Keryx Biopharmaceuticals has received orphan drug designation for KRX-0401 (perifosine), a drug to treat neuroblastoma, by the US Food and Drug Administration (FDA).
Neuroblastoma is a cancer of the nervous system that affects children and infants, for which there is currently no FDA-approved therapy.
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Phase I trial of perifosine showed that the drug was well-tolerated by children and infants with advanced solid tumours, and anti-tumour clinical activity.
In a preclinical study, perifosine slowed down tumour growth and increased the survival of mice.
Orphan drug designation is granted by the FDA for drugs which treat rare diseases affecting less than 200,000 people in the US.
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