“ Selumetinib is being tested for its ability to increase expression of NIS with the potential to add a treatment option for patients who do not respond well to RAI,” it says. A recent Phase s study with selumetinib in patients with advanced thyroid cancer, “clinically meaningful increases in iodine uptake and retention were seen in patients with thyroid cancer that was refractory to RAI,” AstraZeneca adds.