CDSCO Mandates Phase III Trial for Baloxavir

The Subject Expert Committee (SEC) on Antimicrobial and Antiviral drugs, under the Central Drugs Standard Control Organization (CDSCO), rejected Roche Products India’s request for a waiver of local clinical trials for Baloxavir Marboxil (brand name Xofluza) during its meeting on 13-14 February 2025. The committee mandated that Phase III clinical trials must be conducted in the Indian population before the drug can receive marketing approval. The SEC concluded that acute uncomplicated influenza does not constitute an unmet medical need in India, and therefore the conditions for granting a trial waiver under the New Drugs and Clinical Trials Rules, 2019 were not met.

Baloxavir Marboxil is a first-in-class cap-dependent endonuclease inhibitor that targets the polymerase acidic (PA) protein of the influenza virus, blocking the "cap-snatching" mechanism essential for viral mRNA synthesis. Unlike neuraminidase inhibitors such as Oseltamivir (Tamiflu), which prevent viral release from host cells, Baloxavir inhibits viral replication at an earlier stage. The drug was first approved in Japan in 2018 and subsequently in the US, Canada, and Australia. Under India’s New Drugs and Clinical Trials Rules, 2019 (Rule 101), the Drugs Controller General of India (DCGI) may waive local Phase III trials for drugs already approved in specified countries, but only when conditions such as unmet medical need or absence of alternative therapies are satisfied.