U.S. Food and Drug Administration Approves New Formulation of Epclusa®, Expanding Pediatric Indication to Treat Children Ages 3 and Older With Chronic Hepatitis C
– Novel oral pellet formulation enables early childhood treatment with pangenotypic, panfibrotic regimen –
Gilead Sciences, Inc. (Nasdaq: GILD) today announced that the U.S. Food and Drug Administration (FDA) has approved an extension of the pediatric indication of Epclusa® (sofosbuvir/velpatasvir) for the treatment of chronic hepatitis C virus (HCV) to now include children from 3 years, regardless of HCV genotype or severity of liver disease. The FDA has approved a New Drug Application (NDA) for two strengths of an oral pellet formulation of Epclusa (sofosbuvir 200 mg/velpatasvir 50 mg and sofosbuvir 150 mg/velpatasvir 37.5 mg), designed for use by younger children who cannot take tablets. to swallow. The recommended dose of Epclusa in children 3 years of age and older is based on weight.
Treatment with Epclusa for 12 weeks was approved in patients without cirrhosis or with compensated cirrhosis (Child-Pugh A), and in combination with ribavirin (RBV) for patients with decompensated cirrhosis (Child-Pugh B or C). Sofosbuvir/velpatasvir is the only protease inhibitor-free, pangenotypic HCV regimen approved for patients from 3 years of age.
About 35,300 to 60,500 children were living with HCV in the United States as of 2018, and the incidence is increasing. Mother-to-child transmission, the most common cause of HCV infection in children, increased by 161% between 2009 and 2017, with intravenous drug use being the leading cause of HCV infection in women of reproductive age.
“Glead remains steadfast in our commitment to support the elimination of HCV. Today’s decision by the FDA represents a significant advance toward that goal by expanding more treatment options for children living with HCV,” said Merdad Parsey, MD, PhD, Chief Medical Officer, Gilead Sciences. “This approval adds to the robust clinical evidence supporting the safety and efficacy of Epclusa in a broad group of patients, including those with end-stage renal disease and all stages of fibrosis.”
The approval of Epclusa for children from 3 years of age is based on data from a phase 2, open-label clinical trial that enrolled 41 children aged 3 years to less than 6 years for treatment with Epclusa for 12 weeks. At 12 weeks after completion of treatment, Epclusa achieved a sustained virologic response (SVR12) or cure rate of 83% (34/41) in all patients, 88% (28/32) in children with HCV genotype 1, 50% (3/ 6) in children with HCV genotype 2 and 100% in children with HCV genotype 3 (2/2) and HCV genotype 4 (1/1). Of the seven patients who did not achieve a cure, all discontinued within one to 20 days of treatment initiation.
The safety profile of Epclusa in treated children aged 3 to less than 6 years was generally consistent with that observed in clinical studies in adults. Vomiting and problems with product use (spitting up the drug) were reported in 15% and 10% of subjects, respectively; these adverse reactions were mild (Grade 1 or 2) and led to treatment discontinuation in 5 (12%) subjects.
See below for the US indication and important safety information, including BOXED WARNING, for Epclusa.
“The treatment of pediatric HCV remains a major public health priority. The results of the Phase 2 clinical trial previously showed that this drug was effective in treating many HCV-infected patients, regardless of genotype,” said Karen Murray, MD, president of Cleveland Clinic Children’s and lead investigator of the pediatric study. “Now the extended approval and oral pellet formulation provide new treatment strategies in younger patients with HCV.”
IMPORTANT SAFETY INFORMATION AND INDICATION FOR USING EPCLUSA
BOX WARNING: RISK OF REACTIVATION OF HEPATITIS B VIRUS IN HCV/HBV COHBNFECTED PATIENTS
Test all patients for signs of current or previous hepatitis B virus (HBV) infection before initiating treatment with EPCLUSA. HBV reactivation has been reported in HCV/HBV co-infected patients who were undergoing or had completed HCV direct-acting antiviral therapy (DAAs) and not receiving HBV antiviral therapy. Some cases have resulted in fulminant hepatitis, liver failure, and death. Cases have been reported in patients who are HBsAg positive, in patients with serological evidence of cleared HBV and also in patients receiving certain immunosuppressants or chemotherapeutic agents; the risk of HBV reactivation associated with treatment with HCV-DAAs may be increased in patients taking these other agents. Monitor HCV/HBV co-infected patients for hepatitis flare or HBV reactivation during HCV treatment and post-treatment follow-up. Initiate appropriate patient treatment for HBV infection as clinically indicated.
