Unfortunately, in mid-March the FDA advised pharmaceutical company Actelion, which markets Zavesca that they would not yet approve Zavesca for NPC; that more definitive proof is required by the law. We are hopeful that further study efforts will lead to future approval.
The consequence of the FDA’s decision is that too many children with NPC will not be on Zavesca. It is extremely expensive as with all treatments for rare diseases, and while perhaps 25 percent of NPC
patients, including Dana and Andrew, are getting Zavesca “off label” (not specifically authorized for NPC) through their insurance, far too many children have to go without it.