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Keryx Biopharmaceuticals, Inc. (KERX) Receives Orphan Drug Status for Perifosine

Keryx Biopharmaceuticals, Inc. reported that one of its drug candidates used to treat cancer received a major boost towards development through the regulatory actions of the U.S. government.

Keryx Biopharmaceuticals, Inc. said that KRX-0401, also known as Perifosine, was designated an Orphan Drug by the United States Food and Drug Administration. This designation is available only for drugs used to treat illnesses affecting less than 200,000 individuals, and gives the company a seven year exclusive marketing term in the United States.

Keryx Biopharmaceuticals, Inc. is developing Perifosine as a therapy for the treatment of neuroblastoma, a cancer that affects the nervous system of infants and children. There is currently no known treatment for the cancer.

Ron Bentsur, the CEO of Keryx Biopharmaceuticals, Inc. said, “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. 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.”

Keryx Biopharmaceuticals, Inc. recently reported Phase I data on Perifosine. Doctors at the Memorial Sloan-Kettering Cancer Center concluded that the drug was “safe and well tolerated” and “may have antitumor clinical activity.” Perifosine has also been shown to decrease tumor growth in laboratory animals with neuroblastoma.

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