Shares of Keryx Biopharmaceuticals, Inc. (Nasdaq: KERX) climbed as much as 17 percent in morning trading Wednesday after the after the company announced its experimental childhood cancer drug, KRX-0401 (perifosine), had received Orphan Drug designation from U.S. Food and Drug Administration (FDA).
Commenting on receiving Orphan Drug designation, Ron Bentsur, Chief Executive Officer of Keryx stated, “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.
The FDA grants Orphan Drug status to new drugs that treat a rare disease afflicting fewer than 200,000 patients in the U.S. The benefits of Orphan Drug designation include seven-year marketing exclusivity if the drug is the first of its kind approved, 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.
KRX-0401 is being developed for the treatment of neuroblastoma, a cancer of the nervous system affecting mostly children and infants. Although it is a rare form of cancer, neuroblastoma accounts for approximately 7 percent of all cancer deaths among children.
Clinical Phase 1 data 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, the Nationaly Cancer Institute recently published a preclinical study that found perifosine showed a statistically significant reduction in neuroblastoma cell survival, slowed or regressed tumor growth, and increased survival in mice bearing neuroblastoma tumors.