The orphan drug designation encompasses all subsets of multiple myeloma and Waldenstrom’s macroglobulinemia.
The orphan drug act provides the drug developer with several financial benefits and incentives related to the orphan drug, including tax credits for clinical research costs, ability to apply for annual grant funding, clinical research trial design assistance, waiver of Prescription Drug User Fee Act (PDUFA) filing fees, and a seven-year period of US marketing exclusivity if the drug is the first of its type approved for the specified indication.
Susan Molineaux, president and CEO of Proteolix, said: “To date we have been encouraged by carfilzomib’s early-stage clinical results in multiple myeloma, and we continue to believe it has the potential to offer cancer patients a new and effective treatment for their disease.”