Clinical trial establishes effective HIV dosing regimen for full-term newborns

An international study conducted through the International Maternal Pediatric Adolescent AIDS Clinical Trials (IMPAACT) Network including researchers at the University of California San Diego helped inform the U.S. Food and Drug Administration's (FDA) recent approval of dolutegravir, a second-generation integrase inhibitor, for the treatment of human immunodeficiency virus (HIV-1) in newborns from birth. The study, published in The Lancet HIV, identified a dosing regimen that achieves appropriate drug levels in full-term newborns, supporting the use of dolutegravir to treat HIV from the first days of life.

Previously, dolutegravir was approved only for adults and children aged four weeks and older. The new findings help establish an appropriate dosing regimen for use beginning at birth, expanding access to an antiretroviral medication widely used to treat HIV in older children and adults.

Newborns process medications differently than older children and adults, and their ability to eliminate drugs changes rapidly during the first month of life. Our study provides important pharmacokinetic and safety data to guide dolutegravir dosing during this critical period of development."

Jeremiah Momper, PharmD, PhD, first author on the study and professor, UC San Diego Skaggs School of Pharmacy and Pharmaceutical Sciences

The study, known as IMPAACT 2023, was a Phase I clinical trial conducted across sites in South Africa, Thailand and the United States, led by researchers at Boston Medical Center (BMC). Researchers enrolled 48 full-term newborns who were born to women living with HIV-1. Depending on the study group, infants received either two separate single doses of dolutegravir or repeated doses during the first weeks of life, in addition to standard antiretroviral prophylaxis. All infants were followed for 16 weeks to assess safety.

The researchers found that dolutegravir achieved levels in newborns comparable to those observed in older children and adults receiving the medication for HIV treatment. Additionally, the medication was generally well tolerated, with a safety profile similar to that observed in older infants and children.

The FDA approval, announced in August 2026, extends the label for dolutegravir to treat HIV-1 infection in full-term infants weighing at least two kilograms from birth up to four weeks of age in combination with other antiretroviral medications. The expanded approval provides an additional treatment option for newborns, a population with relatively few approved antiretroviral medications.

"For too long, newborns living with HIV have had too few treatment options designed and studied to meet their unique needs, despite the importance of starting treatment as early as possible," said Diana Clarke, PharmD, lead investigator of the study and a pediatric infectious diseases pharmacist at Boston Medical Center. "This represents an important advance for the care of newborns, as early treatment with the most effective therapies can greatly improve health outcomes.