Genentech’s Xofluza Submitted to FDA for New Children’s Formulations

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The U.S. Food and Drug Administration (FDA) accepted a New Drug Application (NDA) and two supplemental NDAs (sNDAs) for Genentech’s Xofluza (baloxavir marboxil). The NDA is for a new formulation of the drug as one-dose granules for oral suspension, would which largely be aimed at children and people who have difficulty swallowing.

Genentech, a Roche company, is also applying to get Xofluza approved for the treatment of acute uncomplicated influenza in healthy children aged one year to less than 12 years who have been symptomatic for no more than 48 hours. Another sNDA is for post-exposure prophylaxis of the flu in children one year of age and older for the oral suspension and currently-available tablet formulations. The target action date for these NDAs is November 23, 2020.

“As this has been one of the hardest-hitting influenza seasons for children in the past decade, there is a critical need for additional treatment options that attack influenza in different ways,” said Levi Garraway, Genentech’s chief medical officer and head of Global Product Development. “Today’s milestone brings us closer to providing single-dose Xofluza to children with influenza. We also look forward to working with the FDA to incorporate Xofluza as a preventive treatment following exposure to influenza.”

The formulation in the NDA is for granules that are reconstituted with water by a pharmacist before dispensing to an individual. If approved, it would be administered as a single one-time dose. Xofluza tablets are approved around the world for influenza A and B.

The regulatory submissions are built on positive data from two Phase III trials, miniSTONE-2 and BLOCKSTONE. Both were recently presented at the 2019 OPTIONS X congress held in Singapore. miniSTONE-2 evaluated the safety, pharmacokinetics and efficacy of single-dose, oral suspension compared to oseltamivir (Tamiflu) in healthy children aged one to less than 12 years of age with flu. BLOCKSTONE studied Xofluza compared to placebo as a preventive treatment for members of a household who were living with someone with the flu.

Xofluza is approved for use to treat the influenza in people 12 years of age and older who have had flu symptoms for no more than 48 hours who are otherwise healthy or at high risk of developing flu-related complications. It was approved in October 2018. It is a first-in-class, one-dose oral drug. It belongs to a class of antiviral medicines that inhibit the cap-dependent endonuclease protein, which viruses need to replicate.

Although it can be easy to forget in the midst of the current COVID-19 pandemic, influenza is a serious, infectious disease that kills thousands each year. The U.S. Centers for Disease Control and Prevention (CDC) have estimated that since 2010, influenza has caused 9.3 to 45 million illnesses in the U.S., 140,000 to 810,000 hospitalizations and 12,000 to 61,000 deaths.

Genentech developed and commercialized Xofluza via its agreements with Shionogi & Co. Shionogi discovered the drug. Under the terms of the arrangement, Roche holds worldwide rights to Xofluza except in Japan and Taiwan, which are retained exclusively by Shionogi.

Xofluza is also being evaluated in a Phase III development program for children under the age of one year, for severely ill, hospitalized patients with influenza, and to evaluate its potential to decrease transmission of flu from an infected person to healthy people.

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