Keryx Biopharmaceuticals Receives Orphan Drug Designation for KRX-0401 (Perifosine) for the Treatment of Neuroblastoma
Keryx Biopharmaceuticals (MM) (NASDAQ:KERX)
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Today : Wednesday 14 July 2010
Keryx Biopharmaceuticals, Inc. (Nasdaq: KERX) today announced that KRX-0401 (perifosine) has received Orphan Drug designation from the U.S. Food and Drug Administration (FDA) for the treatment of neuroblastoma, a cancer of the nervous system affecting mostly children and infants for which there are no FDA approved therapies.
Phase 1 data of perifosine in recurrent pediatric solid tumors, including neuroblastoma, was presented last month in the pediatric solid tumor poster discussion session held at the 46th Annual Meeting of the American Society of Clinical Oncology (ASCO). Investigators from the Memorial Sloan-Kettering Cancer Center concluded that perifosine was demonstrated to be safe and well tolerated in children with advanced solid tumors and that perifosine may have antitumor clinical activity as a single agent in neuroblastoma. Additionally, in a preclinical study recently published in the Journal of the National Cancer Institute, perifosine showed a statistically significant reduction in neuroblastoma cell survival, slowed or regressed tumor growth, and increased survival in mice bearing neuroblastoma tumors. A decreased level of activated Akt was also observed in perifosine-treated neuroblastoma cells and xenograft tumors.
Ron Bentsur, Chief Executive Officer of Keryx, commented, "The Orphan Drug designation is an important component of our development plan for perifosine in neuroblastoma, an indication where no FDA approved therapies currently exist." Mr. Bentsur continued, "We are currently exploring next steps for the development of perifosine in this indication which we hope, ultimately, could provide a new treatment option for children and infants suffering with neuroblastoma."
Perifosine is currently in Phase 3 clinical development for refractory advanced colorectal cancer and multiple myeloma, both of these Phase 3 programs being conducted under Special Protocol Assessment (SPA) agreements with the FDA with Fast Track designations obtained for both indications. Perifosine is also in Phase 1 and 2 clinical development for several other tumor types.
KRX-0401 (perifosine) is in-licensed by Keryx from Aeterna Zentaris, Inc. in the United States, Canada and Mexico.
About Orphan Drug Designation
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.
About Neuroblastoma
