mTOR Inhibitor Designated as Orphan Drug

Filed in archive Drugs, Vaccines and Therapeutics on August 23, 2005

ARIAD Pharmaceuticals, Inc. announced last week that AP23573, its novel mTOR inhibitor, has been designated an orphan drug by the United States Food and Drug Administration (FDA) for the treatment of both soft-tissue and bone sarcomas.

Designated as an "orphan drug", it will enjoy the following perks:

• funding for certain clinical studies


• study-design assistance


• waiver of Prescription Drug User Fee Act (PDUFA) fees


• tax credits


• up to seven years of market exclusivity for the product upon regulatory approval


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  "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, M.D., chairman and chief executive officer of ARIAD. "We are working with key regulatory agencies to advance AP23573 toward its initial registration trial and subsequent regulatory submissions for marketing authorization in these devastating cancers."

  
  
  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. AP23573 is targeted at the treatment of bone and soft-tissue sarcomas and is in Phase 2 trials. Read more here.