(Reuters) - U.S. regulators have inappropriately expanded the approved use of Vanda Pharmaceuticals Inc’s sleep disorder drug Hetlioz beyond its original indication for use in blind people, the consumer watchdog Public Citizen said on Thursday.
The U.S. Food and Drug Administration approved the drug in January 2014 to treat Non-24-hour sleep-wake disorder in blind patients. But that is not what was put on the drug’s label. The label says the drug is indicated for use in patients with Non-24, not blind patients with Non-24.
The discrepancy opens the door for the drug to be used in a wide range of sleep disorders in patients who are not blind, Public Citizen said. Companies are not allowed to market drugs for conditions for which they have not been approved, though physicians are allowed to prescribe them for “off-label” use.
The FDA appears not to have asked the company to change the Hetlioz label. Instead it issued a second approval letter in October last year noting that its initial approval contained an error in the indications for use section.
The revised approval notes the drug is approved for Non-24 in general, while noting the condition is experienced “almost exclusively” by blind people.
Public Citizen has filed a petition with the agency requesting that the label be revised to reflect the use for which the drug was tested and approved. It also requests that the label include information related to carcinogenic and toxicity effects seen in animal studies.
The FDA said it would review the petition and respond directly to Public Citizen. Vanda declined to comment.
Hetlioz was approved under the FDA’s orphan drug pathway, which provides tax and other incentives for drug companies to develop treatments for rare diseases that affect fewer than 200,000 people. Non-24 disorder is estimated by the FDA to affect 100,000 people who are blind.
The drug generated sales of $12.8 million in 2014.
Public Citizen said the FDA is violating legal standards for drug approval, which require companies to provide scientific evidence from clinical trials proving their products are safe and effective.
Sandy Walsh, an FDA spokeswoman, said there are circumstances in which the agency can approve a drug for an unstudied group, for example under a rule allowing approval based on animal data. She did not say agency had used this provision to authorize use of Hetlioz in patients who are not blind.
Reporting by Toni Clarke in Washington; Editing by Lisa Shumaker