Date Rape Drug May Soon Be Approved for Fibromyalgia!

[Pharma Newshound]

Source: Bloomberg.com

Pain Drug With Date-Rape Chemical Is Safe, FDA Says

By Molly Peterson

Aug. 18 (Bloomberg) -- Jazz Pharmaceuticals Inc.’s fibromyalgia drug is safe and effective, said U.S. regulators weighing whether to approve the medicine, which includes a chemical linked to date rape.

The treatment, JZP-6, reduced pain in clinical trials and its potential for side effects “appears to be acceptable,” Food and Drug Administration staff reviewers said in a report issued today. An FDA advisory panel will meet Aug. 20 to evaluate whether the drug’s benefits to patients with fibromyalgia, an illness that causes chronic muscle pain and fatigue, outweigh risks of abuse and accidental overdose. Jazz sells the drug, known chemically as sodium oxybate, under the name Xyrem for people with narcolepsy, a sleep disorder.

Gamma hydroxybutyrate, a sedative also known as GHB, is the active ingredient in Xyrem and the new fibromyalgia medicine. Illegal forms of GHB have been used as club drugs and are implicated in date rapes, according to the National Institute on Drug Abuse. The FDA panel also will assess whether Jazz’s risk evaluation and mitigation strategy is sufficient to curb potential abuse of the fibromyalgia drug.

“The fact that the FDA didn’t raise a whole lot of questions on the side effects is a major positive,” Difei Yang, an analyst with San Diego-based Capstone Investments Inc., said in a telephone interview. “I think eventually the FDA is going to approve the drug; it’s just a matter of how restrictive the REMS program will have to be to mitigate the risk.”

Shares Rise

Jazz, based in Palo Alto, California, rose 36 cents, or 3.5 percent, to $10.61 at 4 p.m. New York time in Nasdaq Stock Market composite trading, after jumping as much as 16 percent earlier today. The shares have gained 35 percent this year.

Sales of Xyrem, which were $96.8 million last year, may reach $178 million in 2014, William Tanner, an analyst with Lazard Capital Markets in New York, said in a research note on Aug. 12. Revenue from JZP-6, if it’s approved, may be $134 million that year, he said.

Jazz expects the FDA to complete its review of the drug by Oct. 11. The company has proposed selling it under the name Rekinla through about 15 specialty pharmacies that would mail it to patients.

The FDA approved Xyrem for narcolepsy patients in 2002 as an orphan drug, a category used for medications that treat conditions affecting 200,000 or fewer people. Fibromyalgia affects an estimated 5 million Americans, according to the National Institutes of Health.

Controlled Substance

The Drug Enforcement Administration classifies Xyrem as a schedule III controlled substance, meaning distribution is restricted because of the potential for abuse.

“Do we need a schedule III drug, with all sorts of abuse potential, for fibromyalgia? I don’t know,” said Ira Loss, an analyst at Washington Analysis who has followed the FDA for more than three decades, in a telephone interview on Aug. 3. “I’m very skeptical about the prospects of that drug for that indication” because other treatments are available.

Jazz, as part of its FDA application for JZP-6, proposed an “effective, proven” risk mitigation process that “leverages our experience over the past eight years with Xyrem,” Bruce Cozadd, chairman and chief executive officer, said on a conference call on Aug. 10.

Central Pharmacy

Xyrem is mailed to patients through a central pharmacy rather than sold in retail drugstores. A registry tracks doctors who prescribe the drug and patients who use it. Xyrem also comes with a black box warning, the FDA’s strictest caution, alerting patients that misuse can lead to seizures, coma and death.

Selling sodium oxybate under two trade names could cause potentially fatal medication errors for patients who suffer from both narcolepsy and fibromyalgia, FDA staff said. Jazz failed to convince the agency that the patient populations don’t overlap, and it’s unclear whether the company’s safeguards would prevent such errors, according to the report.

Approving sodium oxybate for the fibromyalgia population also would “almost assuredly increase the likelihood of abuse and misuse” of the drug, agency staff said.

Ten cases of abuse occurred among the 26,000 narcolepsy patients worldwide who received Xyrem from 2002 to 2008, according to a Jazz-sponsored study published last year in the Journal of Clinical Sleep Medicine. The drug also was linked to one death and two cases of sexual assault. Five of the 600,000 bottles of Xyrem distributed to narcolepsy patients during that period were used by other people, the study found.

