On the strength of the results from an early stage clinical trial, Keryx Biopharmaceuticals Inc. has gained orphan drug status from the US FDA for its experimental cancer drug perifosine in the treatment of people suffering from neuroblastoma.
Neuroblastoma is an extremely rare cancer that is generally found in children under the age of 5 and rarely found in anyone over age 10.
According to the American Cancer Society, of the 650 or so cases of neuroblastoma diagnosed each year, about a third begin in the adrenal glands, a third begin in the sympathetic nerve ganglia of the abdomen, and the remainder begin "in sympathetic ganglia near the spine in the chest or neck or in the pelvis."
There are currently no FDA approved treatments that specifically address neuroblastoma.
The FDA has previously granted perifosine orphan drug status for the treatment of colorectal cancer.
DRUG(S)
Perifosine
CANCER TYPE(S)
Neuroblastoma
By Ross Bonander
SOURCE(S)
- Associated Press
- National Cancer Institute
- American Cancer Society
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