FDA HIV draft recommends seeking full approval

FDA issued draft guidance on developing antiretrovirals to treat HIV-1 infection that recommends sponsors seek full approval of therapies from the outset -- shifting away from prior practice of seeking accelerated approval based on 24-week virologic response data and subsequently seeking full approval based on 48-week data. FDA said since HIV-RNA is now a validated surrogate for predicting efficacy of antiretrovirals, sponsors can seek full approval, with the duration of viral load assessment dependent on the patient population.

The guidance recommends evaluating as the primary endpoint the proportion of patients with HIV-RNA levels below the limit of assay detection at 48 weeks in treatment-naive patients. In treatment-experienced patients with other options, the agency recommends evaluating the endpoint at 48 weeks if showing non-inferiority to comparators or at 24 weeks if showing superiority. For both treatment-naïve and treatment-experienced patients, FDA recommends active-controlled, non-inferiority trials, though the guidance notes that add-on superiority trials are also an option in treatment-experienced patients. ...