The FDA has approved a new fixed-dose combination for the treatment of chronic hepatitis C (HCV) infections. The combination of glecaprevir (an NS3/4A inhbitor) and pibrentasvir (an NS5A inhibitor) is taken once a day and treats all six genotypes of HCV. It is the third approved combination that treats all six HCV genotypes, after sofosbuvir/velpatasvir (Epclusa) and sofosbuvir, velpatasvir, voxilaprevir (Vosevi). It is approved for eight weeks of therapy in treatment-naïve patients and 12 weeks for those with compensated cirrhosis. Some clinical scenarios may require 16 weeks of therapy. Approval was based on studies of more than 2,300 subjects across all six HCV genotypes in whom the sustained viral response at 12 weeks (SVR12) was 93-100%. A new study shows the efficacy of the combination across all six genotypes in patients with and without cirrhosis. Treatment for 12 weeks resulted in SVR12 of 99% (Lancet Infect Dis 2017. doi: 10.1016/S1473-3099(17)30496-6). Glecaprevir/pibrentasvir is marketed as Mavyret. AbbVie has priced the drug significantly lower than other HCV treatments at $13,200 per month or $26,400 per eight-week treatment course.
In what the FDA calls a “historic action,” the agency approved the first gene therapy available in the United States. Tisagenlecleucel is a cell-based gene therapy approved for the treatment of patients up to 25 years of age with B-cell precursor acute lymphoblastic leukemia that is refractory or in second or later relapse. Clinicians collect a patient’s T cells and send them to a manufacturing center, where a technician genetically modifies the cells with a new gene that contains a chimeric antigen receptor. This new combination directs the T cells to target and kill leukemia cells that contain the CD10 antigen on their surfaces. Once the T cells are modified, clinicians infuse the mixture into the patient. Safety and efficacy was demonstrated in a trial of 63 pediatric and young adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia. The overall remission rate within three months of treatment was 83%. Side effects included cytokine release syndrome and neurologic events, both of which can be life threatening. The drug is approved with a risk evaluation and mitigation strategy. Hospitals and clinics that administer the drug must be certified. Tocilizumab recently was approved to treat cytokine release syndrome, and the FDA is requiring that all certified hospitals and clinics make it available before administering tisagenlecleucel. The FDA granted the drug priority review and breakthrough therapy designations. It is marketed as Kymriah. The cost is projected to be $475,000 for one course of treatment.
The FDA has approved a new intravenous antibiotic combination to treat adults with complicated urinary tract infections (UTIs), including pyelonephritis. The new product pairs the antibiotic meropenem with vaborbactam, which inhibits resistance mechanisms used by bacteria. It is approved to treat infections caused by Escherichia coli, Klebsiella pneumoniae, or Enterobacter cloacae species complex. Safety and efficacy was shown in a study of 545 adults with complicated UTIs, which showed a cure rate of 98% for patients treated with meropenem/vaborbactam vs. 94% for patients treated with piperacillin/tazobactam. Headache, infusion site reactions, and diarrhea are the most common side effects. Meropenem/vaborbactam received priority review and was designated a qualified infectious disease product, a designation given to antibacterial products that treat serious or life-threatening infections under the Generating Antibiotic Incentives Now title of the FDA Safety and Innovation Act. Meropenem/vaborbactam is marketed as Vabomere.
In this section: The agency approves fixed-dose combination hepatitis C treatment, greenlights first U.S. gene therapy, and approves new urinary tract infection drug.
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