A Food and Drug Administration (FDA) advisory panel on Friday significantly rejected the application of Jazz Pharmaceuticals for the approval of Xyrem (sodium oxybate) for the treatment of fibromyalgia.
The drug is chemically similar to GHB — the “date-rape” drug. Approving the drug for such a large patient population would risk flooding the streets with a pharmaceutical-grade version of the highly controlled substance.
“Sodium oxybate and GHB are the same thing,” said Lewis Nelson, MD, of the New York University School of Medicine, one of the FDA panelists. “This is much better than the stuff you get on the street, and that is the problem.”
The drug is only approved for the treatment of narcolepsy. It has been prescribed for 35,000 people since being introduced in 2002.
RISKY BUSINESS
Thomas Kosten, MD, of Baylor University, who loudly opposed approval, cited a lack of convincing evidence by Jazz that the risks involved in releasing the drug to such a large population were balanced by its effectiveness in treating fibromyalgia and narcolepsy, the two conditions for which the drug maker was seeking approvals.
“Without any data to show that this is better than existing medications, I think we are foolish to consider approving this drug,” Kosten said in the discussion immediately preceding the vote.
The panel’s vote contrasted with the FDA’s view of the data provided by Jazz, which included two studies measuring pain relief, and another that tested effectiveness as a sleep aid.
During a public comment period, the majority of speakers supported approval of the drug. Many of them had participated in the studies conducted by Jazz. They explained how much they had benefited from sodium oxybate after failing to respond to any of the three other drugs approved for fibromyalgia.
As many as half of all patients with fibromyalgia do not respond to available medications, according to Jon Russell, MD, director of the University Clinical Research Center at the University of Texas Health Science Center at San Antonio.
CONCERNS
FDA panelists were not convinced that the company had made enough of an effort to prove the drug’s effectiveness, nor did they have confidence in the company’s ability to monitor and manage the potential risks associated with sodium oxybate.
The panel said there was a lack of data concerning drug interactions for patients on multiple medications, as well as risks for people with multiple conditions. They also expressed concerns for the likelihood of misuse, and confusion over what the FDA calls the drug’s “unusual and complex dosing arrangement.”
But it was the link to GHB and the potential for abuse in the difficult-to-monitor market that drove the 20-2 vote.
If a drug needs to get on the market, there needs to be a mechanism in place to mitigate risk, which the panel clearly found lacking.
Via: redorbit.com