When EPCLUSA is used in combination with ribavirin (RBV), all contraindications, warnings and precautions, especially avoidance of pregnancy, and side effects of RBV also apply. Refer to RBV’s prescribing information.
Warnings and Precautions
Severe symptomatic bradycardia when co-administered with amiodarone: Amiodarone is not recommended for use with EPCLUSA due to the risk of symptomatic bradycardia, especially in patients also taking beta-blockers or with underlying cardiac co-morbidities and/or with advanced liver disease. A fatal cardiac arrest was reported in a patient taking amiodarone who was co-administered a sofosbuvir-containing regimen. Cardiac monitoring is recommended in patients without alternative viable treatment options. Patients should seek immediate medical attention if they develop signs or symptoms of bradycardia.
Risk of Reduced Therapeutic Effect Due to Use with P-gp inducers and/or moderate to strong inducers of CYP2B6, CYP2C8, or CYP3A4: Rifampine, St. John’s wort, and carbamazepine are not recommended for use with EPCLUSA because they contain sofosbuvir and/or or velpatasvir plasma concentrations.
The most common adverse reactions (≥10%, all grades) with EPCLUSA in adults and pediatric patients 6 years of age and older were headache and fatigue; and when used with RBV in adults with decompensated cirrhosis, there were fatigue, anemia, nausea, headache, insomnia, and diarrhea. The most common adverse reactions (≥10%, Grade 1 or 2) in pediatric patients less than 6 years of age were vomiting and spitting up the drug.
Co-administration of EPCLUSA is not recommended with proton pump inhibitors, phenobarbital, phenytoin, rifabutin, rifapentin, efavirenz and tipranavir/ritonavir due to decreased concentrations of sofosbuvir and/or velpatasvir.
Please refer to the full prescribing information for EPCLUSA for more information on potentially significant drug interactions, including clinical comments.
EPCLUSA is indicated for the treatment of adult and pediatric patients 3 years of age and older with chronic infection with hepatitis C virus genotype 1, 2, 3, 4, 5 or 6 without cirrhosis or with compensated cirrhosis and in combination with ribavirin for humans with decompensated cirrhosis.
About Gilead Sciences
Gilead Sciences, Inc. is a biopharmaceutical company that has been pursuing and achieving breakthroughs in medicine for more than three decades, with the goal of creating a healthier world for all people. The company is committed to advancing innovative medicines to prevent and treat life-threatening diseases, including HIV, viral hepatitis and cancer. Gilead operates in more than 35 countries worldwide, with headquarters in Foster City, California.
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 that are subject to risks, uncertainties and other factors, including the risk that physicians may not see the benefits of prescribing Epclusa for the treatment of chronic HCV. infection and the possibility of adverse results from ongoing and additional clinical studies with Epclusa. These and other risks, uncertainties and other factors are described in detail in Gilead’s Quarterly Report on Form 10-Q for the quarter ended March 31, 2021, as filed with the U.S. Securities and Exchange Commission. These risks, uncertainties and other factors could cause actual results to differ materially from those referred to in the forward-looking statements. All statements other than statements of historical fact are statements that may be considered forward-looking statements.
Investors are cautioned that such forward-looking statements are not guarantees of future performance and involve risks and uncertainties, and are cautioned not to place undue reliance on such forward-looking statements. All forward-looking statements are based on information currently available to Gilead, and Gilead assumes no obligation and disclaims any intention to update such forward-looking statements.
US prescribing information for Epclusa, including BOXED WARNING, is available at www.gilead.com.
Epclusa is a registered trademark of Gilead Sciences, Inc. or its related companies.
For more information about Gilead, visit the company’s website at www.gilead.com, follow Gilead on Twitter (@Gilead Sciences), or call Gilead Public Affairs at 1-800-GILEAD-5 or 1-650-574-3000.
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Nat Sillin, Media