Hepatitis C drug raises cure rate in late trial

An experimental drug for hepatitis C from Vertex Pharmaceuticals, telaprevir, sharply increased the cure rate in a clinical trial, while reducing the time needed for treatment. Experts said the results could herald a new era in treating the sometimes-fatal disease that is often overlooked, as reported by The New York Times.

About 75% of patients in the trial who received the standard dose of telaprevir in combination with the existing treatment were essentially cured, compared with 44% of those who received only existing therapy.

“These data support the vision that we have long had for fundamentally changing the treatment for hepatitis C,” Robert Kauffman, MD, PhD, chief medical officer of Cambridge, Mass.-based Vertex Pharmaceuticals, told reporters in a conference call.

Between 2.7 million and 3.9 million Americans and 170 million people worldwide are estimated to have hepatitis C, and 12,000 die from it each year, according to a recent report by the Institute of Medicine.

The disease can cause cirrhosis, which is a scarring of the liver, as well as liver cancer. The virus is transmitted by blood, so most people who have hepatitis C either have injected illegal drugs or received blood transfusions or blood products before the early 1990s.

The existing treatment is a combination of alpha interferon, which is injected, and ribavirin, which is administered orally. It is not quite clear how those drugs work.

For patients with Genotype 1, the most prevalent type of hepatitis in the United States, the treatment lasts nearly a year and can cause side effects that include flu-like symptoms, anemia, and depression. It is so arduous that many patients opt not to get treated or drop out before completing the course.

But in Vertex’s trial, about 70% of those who achieved an effective cure were able to do so in 24 weeks. That shorter timeline, plus a higher chance of success, could entice more people to be treated, experts said.

Vertex said it expects results from 2 additional phase 3 trials of telaprevir in the third quarter and will then apply to FDA for approval of the drug.

If approved, telaprevir would have to be used initially with existing drugs to prevent resistance. But experts hope that eventually several of the pills now in development could be used together, much as AIDS is treated, doing away with the need for interferon.

Vertex’s clinical trial enrolled 1,095 patients from the United States and other countries who had Genotype 1 hepatitis C and had not been treated previously. Patients received telaprevir for either 12 or 8 weeks or a placebo. The standard therapy of interferon and ribavirin was given for either 24 weeks or 48 weeks, depending on how the patients were faring after 4 weeks and 12 weeks.

Of those who received telaprevir for 12 weeks, 75% had a sustained viral response, meaning the virus was not detectable in their blood 24 weeks after completing treatment. That is seen as essentially a cure. The rate was 69% for those who received telaprevir for 8 weeks.

The drug can cause a rash, and it increases the rate of anemia. About 7% of the patients who received telaprevir dropped out of the trial, about double the figure for the control group.

Lorren Sandt, chairwoman of the National Viral Hepatitis Roundtable, called the trial results impressive but added that if telaprevir gains FDA approval, it will be accessible and affordable to everyone.