Ariad cancer agent wins FDA's orphan drug status

The FDA has given orphan drug status to Ariad's experimental cancer treatment, AP23573. AP23573 starves cancer cells and shrinks tumors by inhibiting the critical cell-signaling protein, mTOR, which regulates the response of tumor cells to nutrients and growth factors, and controls tumor blood supply and angiogenesis through effects on Vascular Endothelial Growth Factor in tumor and endothelial cells.

"In the absence of any effective treatments for advanced soft-tissue sarcomas or refractory bone sarcomas that have spread, the orphan drug designations for AP23573 provided by the FDA should help us address this unmet medical need more effectively," said Harvey J. Berger, MD, chairman and CEO of Ariad.

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