This site is intended for health professionals only

Orphan status for child cancer drug


An experimental treatment which could help fight a cancer that attacks the nervous system of young children has been granted orphan drug status by the Food and Drug Administration.

Perifosine manufacturer Keryx Biopharmaceuticals said it was hopeful the product could help those with the condition, which is thought to affect around 650 infants each year, explaining that treatment options were currently limited because no drugs were approved for the disease.

Keryx added that the medication had the potential to fight other cancers, saying it was running late-stage trials for use in advanced colorectal cancer and multiple myeloma.

The orphan status, which is granted to potential drugs for diseases that affect less than 200,000 people, gives Keryx seven years of marketing exclusivity if the product is the first to treat an illness, or if it works better than older therapies or significantly helps patients.

Copyright Press Association 2010

Keryx Biopharmaceuticals

Latest Issue

Be in the know
Subscribe to Hospital Pharmacy Europe newsletter and magazine