On Jun. 12, 2020, the Food and Drug Administration (FDA) announced it had approved an expanded indication for Merck’s GARDASIL9 for the prevention of oropharyngeal and other head and neck cancers caused by HPV Types 16, 18, 31, 33, 45, 52, and 58. The oropharyngeal and head and neck cancer indication is approved under accelerated approval based on effectiveness in preventing HPV-related anogenital disease. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

Gardasil 9 was licensed in 2014 for prevention of HPV-related cervical, vaginal and vulvar cancers in females and HPV-related anogenital lesions and anal cancers in males and females. The expanded indication broadens the rationale for use to prevent HPV-related cancers in both sexes and includes individuals ages 9 through 45 years.

The FDA granted the expanded indication under the accelerated approval licensure pathway. This pathway uses a surrogate endpoint for earlier approval of drugs or biologics that treat serious conditions and that fill an unmet medical need.

The FDA relied on effectiveness data used to support approval for prevention of HPV-related anogenital infection and disease as a surrogate to predict clinical benefit in prevention of HPV-related oropharyngeal infection and disease. It also used epidemiologic evidence to support that prevention of persistent oropharyngeal HPV infection is reasonably likely to predict prevention of HPV-related head and neck cancer.

As required with accelerated approval, a post-approval confirmatory study is underway to evaluate the safety and efficacy of Gardasil 9 vaccination for prevention of persistent oropharyngeal HPV infection. Analyses of the randomized, blinded, placebo-controlled trial will be driven by the number of oropharyngeal HPV-positive cases identified through polymerase chain reaction testing at six-month intervals. Once complete, the FDA will evaluate the study data to determine whether to grant traditional approval for this indication or remove the indication from the label.