The first new drug to treat hepatitis C in over 20 years moved one step closer to approval Wednesday when a panel of government experts endorsed Merck's boceprevir as an effective treatment for the virus.
The Food and Drug Administration's panel of experts voted unanimously, 18-0, that the overall benefits of the tablet-based drug outweigh its risks. The agency generally follows the recommendations of its panels. A final decision is expected mid-May.
"I think this is going to be a real game changer for our hepatitis C practice and I can't wait to tell my patients about it," said panelist Barbara McGovern of Tufts University School of Medicine.
Panelists said the drug is largely safe, but noted side effects including anemia and lower blood cell counts. Some panelists also worried about patients' ability to stick to the drug's three-tablets-a-day regimen.
The FDA convened a two-day meeting to review two new drugs that block the enzyme which allows the hepatitis virus to reproduce. Vertex Pharmaceuticals Inc. will present its drug Thursday. The meeting brings to a head more than 15 years of research by both drugmakers to find a more effective therapy for a virus that infects about 3.2 million people in the U.S., or about 1.5 percent of the population.
Like HIV drugs, the new drugs from Merck and Vertex will be prescribed as part of a cocktail with two older drugs.
Martha Saly, director of the National Viral Hepatitis Roundtable, said the new drugs are an important step forward, but patients will still have to deal with the side effects of the older drugs _ ribavirin pills and interferon-alpha injections.
"There are a lot of patients out there who have been waiting for this drug before seeking treatment," said Saly, who spoke during a public comment period at the meeting. "But they're probably not going to be as excited when they realize it isn't replacing the older drugs."
The current two-drug treatment for hepatitis C cures only about 40 percent of people with the most common variety of the virus, and causes side effects like anemia, nausea and vomiting.
Merck & Co.'s boceprevir has been shown to boost cure rates to between 60 and 65 percent when combined with the older drugs. Another key advantage highlighted by Merck is the potential to cut treatment time in half, to six months, lessening exposure to negative side effects from the older drugs.
But panelists said some patients probably should not get the shorter treatment because they have not shown a strong response to boceprevir. Those patients include African Americans, those with liver disease and those who have failed prior treatment.
Analysts expect boceprevir to reach annual sales between $700 and $800 million after it is approved. Vertex Pharmaceuticals' telaprevir is expected to garner sales of up to $3 billion due to higher efficacy data and a simpler treatment regimen.
Whitehouse Station, N.J.-based Merck & Co. Inc. was the first company to market a drug for hepatitis C in 1991 when it launched interferon-alpha. The company began developing boceprevir soon thereafter.
Shares rose 57 cents, or 1.6 percent, to close at $35.63 Wednesday.