Eye Cancer Drug Granted Orphan Designation

The FDA has granted Orphan Drug Designation to AstraZeneca’s selumetinib for the treatment of a rare disease in which cancer cells form in eye tissues.

“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,” said Antoine Yver, head of oncology in Global Medicines Development at AstraZeneca, in a press release. “Selumetinib could potentially become the first effective treatment for these patients.”

Uveal melanoma comprises 5% of all melanomas and is the most common primary intraocular malignancy in adults. Selumetinib helps treat the disease by inhibiting the MEK pathway in cancer cells to prevent a tumor from growing.

Phase 3 study data on the drug in combination with chemotherapy is expected later this year. In addition to being evaluated in patients with first-line metastatic uveal melanoma, selumetinib is currently being investigated in KRAS mutation lung cancer and thyroid cancer, as well as children with neurofibromatosis Type 1.

Initial data from a study examining selumetinib in combination with AstraZeneca’s other pipeline treatments in non-small cell lung cancer will be presented at the American Society of Clinical Oncology 2015 annual meeting.