Xofluza safe and effective for flu treatment in children: MINISTONE-2 Study

Treatment with one-dose oral suspension medicine, XOFLUZA (baloxavir marboxil) was well tolerated in children with the flu, demonstrate results from Phase III MINISTONE-2 study that examined the safety and efficacy of Xofluza versus oseltamivir in children with flu (aged 1 to less than 12 years). Also, the drug was found to be equally effective as oseltamivir in reducing the duration of flu symptoms including fever. Oseltamivir is a proven effective treatment for children with flu.

The results were announced by the biotechnology company Genentech.

XOFLUZA is the first and only one-dose oral medicine approved to treat the flu. It is the first novel medicine with a novel mechanism of action approve by the U.S. Food and Drug Administration. It is proven to be effective in otherwise healthy people with flu (CAPSTONE-1), people at high risk of complications from the flu (CAPSTONE-2), and preventive measure for the people exposed to an infected household member (BLOCKSTONE)

"Children need new medicines for the flu because they are at higher risk of developing the flu and more likely to have complications such as breathing problems and pneumonia. These flu complications, which in some cases can be fatal, lead to approximately one million children under five being admitted to the hospital globally every year," said Sandra Horning, chief medical officer and head of Global Product Development, said in the company's press release. "As a one-dose oral suspension medicine, XOFLUZA could potentially provide a convenient treatment option for children with the flu, and we look forward to sharing these data with health authorities around the world."

XOFLUZA is currently approved in several countries, including Japan for the treatment of influenza types A and B in children, adolescents, and adults, and in the U.S. for the treatment of acute, uncomplicated influenza in people 12 years of age and older. In addition, the FDA recently accepted a supplemental New Drug Application (sNDA) for XOFLUZA as a one-dose oral treatment for people at high risk of complications from the flu. The FDA is expected to decide on whether to approve this additional indication by November 4, 2019.

MINISTONE-2 is a Phase III, multicenter, randomized, double-blind study that evaluated the safety, pharmacokinetics and efficacy of one-dose of XOFLUZA compared with oseltamivir in otherwise healthy children aged one to less than 12 years with an influenza infection confirmed by a rapid influenza diagnostic test and displaying influenza-like symptoms (a temperature of 38°C or over, and one or more respiratory symptoms).

Participants enrolled in the study were recruited in parallel into two cohorts: patients aged five to less than 12 years and patients aged one to less than 5 years. Patients in both cohorts were randomly assigned to receive one-dose of XOFLUZA (2mg/kg for patients under 20kg or 40mg for patients 20kg or over) or oseltamivir twice a day over five days (dosing according to body weight). The primary endpoint of the study was the proportion of patients with adverse events or severe adverse events up to study day 29. Secondary endpoints include pharmacokinetics, time to alleviation of influenza signs and symptoms, and duration of symptoms, including fever.

About XOFLUZA (baloxavir marboxil)

XOFLUZA is a first-in-class, one-dose oral medicine with a novel proposed mechanism of action that has demonstrated efficacy in a wide range of influenza viruses, including in vitro activity against oseltamivir-resistant strains and avian strains (H7N9, H5N1) in non-clinical studies. Unlike other currently available antiviral treatments, XOFLUZA is the first in a new class of antivirals designed to inhibit the cap-dependent endonuclease protein, which is essential for viral replication.

XOFLUZA is being further studied in a Phase III development program, including children under the age of one, and severely ill, hospitalized people with the flu, as well as to assess the potential to reduce transmission in otherwise healthy people.