FDA committee recommends 2nd new hepatitis C drug for approval

A Food and Drug Administration advisory committee is unanimously recommending approval of a second new treatment–in as many days–for hepatitis C genotype 1 infection in adults with liver damage who have not been treated or whose treatment failed. Hepatitis C is a chronic viral disease that causes inflammation and swelling of the liver.

The drug, telaprevir, is a new class of protease inhibitor manufactured by Vertex Pharmaceuticals. It too would be used in combination with ribavirin and peginterferon–the current standard of care. Telaprevir also prevents the virus from replicating, and studies show the three-drug cocktail is more effective than the two-drug regimen.

"We are thrilled with today's FDA Committee decision," said Dawn Kalmar, a Vertex spokeswoman. "This is an important step in our more than 15 year effort to bring telaprevir to people living with hepatitic C."

Wednesday, the committee unanimously approved boceprevir, a similar protease inhibitor manufactured by Merck & Co.

According to Vertex, about 79% of study patients who hadn't been treated and 85% of those who were treated but relapsed showed significant improvement when treated with telaprevir. The drug is administered orally 3 times a day with food.

"It's a stunning achievement that we will be able to cure nearly 80% of naive patients and probably close to the same number of relapsers," said Dr. Lawrence S. Freidman, Chair, Department of Medicine Newton-Wellseley Hospital. "There are so many positive aspects of this drug and for those of us who have been in the field, this is a very exciting time."

"The benefits far outweigh the risks," said Patrick Clay, Director, Dybedal Center for Clinical Research, Kansas City University of Medicine. "This is yet another step and that's all it is. This is a marathon not a sprint."

As with boceprevir, data for hard to treat patients like African Americans,  Hispanics and Latinos was very limited but those involved in the studies responded extremely well to therapy.

The most common side effects, according to Vertex and the FDA were rash and anemia. While panelists expressed concern, all felt the risks were manageable and stressed the importance of management programs that educate both patients and physicians.

Risks were not enough to sway KellyAnn Mann Hester, a mother of 4 and grandmother of 5 who was diagnosed with hepatitis C almost 20 years ago. Hester had a number of failed treatments and said she was once told she would not see her 5 year old son graduate high school. She participated in the telaprevir study and gave an impassioned plea for approval.  "I had no hope left that I was going to live with this disease. I was living to die," said Hester. "Now I'm living until I die because now I have many windows and avenues available to me that I did not have before.  Please approve this drug."

The FDA will now consider the committee's recommendation and decide if one or both of these drugs will be approved.