AstraZeneca, a global, innovation-driven biopharmaceutical business, announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation for the MEK inhibitor selumetinib, for the treatment of uveal melanoma.
Uveal melanoma is a cancer (melanoma) of the eye involving the iris, ciliary body, or choroid (collectively referred to as the uvea). Tumors arise from the pigment cells (melanocytes) that reside within the uvea giving color to the eye. These melanocytes are distinct from the retinal pigment epithelium cells underlying the retina that do not form melanomas. It is the most common primary intraocular malignancy in adults and comprises 5% of all melanomas.
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 plans for selumetinib in uveal melanoma. The Orphan Drug Designation programme provides orphan status to drugs and biologics, which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the US.
Selumetinib is an oral small-molecule MEK inhibitor invented by Array and licensed to AstraZeneca. MEK is a key protein kinase in the RAS/RAF/MEK/ERK pathway, which regulates several key cellular activities including proliferation, differentiation, migration, survival and angiogenesis. Data from a phase III study evaluating selumetinib in combination with chemotherapy in patients with first-line metastatic uveal melanoma is expected to be available later this year. In addition to uveal melanoma, selumetinib is being investigated in phase III studies in KRAS mutation positive lung cancer and thyroid cancer and in phase II in children with neurofibromatosis Type 1.
Initial data from a combination study of selumetinib with other AstraZeneca pipeline molecules including AZD9291 (T790M-directed EGFR inhibitor) and MEDI4736 (anti-PD-L1) in non-small cell lung cancer will be presented at the American Society of Clinical Oncology (ASCO) annual meeting 2015.
Subscribe to PharmaTutor News Alerts by Email >>
