Ariad cancer drug given orphan drug status

The novel mTOR inhibitor, AP23573, is designed to fight cancers of the connective tissues including bones, muscles, fat, cartilage, and joints. These sarcomas usually belong to one of two groups; bone tumors or soft tissue sarcomas, both of which presently represent an unmet medical need. This factor, together with the relative rarity of the disease, has led to the orphan drug designation.

Orphan drug designation, which is intended to facilitate drug development, provides substantial potential benefits to the sponsor, including funding for certain clinical studies, study-design assistance, and up to seven years of market exclusivity for the product upon regulatory approval.

The small molecule drug, AP23573, works to starve cancer cells and shrink 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 (VEGF) in tumor and endothelial cells.

AP23573 is currently in phase I and II clinical trials in patients with solid tumors and hematologic cancers. The product has been designated both as a fast-track product and an orphan drug by the FDA for the treatment of soft-tissue and bone sarcomas.