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news.The US Food and Drug Administration (FDA) has granted AstraZeneca an orphan drug designation (ODD) for the MEK inhibitor selumetinib, to treat uveal melanoma, a rare disease.
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In uveal melanoma, cancer cells form in the tissues of the eye and it is the most common primary intraocular malignancy in adults and comprises 5% of all melanomas.
Selumetinib, which is originally licensed from Array BioPharma, inhibits the MEK pathway in cancer cells to prevent growth of the tumor.
AstraZeneca Oncology, Global Medicines Development head Antoine Yver 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."
Currently, selumetinib is being evaluated in combination with chemotherapy in patients with first-line metastatic uveal melanoma in a Phase III trial and its data is expected to be available later in 2015.
Apart from uveal melanoma, selumetinib is being investigated in Phase III trials in KRAS mutation positive lung cancer and thyroid cancer and in Phase II in children with neurofibromatosis Type 1.
At the American Society of Clinical Oncology (ASCO) annual meeting 2015, the company will present initial data from a combination study of selumetinib with its other pipeline molecules including AZD9291 (T790M-directed EGFR inhibitor) and MEDI4736 (anti-PD-L1) in non-small cell lung cancer (NSCLC).
Image: AstraZeneca headquarters in London, UK. Photo: courtesy of AstraZeneca plc.