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Two hepatitis C drugs approved for pediatric use

By: Anna Diyamandoglu, PharmD Candidate c/o 2020

Hepatitis C is an infectious disease which is caused by the hepatitis C virus (HCV) and is characterized by inflammation of the liver. Its effects on the body range from a mild illness that lasts several weeks to a chronic illness that attacks the liver and affects a patient’s life long-term. Acute hepatitis C infection has the propensity to become chronic and does so in 75-80% of patients.1 It is primarily spread through direct contact with the blood of an infected individual. During initial infection, symptoms are expressed very mildly if at all. In fact, a patient can live for years with the illness without experiencing any symptoms, a characteristic which contributes to its lethality. Complications of the disease include cirrhosis, a condition which the liver slowly breaks down and is replaced with scar tissue making it unable to function normally, liver failure, a result of cirrhosis, and liver cancer.1

The only FDA approved medications used to treat hepatitis C were pegylated interferon (Peg-IFN) and ribavirin (RBV) until very recently.2 These two medications were used in combination in order to increase the likeliness of getting rid of HCV from the body even though they often induced side effects that would force the patients to discontinue their therapy.2 Between 2011 and 2016, the FDA approved nine additional medications for the specific use of treating HCV including protease inhibitors boceprevir (Victrelis®) – voluntarily discontinued by Merck in 2015, telaprevir (IncivekÔ) and simeprevir (Olysio®); polymerase inhibitor sofosbuvir (Sovaldi®), enzyme inhibitor ledipasvir/sofosbuvir (Harvoni®); combination medication ombitasvir, paritaprevir, ritonavir (Viekira PakÔ) which can be given without ribavirin thereby avoiding its negative side effects; anti-viral enzyme inducers ombitasvir, paritaprevir, ritonavir (TechnivieÔ)) and daclatasvir (DaklinzaÔ); phosphoprotein/protease inhibitor elbasvir, grazoprevir (Zepatier®); and oral combination medication sofosbuvir, velpatasvir (Epclusa®) which is the first HCV therapy that treats all genotypes of the disease.2

While there have been tremendous strides in terms of increasing available therapies for adult HCV patients, the same could not be said for pediatric patients with the disease until this year. In April 2017, the FDA approved two of the aforementioned medications which had originally been used to treat adults, sofosbuvir and ledipasvir/sofosbuvir, for the treatment of pediatric HCV patients between the ages of 12 and 17.3 The efficacy of these two medications was established from clinical trial data which displayed that after treatment with sofosbuvir and ledipasvir/sofosbuvir, at least 97% of pediatric patients aged 12 or older with varying genotypes of HCV showed no detection of the virus. This concept is called sustained virologic response (SVR).           When the HCV virus is undetectable 12 weeks or more after completing treatment, a sustained virologic response has been achieved. These results are similar to those found in clinical trials which tested the efficacy of the same medications in adult HCV patients.3 This marks the first direct-acting antiviral treatments approved for children and adolescents with HCV.3 In the FDA’s press release statement regarding the recent approval, the importance of this development in pediatric HCV therapy was emphasized. The director of the Office of Antimicrobial Products in the FDA’s Center for Drug Evaluation and Research, Dr. Edward Cox, M.D, stated that it would “help change the landscape for HCV treatment by addressing an unmet need in children and adolescents.”4


  1. Viral hepatitis. Centers for Disease Control and Prevention. Web. Published 05/31/2015. Accessed 04/12/2017.
  2. Advances in medications to treat hepatitis C. American Liver Foundation, n.d. Web. Accessed 04/10/2017.
  3. Barrett, Jennifer. FDA approves 2 hepatitis C drugs for use in pediatric patients. Pharmacy Times. Web. Published 04/07/2017. Accessed 04/11/2017.
  4. Press announcements – FDA approves two hepatitis C drugs for pediatric patients. U.S. Food and Drug Administration. Web. Published 04/07/2017. Accessed 04/10/2017.
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