AstraZeneca recently announced that selumetinib has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA).
Selumetinib is a MEK 1/2 inhibitor being investigated for treatment of stage III or IV differentiated thyroid cancer (DTC).
The FDA's Office of Orphan Products Development (OOPD) provides grants to help companies develop new treatments and medications for rare diseases and conditions. The program was started in 1983. Funding for clinical trials lasts three to four years, giving companies time to fully investigate the effectiveness of the products.
Approximately 60,000 people are diagnosed annually with DTC. While radioactive iodine (RAI) is the standard treatment for patients with metastases or at risk of recurrence, some patients do not respond to RAI. If the body does not have enough sodium/iodine symporter (NIS), the thyroid cells do not absorb the RAI.
“Uptake of radioactive iodine is crucial for patients with thyroid cancer where no other therapies have proven beneficial," AstraZeneka Global Medicines Development EVP and Chief Medical Officer Sean Bohen said. "Selumetinib could significantly enhance currently available treatment options for these patients. The Orphan Drug Designation is an important achievement as we advance our development plans for this potential treatment in differentiated thyroid cancer.”
Selumetinib facilitates the production of NIS, making it easier for the thyroid cancer cells to take up the RAI. It also inhibits the MEK pathway in cancer cells, which helps prevent tumor growth. The Phase III ASTRA clinical trial is testing selumetinib in patients with a high risk of recurrence. The Phase II trial showed positive results in patients with advanced thyroid cancer.