February 21, 2014
The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended a fixed-dose combination of ledipasvir and sofosbuvir (Gilead Sciences, Inc) for chronic hepatitis C virus (HCV) treatment in a compassionate-use program, the EMA announced today.
The drug combination contains the nucleotide analog polymerase inhibitor sofosbuvir (400 mg) and the NS5A inhibitor ledipasvir (90 mg), according to a company statement.
The committee recommended the drug combination to be used in those who urgently need therapy to prevent liver disease progression.
Compassionate-use programs are established at the level of individual member states to provide access to treatments that are still being developed and have not yet received marketing authorization to patients with life-threatening, long-lasting, or seriously disabling disease and no available treatment options.
Sweden requested the CHMP's opinion on providing early access under the compassionate-use program for the ledipasvir/sofosbuvir combination, with or without ribavirin, to adults with genotype 1 HCV infection and advanced liver disease who have a high risk for liver decompensation or death within 12 months without treatment.
In clinical trials, the ledipasvir/sofosbuvir combination, with or without ribavirin, was highly effective for those with genotype 1 virus when used for 12 or 24 weeks. Patients included those with compensated cirrhosis (scarring of the liver with normal liver function) and those in whom previous treatment with the protease inhibitors telaprevir or boceprevir was ineffective. Many of these patients urgently need effective therapy to stop the progression of liver injury in disease that is already very advanced.
This is the third opinion the CHMP has given since October 2013 on the compassionate use of treatments in development for HCV treatment.
The CHMP aims to follow a standard approach regarding the compassionate-use program for new medicinal products with regard to the criteria and conditions of their use before their authorization under member states' legislation.
The CHMP's opinion describes which patients may benefit from the medication, explains how to use the medication, and provides safety information to the EU member states that are considering developing a compassionate-use program; these recommendations are not mandatory.
The agency will publish the assessment report and conditions of use of the ledipasvir/sofosbuvir combination with or without ribavirin in this setting on its Web site in the near future.
Sofosbuvir was granted marketing authorization valid throughout the European Union under the trade name Sovaldi on January 16, 2014.
The US Food and Drug Administration approved sofosbuvir in December 2013.