FDA Approves HIV Drug for Adults With Multidrug-Resistant Infection

Heavily treated patients living with HIV have few treatment options, but this critical unmet need may be addressed by a new FDA-approved drug. Fostemsavir (Rukobia), from ViiV Healthcare, was approved for the treatment of HIV-1 infection for use in combination with other antiretroviral therapies in adults with multidrug-resistant infection.

Heavily treatment-experienced (HTE) adults account for approximately 6% of adults living with HIV. The patients indicated for fostemsavir are failing their current antiretroviral regimen because of resistance, intolerance, or safety considerations. These patients have few options and are at risk of progressing to AIDS and death, according to ViiV Healthcare.

“There is a small group of heavily treatment-experienced adults living with HIV who are not able to maintain viral suppression with currently available medication and, without effective new options, are at great risk of progressing to AIDS,” Deborah Waterhouse, CEO of ViiV Healthcare, said in a statement. “The approval of Rukobia is a culmination of incredibly complex research, development, and manufacturing efforts to ensure we leave no person living with HIV behind.”

The FDA reviewed and approved the treatment under the Fast Track and Breakthrough Therapy Designations. Approval was based on findings from a phase 3 study in which a majority of the patients who received fostemsavir achieved and maintained viral suppression. The BRIGHTE study enrolled 371 HTE adults with HIV-1 infection with multidrug resistance.

The participants were enrolled in either a randomized or nonrandomized cohort. The randomized cohort included 272 participants who were on 1 or 2 fully active and available antiretroviral agents at screening that could be combined as part of a background regimen. Participants received either blinded fostemsavir 600 mg twice daily (n = 203) or placebo (n = 69) in addition to the regimen they were failing on for 8 days. After day 8, they received open-label therapy plus an investigator-selected optimized background therapy (OBT).

In the nonrandomized cohort, 99 patients were treated with open-label fostemsavir 600 mg twice daily plus OBT. In the nonrandomized cohort, the participants had no fully active and approved antiretroviral agents at screening.

A total of 163 (60%) patients who received fostemsavir in addition to OBT achieved undetectable HIV viral load and clinically meaningful improvements to CD4⁺ T-cell count by week 96 of the study.

At week 96, 5% of the patients in the randomized cohort and 7% in the nonrandomized cohort had discontinued fostemsavir due to an adverse event (AE). The most common AEs (all grades) in both cohorts were nausea, fatigue, and diarrhea. AEs related to infections most commonly led to discontinuation of treatment.

“Some members of the HIV community face very challenging treatment journeys and do not respond to available therapies for a variety of reasons,” said Gabriel Maldonado, founder and CEO, TruEvolution, Inc, a nonprofit organization that fights for health equity and racial justice to advance the quality of life and human dignity of LGBTQ people. “The approval of fostemsavir provides a sense of renewed hope for these adults who have few or no viable treatment options left and have been awaiting alternative medicines to control the virus.”

Fostemsavir is currently under review by the European Medicines Agency.