Roche announces positive results for first global phase III study investigating one-dose Xofluza (baloxavir marboxil) in children with flu

Roche announced that the phase III MINISTONE-2 study met its primary endpoint, demonstrating that Xofluza (baloxavir marboxil) was well tolerated in children with flu. The study also showed that Xofluza is comparable to oseltamivir – a proven effective treatment for children with flu – at reducing the duration of flu symptoms, including fever. The study assessed Xofluza versus an active comparator (oseltamivir) in children aged between one and less than 12 years old with flu. Full results from MINISTONE-2 will be presented at an upcoming medical meeting.

“Children need new medicines for flu because they are at higher risk of developing 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 hospital globally every year,” said Sandra Horning, MD, Roche’s Chief Medical Officer and Head of Global Product Development. “As a one-dose oral suspension medicine, Xofluza could potentially provide a convenient treatment option for children with flu, and we look forward to sharing these data with health authorities around the world.”

The safety and efficacy of Xofluza in children with flu under the age of one is also being studied in the global phase III MINISTONE-1 study (NCT03653364). Xofluza is the first and only one-dose oral medicine approved to treat flu and the first new flu medicine with a novel proposed mechanism of action approved by the FDA in nearly 20 years. Xofluza is also the only flu treatment shown to be efficacious in both otherwise healthy people with flu (CAPSTONE-1) and people at high risk of complications from flu (CAPSTONE-2), as well as a preventive measure against developing flu following exposure to an infected household member (BLOCKSTONE).

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 United States (US) 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 flu. The FDA is expected to decide on whether to approve this additional indication by 4 November 2019.