Keryx Biopharma says FDA granted orphan drug status to perifosine as neuroblastoma treatment

Keryx’s perifosine gets second orphan drug status

NEW YORK — Keryx Biopharmaceuticals Inc. said Wednesday that its experimental cancer drug perifosine received orphan drug status as a treatment for neuroblastoma, a cancer that affects the nervous system and mostly strikes young children.

The Food and Drug Administration grants orphan drug incentives to potential drugs that treat disease that affect less than 200,000 people in the U.S. Orphan drug status includes incentives during development and seven years of marketing exclusivity if the drug is the first to treat an illness, or if it works better than older therapies or significantly helps patients.

Keryx said neuroblastoma is the most common cancer in infants, with about 650 new cases each year. It said there are no approved treatments specifically for the disease and treatment options are limited.

The company recently reported results from an early stage clinical trial of perifosine as a treatment for neuroblastoma. The drug is also being tested against other cancers. Keryx is running late stage trials of perifosine as a treatment for advanced colorectal cancer and for multiple myeloma, a cancer of the blood.

Perifosine also has orphan drug status as a colorectal cancer treatment, and the FDA has said it will conduct a faster-than-normal review of the drug in both colorectal cancer and multiple myeloma.

Keryx has the rights to market perifosine in North America. Canadian drugmaker Aeterna Zentaris holds the rights in all other countries except South Korea.

Keryx stock jumped 55 cents Wednesday, or 15.5 percent, to $4.11. Shares of Aeterna Zentaris rose 10 cents, or 9.2 percent, to $1.19.