FDA Approves First Combination Pill for Hepatitis C Virus

The FDA today approved Gilead’s Harvoni, the first combination pill to treat chronic hepatitis C virus (HCV) genotype 1 infection.

Harvoni is comprised of sofosbuvir, a previously approved HCV drug marketed under the brand name Sovaldi, and a new drug called ledipasvir. Both drugs interfere with the enzymes needed by HCV to multiply.

Harvoni is also the first approved regimen that does not require administration with interferon or ribavirin, 2 FDA-approved drugs also used to treat HCV infection.

“With the development and approval of new treatments for HCV, we are changing the treatment paradigm for Americans living with the disease,” said Edward Cox, MD, MPH, director of the Office of Antimicrobial Products in the FDA’s Center for Drug Evaluation and Research, in a press release. “Until last year, the only available treatments for HCV required administration with interferon and ribavirin. Now, patients and health care professionals have multiple treatment options, including a combination pill to help simplify treatment regimens.”

Harvoni’s efficacy was evaluated in 3 clinical trials enrolling 1518 participants who had not previously received treatment for their infection or had not responded to previous treatment, including those with cirrhosis. Participants were randomly assigned to receive Harvoni with or without ribavirin.

The trials were designed to measure whether HCV was no longer detected in the blood at least 12 weeks after finishing treatment, indicating that a participant’s HCV infection has been cured.

In the first trial, comprised of treatment-naive participants, 94% of those who received Harvoni for 8 weeks and 96% of those who received Harvoni for 12 weeks achieved sustained virologic response (SVR). The second trial showed 99% of such participants with and without cirrhosis achieved SVR after 12 weeks. And, in the third trial, which examined Harvoni’s efficacy in treatment-experienced participants with and without cirrhosis, 94% of those who received Harvoni for 12 weeks and 99% of those who received Harvoni for 24 weeks achieved SVR.

In all trials, ribavirin did not increase response rates in the participants. The most common side effects reported in participants were fatigue and headache.

Harvoni is the seventh new drug with breakthrough therapy designation to receive FDA approval and the third drug approved in the past year to treat chronic HCV infection. The FDA approved simeprevir (Olysio) in November 2013 and Sovaldi in December 2013.

According to the US Centers for Disease Control and Prevention, about 3.2 million Americans are infected with HCV, and without proper treatment, 15% to 30% of these patients will go on to develop cirrhosis.