NEW YORK, Sept. 16 /PRNewswire/ -- Keryx Biopharmaceuticals, Inc. today announced that KRX-0401 (perifosine) has received Orphan-Drug designation from the U.S. Food and Drug Administration (FDA) for the treatment of multiple myeloma. In August, the Company announced that it had reached an agreement with the FDA regarding a Special Protocol Assessment (SPA) on the design of a Phase 3 trial in relapsed/refractory multiple myeloma and that the study is expected to start by year-end.
KRX-0401 (perifosine) is in-licensed by Keryx from Aeterna Zentaris, Inc. in the United States, Canada and Mexico.
Orphan-drug designation is granted by the FDA Office of Orphan Drug Products to novel drugs or biologics that treat a rare disease or condition affecting fewer than 200,000 patients in the U.S. The designation provides the drug developer with a seven-year period of U.S. marketing exclusivity if the drug is the first of its type approved for the specified indication or if it demonstrates superior safety, efficacy, or a major contribution to patient care versus another drug of its type previously granted the designation for the same indication, as well as with tax credits for clinical research costs, the ability to apply for annual grant funding, clinical research trial design assistance and waiver of Prescription Drug User Fee Act (PDUFA) filing fees.
The Special Protocol Assessment (SPA) process is a procedure by which the FDA provides official evaluation and written guidance on the design and size of proposed protocols that are intended to form the basis for a new drug application.
About Multiple Myeloma
About Keryx Biopharmaceuticals
Cautionary Statement
Keryx Biopharmaceuticals
