FDA Approves Gilead’s Harvoni, the First Once-Daily Single Tablet Regimen for the Treatment of Genotype 1 Chronic Hepatitis C

Gilead Sciences has announced that FDA has approved Harvoni (ledipasvir 90 mg/sofosbuvir 400 mg), the first once-daily single tablet regimen for the treatment of chronic hepatitis C genotype 1 infection in adults. Harvoni combines the NS5A inhibitor ledipasvir with the nucleotide analog polymerase inhibitor sofosbuvir, approved under the tradename Sovaldi in December 2013. Harvoni’s efficacy has been established in patients with chronic hepatitis C virus (HCV) genotype 1 infection, with a treatment duration of eight, 12 or 24 weeks depending on prior treatment history, cirrhosis status and baseline viral load. Eight weeks of treatment with Harvoni can be considered for treatment-naïve patients without cirrhosis who have baseline HCV viral load below 6 million IU/mL. 

FDA granted Harvoni a Priority Review and Breakthrough Therapy designation, which is given to investigational medicines that may offer major advances in treatment over available therapies.

“By providing very high cure rates in as little as eight weeks and completely eliminating the need for interferon and ribavirin, which are challenging to take and tolerate, Harvoni significantly advances treatment for patients with the most common form of hepatitis C in the US,” said Nezam Afdhal, MD, Director of Hepatology at Beth Israel Deaconess Medical Center, Professor of Medicine at Harvard Medical School and a principal investigator in the Harvoni clinical trials. “For the first time, the vast majority of patients can be cured with a once-daily pill in only eight or 12 weeks.”

Harvoni’s approval is supported by data from three Phase III studies, ION-1, ION-2 and ION-3. These studies evaluated eight, 12 or 24 weeks of treatment with Harvoni, with or without ribavirin, among nearly 2000 genotype 1 HCV patients with compensated liver disease. These studies included non-cirrhotic treatment-naïve patients (ION-3), cirrhotic and non-cirrhotic treatment-naïve patients (ION-1) and cirrhotic and non-cirrhotic patients who failed prior therapy with an interferon-based regimen, including regimens containing an HCV protease inhibitor (ION-2). The primary endpoint for each study was sustained virologic response (HCV undetectable) 12 weeks after completing therapy (SVR12).

Patients who achieve SVR12 are considered cured of HCV. In these studies, ribavirin was not shown to increase response rates. Trial participants in the ribavirin-free arms (n=863) achieved SVR12 rates of 94 to 99 percent.

“Unlike other serious chronic diseases, hepatitis C can be cured and Harvoni offers patients the potential for a cure in as little as 8 weeks,” said John C. Martin, PhD, Chairman and CEO, Gilead Sciences. “Gilead is proud to have played a role in developing a once-daily therapy that is safe, simple and well tolerated. We are now working to ensure rapid and broad access to Harvoni.”