AstraZeneca announce orphan drug designation and progression-free survival data for oncology products

Anglo-Swedish drug major AstraZeneca (LSE: AZN) has been granted orphan drug designation by the US Food and Drug Administration for selumetinib, and has announced updated progression-free survival data for AZD9291.

The MEK inhibitor selumetinib has been granted orphan drug designation for the treatment of uveal melanoma, a rare disease in which cancer cells form in the tissues of the eye. It was originally licensed from Array BioPharma, and inhibits the MEK pathway in cancer cells to prevent the tumor from growing. Data from a Phase III study evaluating it in combination with chemotherapy in patients with first-line metastatic uveal melanoma is expected to be available later this year.

Antoine Yver, head of oncology, global medicines development at AstraZeneca, said: “Uveal melanoma is a rare and devastating disease for which there are currently no effective treatment options once it spreads beyond the tissues of the eye. Selumetinib could potentially become the first effective treatment for these patients. The Orphan Drug Designation is an important regulatory advancement as we further our development plans for selumetinib in uveal melanoma.”