‘Low’ Abuse Rates

“What we found was that really, the rates of abuse and misuse were low,” said Lawrence Carter, an assistant professor of psychiatry at the University of Arkansas for Medical Sciences, in Little Rock, who was a co-author of the report. “We concluded that this was likely due to the risk management plan that the company had in place.”

There isn’t any reason to believe fibromyalgia patients would be more likely than narcolepsy patients to abuse the drug, said Carter, a former Jazz employee who is now a paid consultant to the company, in a telephone interview on Aug. 4.

“Most of what you hear about GHB comes from the use of illicit GHB, not Xyrem,” he said.

Concerns about Jazz’s risk-management plan may prompt the FDA advisory panel to vote against approval of the fibromyalgia drug, Cole Werble, an analyst at Prevision Policy LLC in Washington, and two of his colleagues said today in a research note. Still, the drug is likely to win FDA clearance once the company addresses the agency’s concerns, they said.

“A negative vote coming out of committee could be confusing, considering the overall prospects for approvability are more positive,” the Prevision analysts said.

‘More Cumbersome’

JZP-6 may have “relatively modest commercial potential” because it will be “more cumbersome” for fibromyalgia patients than other drugs, Tanner said in his research note. Pfizer Inc.’s pain drug Lyrica, Eli Lilly & Co.’s antidepressant Cymbalta, and Savella, from Forest Laboratories Inc. and Cypress Bioscience Inc., are approved by the FDA for fibromyalgia and are available in retail pharmacies.

“How JZP-6 fits in alongside other fibromyalgia drugs remains to be determined,” Tanner said.

To contact the reporter on this story: Molly Peterson in Washington at mpeterson9@bloomberg.net

[Pharma Newshound]

Source: WSJ

By JENNIFER CORBETT DOOREN

WASHINGTON—The Food and Drug Administration raised safety concerns about a proposed Jazz Pharmaceuticals Inc. drug to treat the pain disorder fibromyalgia, including the potential for misuse and abuse of the product.

The drug, which is currently sold under the brand name Xyrem to treat narcolepsy, faces a review Friday during a joint meeting of the FDA's arthritis and drug-safety advisory committees. The FDA posted background documents prepared for the meeting on its website Wednesday.

Xyrem is known by its generic name, sodium oxybate. However, the company is proposing to sell the product as Rekinla to treat fibromyalgia. It would be dosed in a different manner than currently used for narcolepsy, a condition marked by excessive daytime sleepiness.

Sodium oxybate is sold as part of a controlled distribution system through a central pharmacy that was designed to limit abuse and misuse of the product. In an illegal form, sodium oxybate is known as the street drug GHB.

Patients and doctors are also required to be enrolled in the program in order to prescribe and receive Xyrem, which is an oral drug taken twice in the evening. The product carries the FDA's toughest boxed warning, stating the drug is a central nervous system depressant "with abuse potential." The product label also warns the drug has been associated with confusion, depression and other neuropsychiatric events.

Jazz is proposing to sell the product under a different restricted distribution program than currently used for narcolepsy, which would involve 15 specialty pharmacies.

The FDA said the existence of two restricted distribution programs may create confusion among prescribers and would be burdensome to the health-care system. The agency also said it was concerned that having two different brand names for the product could lead to medication errors. Additionally, the agency said it was concerned there was a "higher risk" for dosing errors if the proposed 375 milligram per milliliter dose is used for fibromyalgia patients. The drug is currently sold in a 500-milligram concentration to treat narcolepsy.

However, the agency said clinical data submitted by Jazz suggests sodium oxybate is effective at reducing fibromyalgia pain. The agency said the data wasn't strong enough to show that sodium oxybate improved sleep.

Fibromyalgia is a poorly understood pain condition which is believed to affect three to six million Americans, mostly women. Fibromyalgia is marked by widespread muscle pain, tenderness and fatigue. The condition traditionally has been treated with a combination of painkillers and antidepressants.

There are currently three other products approved to treat fibromyalgia.

The FDA has asked the panel to vote on whether it thinks sodium oxybate should be approved to treat fibromyalgia. The FDA usually follows its panels' advice, but isn't required to.

Write to Jennifer Corbett Dooren at jennifer.corbett-dooren@dowjones.com