April 27, 2011 – A new treatment for hepatitis C could soon be on the market if the Food and Drug Administration takes the advice of an advisory committee. The committee unanimously approved the first of two new drugs to treat chronic hepatitis C genotype 1 infection. Hepatitis C is a chronic viral disease that causes inflammation and swelling of the liver.
The drug, boceprevir, is a new class of protease inhibitor manufactured by pharmaceutical giant Merck & Co., and would be used in combination with ribavirin and peginterferon�the current standard of care. Boceprevir prevents the virus from replicating, and studies show the three-drug cocktail is more effective than the two-drug regimen.
According to Merck, about 66% of study patients that hadn’t been treated or did not respond well to current treatment responded well to boceprevir.
The FDA’s outside panel of experts considered the risks and benefits of the drug and determined the benefits far outweigh the risks.
“The benefit for the people who do achieve sustained virologic response is fantastic,” said Dr. Elizabeth Connick, University of Colorado Denver. “This is a miraculous advance.”
“The risks are not trivial, but we do know how to manage these risks. Still, there is a lot to learn about using these drugs appropriately,” said Dr. Thomas Giordano of Baylor College of Medicine.
“This is going to be a real game changer for our hepatitis C practices, ” said Dr. Barbara McGovern, Tufts University School of Medicine. “I can’t wait to get back and talk to my patients about it.”
Even hard-to-treat patients like African Americans, and people with HIV and diabetes responded extremely well to the boceprevir combination therapy.
Dr. Nizar Zein, section head of hepatology at the Cleveland Clinic says the disease is of epidemic proportions and quite costly. Zein say the new medication will usher in a very important new era for treating the disease.
“We can now say for the first time that we can cure hepatitis C,” Zein said. “We are talking about complete cure, cure for life. Several studies have shown that once you achieve that endpoint, the sustained virological response, you will not get hepatitis C ever again and the risk of getting cirrhosis, needing liver transplant will go down substantially.”
But this is a powerful drug and a major concern is that drug resistance can develop in patients very quickly if the medication is not taken properly. The proposed dosage is 800 mg three times a day with food.
Treatment can be individualized but Zein says both doctors and patients need to be educated and clear about when and how this drug is to be taken. “If used inappropriately, the virus will rapidly develop resistance to these medications � rapidly, I mean 24 to 48 hours.”
Close attention was paid to the drug’s side effects. The most common, according to Merck included anemia, abnormally low white blood cell count, fatigue, nausea, headache, hair loss and an impaired sense of taste.
According to FDA’s Dr. Poonam Mishra, the most notable safety concern is the additional decrease in hemoglobin� the protein in red blood cells that carries oxygen. Hemoglobin levels lower than normal could mean anemia, bleeding or a number of other conditions. Mishra said anemia was managed effectively during the clinical trials and was reversible after the drug was discontinued. Only a few serious and life-threatening infections were reported.
Martha Saly, director of the National Viral Hepatitis Roundtable, who learned that she had hepatitis C in 1998 and was successfully treated with interferon, said even though treatment will be extremely challenging, for the first time she has hope for a universal cure.
“I believe that approval of these drugs will be the biggest thing that has happened in years,” Saly said. “I am hopeful that a shortened course of treatment for many patients will equate with a greater ability to tolerate the drugs and manage the side effects, but I fear that the issue of compliance with strict medication regimens might affect treatment success.”
Saly says the drugs will help few patients if awareness of the hepatitis C epidemic, improved screening and affordable access to care are not addressed.
More than 3.2 million Americans have hepatitis C, the most common bloodborne infection in the country. According to the Centers for Disease Control and Prevention about 10,000 people die of liver disease and liver cancer directly related to hepatitis C each year. It’s usually caused by contact with infected blood products, though it can also be transmitted sexually or contracted through IV-drug use.
The disease is the leading cause of liver cirrhosis, and the need for liver transplants in the United States. Cirrhosis is when scar tissue forms in the liver, replacing healthy liver tissue and blocking blood flow through the liver.
Symptoms included abdominal pain, dark urine, fever, and jaundice but most people don’t have symptoms until liver damage has occurred which, according to the National Institute of Diabetes and Digestive and Kidney Diseases , could be 10 years or more.
The FDA is not bound by their advisory committee recommendations but often follow their advice. Merck says it anticipate FDA action on boceprevir by mid-May.
“The positive recommendation brings us one step closer to bringing Victrelis (boceprevir) to men and women who need it, and reinforces our ongoing commitment to developing innovative therapies to treat chronic hepatitis C,” said Peter S. Kim, Ph.D., president, Merck Research Laboratories.
On Thursday, the panel will make recommendations on Vertex Pharmaceuticals’ telaprevir, the other new drug in this class. The FDA released a preliminary review of telaprevir saying clinical trials showed, when combined with standard treatments, it was more effective than those therapies. Vertex is hoping to beat Merck to the finish line by winning FDA approval first.
