Regulatory Affairs

FDA approves supplemental applications of two hepatitis C drugs for pediatric patients

Published 10 April 2017

Gilead Sciences has secured approval from the US Food and Drug Administration (FDA) for supplemental indications of Harvoni (ledipasvir 90 mg/sofosbuvir 400 mg) tablets and Sovaldi (sofosbuvir 400 mg) tablets for chronic hepatitis C virus (HCV) infection treatment in adolescents without cirrhosis or with compensated cirrhosis, 12 years of age and older, or weighing at least 35kg.

Harvoni was approved for pediatric patients with genotype 1, 4, 5 or 6 chronic HCV infection. Sovaldi was approved for pediatric patients with genotype 2 or 3 chronic HCV infection, in combination with ribavirin.

There are an estimated 23,000-46,000 pediatric HCV patients in the United States, most of whom were infected with the virus at birth.

Karen Murray, M.D., professor of pediatrics at the University of Washington School of Medicine and Seattle Children's said: “The approvals of Sovaldi and Harvoni for pediatric patients will enable adolescents to finally benefit from interferon-free treatment for HCV infection.

“These therapies address a significant unmet medical need and represent an important advance for HCV-infected adolescents.”

Norbert Bischofberger, Ph.D., Executive Vice President of Research and Development and Chief Scientific Officer at Gilead said: “Gilead’s goal is to develop and deliver treatments that provide all patients with HCV the potential to be cured.

“For the first time, children 12 and older in the United States with genotypes 1 through 6 chronic HCV infection now have options of two direct-acting antiviral regimens that offer high cure rates while eliminating the need for interferon injections.”

Harvoni and Sovaldi each have a boxed warning in their respective product labels regarding the risk of hepatitis B virus reactivation in HCV/HBV co-infected patients. See below for important safety information.

Harvoni for Pediatric Patients

The supplemental new drug application (sNDA) approval is supported by data from an open-label clinical trial (Study 1116), which evaluated 12 weeks of treatment with Harvoni once-daily in genotype 1 treatment-naïve and treatment-experienced HCV-infected adolescents 12 years of age and older without cirrhosis or with compensated cirrhosis. The SVR12 rate was 98 percent overall (98/100).

No subject experienced on-treatment virologic failure or relapse. Two subjects were lost to follow-up.

Adverse events were consistent with those observed in clinical studies of Harvoni in adults. The most common adverse reactions (≥10 percent, all grades) observed with treatment with Harvoni in HCV-infected pediatric patients were fatigue, headache and asthenia.

Sovaldi for Pediatric Patients

The sNDA approval is supported by data from an open-label clinical trial (Study 1112), which evaluated 12 or 24 weeks of treatment with Sovaldi and weight-based ribavirin in adolescents 12 years of age and older with HCV genotype 2 or 3.

The SVR12 rate was 100% (13/13) in genotype 2 patients and 97% (36/37) in genotype 3 patients. No subject experienced on-treatment virologic failure or relapse. One patient was lost to follow up.

Adverse events were consistent with those observed in clinical studies of Sovaldi in adults. The most common adverse reactions (≥15 percent, all grades) observed with treatment with Sovaldi and ribavirin for 12 or 24 weeks in HCV-infected pediatric patients were fatigue, headache and nausea.

U.S. Patient Support Program

To support these patients and their families, Gilead’s U.S. Support Path program provides information regarding access and reimbursement coverage options to patients in the United States who need assistance with coverage for their medications, including Harvoni and Sovaldi.

Support Path conducts benefits investigations and provides patients with information regarding their insurance options.

Further, the Harvoni and Sovaldi Co-pay Coupon Programs offer co-pay assistance for eligible patients with private insurance who need assistance paying for out-of-pocket medication costs.



Source: Company Press Release