With declarations that a new day is dawning in the treatment of hepatitis C, members of a federal advisory panel unanimously approved the first of two new drugs to treat the stubborn liver infection on Wednesday.
The committee is expected to green light the second hep-C drug today. Few doubt the Food and Drug Administration will clear the new drugs for market, possibly as soon as next month.
"This changes the game completely," says Dr. Victoria Cargill of the National Institutes of Health, acting chair of the FDA's advisory committee. "I can look into the faces of the people (with hepatitis C) and offer them some hope."
"I can't wait to get back and talk to my patients about it," enthused panel member Dr. Barbara McGovern of Tufts Medical School in Boston.
Studies show that the new drugs – so-called protease inhibitors that interfere with the hep-C virus' ability to replicate – eliminate the virus in 65 to 80 percent of patients. Standard treatments, which involve the drugs ribavirin and pegylated interfereon, cure less than half of patients.
Many patients with hepatitis C have reportedly been delaying treatment in the hope of more effective new drugs.
The drug approved Wednesday, by an 18-0 vote, is Merck's boceprevir, which will carry the brand name Victrelis. Studies indicate it is somewhat less effective than Vertex Pharmaceutical's telaprevir, which is up for a vote today. Both are effective in many cases against the worst form of hepatitis C, type 1.
Analysts predict Merck will pull in around $800 million a year from boceprevir, while Vertex might sell $3 billion worth of teleprevir. At least 3.2 million Americans have hepatitis C. The strongest risk factors are a history of injection drug use, multiple sex partners and blood transfusion before 1992.
Yet enthusiasm for the drugs is tempered by a lot of questions about who is likely to benefit and how to manage serious side effects, such as anemia and severe rashes. The advisory panel came up with three dozen post-marketing studies it wants Merck to do.
"I do think you have to be somewhat of a Talmudic scholar to prescribe this drug," says panel member Dr. Lawrence Friedman of Massachusetts General Hospital.
That's because many hepatitis C patients were excluded from efficacy studies – for instance, if they had failed on standard treatments, or if they were resistant to conventional drugs.
Advisory committee members also complained about the lack of information on African-American patients, who often don't respond as well to standard hepatitis C therapy. Cargill, who specializes in treating minorities with HIV infection, says this is a big concern. "I practice in a setting where approximately 95 percent of our patients are co-infected with hepatitis C" and HIV, she says.
Committee members also worry about the complexity of taking the new drugs (which are added to conventional treatments), patients' ability to adhere to treatment, the need to monitor them for signs of anemia, and the drugs' value for patients who have not responded to standard treatment.
But ultimately the panelists agreed that the benefits of the new pills clearly outweigh the risks. And this isn't the end of the story. Panel member Doris Strader of the University of Vermont points out that "there are drugs (for hepatitis C) coming along that may be better and simpler to use."
Update: The FDA advisory panel voted unanimously, 18-0, to approve telaprevir on Thursday.
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