The FDA approved Jazz Pharmaceuticals' drug Xywav Aug. 12 to treat a rare sleep condition, a sedative that has faced controversy and bypassed the FDA's advisory panel, The New York Times reported.
One of the active ingredients in Xywav is a form of gamma-hydroxybutyrate, or GHB. An older iteration of the drug called Xyrem, which contains much more sodium than Xywav, has been approved since 2002 as a treatment for narcolepsy, but tightly regulated due to its potential for addiction, according to the Times. Xywav was approved in July 2020 to treat narcolepsy.
Lewis Nelson, MD, director of medical toxicology at Rutgers New Jersey Medical School in Newark, told the Times that GHB has "serious safety concerns, both in terms of its abuse liability and its addictive potential."
The FDA has now approved it to treat a rare sleep condition called idiopathic hypersomnia, a condition in which people sleep nine or more hours a day but never feel rested. It is thought to work by giving patients more restorative sleep at night and is the first approved treatment for the condition, the Times reported.
But some experts told the Times there is little publicly available evidence that the drug works against the sleep condition, and it can have severe side effects, including breathing problems, anxiety, depression, sleepwalking, hallucinations and suicidal thoughts. The drug also acts so quickly that the label advises patients to take it while in bed.
The FDA granted the approval for treatment of idiopathic hypersomnia without asking its advisory panel to weigh in. Clinical trials testing the drug for the condition included just 115 people who were given the drug for 12 to 16 weeks.
GHB has been linked to sexual assaults and deaths, and in 2000, Congress passed a law that made it illegal, but allowed for future medical uses, according to the Times.
As of June 30, the FDA has recorded more than 27,000 serious adverse events in patients taking Xywav or Xyrem. The serious adverse events include 753 deaths, the Times reported.
After Xyrem was approved to treat narcolepsy, Orphan Medical —the company that developed Xyrem and was later acquired by Jazz — was ordered to pay $20 million in fines for inappropriately marketing the drug for unapproved conditions, including insomnia, depression and fibromyalgia, the Times reported.
Nightly treatments of the drugs cost about $100,000 per year. Jazz pharmaceuticals made more than $1.7 billion last year selling Xyrem and Xywav to about 15,000 people, most of them with narcolepsy, according to the Times.
It is estimated that 40,000 people in the U.S. have been diagnosed with idiopathic hypersomnia, but Dr. Nelson told the Times that many more people with daytime drowsiness may be diagnosed with the condition now that there's an FDA-approved treatment.
Read the full article here.
Editor's note: This article was updated Aug. 18 at 5:30 p.m. CT to reflect corrections and clarifications from Jazz Pharmaceuticals.