NEW YORK The Food and Drug Administration has given special designation to an investigational treatment for a cancer of the nervous system that affects children.
Keryx Biopharmaceuticals announced Wednesday that it received orphan drug designation for KRX-0401 (perifosine), a treatment for neuroblastoma. No FDA-approved treatments exist for the disease.
The FDA gives orphan drug designation to investigational treatments for rare illnesses or those affecting fewer than 200,000 patients in the United States. The designation entitles the drug maker to seven years’ market exclusivity following approval, tax benefits and exemption from certain application filing fees.