Citizen Petition Alleges FDA Incorrectly Expanded Sleep Disorder Drug's Indication

A citizens advocacy group is accusing the FDA of wrongly expanding the indication of Vanda Pharmaceuticals’ sleep disorder drug Hetlioz by labeling it for use in sighted people.  

The FDA approved Hetlioz (tasimelteon) on Jan. 31, 2014, granting it orphan drug status to treat totally blind patients with the rare sleep-wake disorder “Non-24” and no perception of light. Clinical studies of the drug were only conducted in this group of patients, whose body clocks aren’t able to sync with the 24-hour light-dark cycle.

However, the label the FDA issued for the product omitted the “in blind patients without light perception” part of the statement, therefore allowing Hetlioz to be used by anyone with Non-24 disorder, says Public Citizen in a petition filed with the agency.

Instead of fixing the error, the FDA compounded the mistake by issuing a second approval letter, saying the original letter was erroneous and the drug’s approved use was that on the incorrect label, Public Citizen contends. The group notes that the second letter was also dated Jan. 31, despite being issued sometime later.

The group notes that 26 FDA review documents signed by 42 agency officials explicitly state that Vanda was seeking approval for use only in blind patients, and the agency’s approval letter and press release confirmed that indication.

According to the group, an FDA medical officer who reviewed the product provided rationale for the change in an Oct. 1, 2014 addendum, saying trials had been conducted in sighted individuals with jet lag disorder and other models of circadian rhythm disruption, and that reports of sighted individuals with Non-24 had also been successfully treated with the drug. Public Citizen argues these limited observations are insufficient to support an expanded indication.

The group is urging the FDA to change the description of approved use back to the original indication. It also wants the agency to require Vanda to conduct a postmarket safety study of Hetlioz. — Kellen Owings