An expert FDA panel has voted 10-3 to support Actelion's Zavesca for a rare illness called Niemann-Pick type C disease. Those who voted in favor of the drug maintain that even though Zavesca did not meet the main endpoint of effectiveness in trials, patients could still benefit from it. There are no other approved drugs for NP-C. "The risk from the drug is not devastating, but the disease is devastating. I think patients deserve an opportunity to see if they can benefit," said Katherine Flegal, a senior research scientist at the Centers for Disease Control and Prevention, as quoted by Reuters.
An expanded approval of Zavesca is important to Actelion, which is trying to wean itself off its dependence on Tracleer, its blockbuster drug that brought in $1 billion last year. Zavesca is already approved for Gaucher disease, and while physicians can prescribe it off-label for NP-C, many insurers balk at the $159,000 price tag. The FDA's final decision is expected on March 10.
- read the Reuters report