Potentially life-saving treatment is safe to use for babies with neonatal sepsis, study reveals

GARDP (Global Antibiotic R&D Partnership) has announced that results from a clinical trial completed in Kenya have determined that a safe dose of the antibiotic, fosfomycin, can be used to treat babies with neonatal sepsis.

This is a significant development, as there are very few antibiotics specifically licensed to treat multidrug-resistant infections in babies, GARDP said in a press release.

The GARDP explained that NeoFosfo trial investigated the pharmacokinetics (PK) and safety of fosfomycin in 120 babies aged under 28 days who were hospitalized with clinically diagnosed sepsis at the Kilifi County hospital in Kenya.  The PK studies define which dose to use in babies.

The trial was sponsored by the Global Antibiotic Research and Development Partnership (GARDP) and the Drugs for Neglected Diseases initiative (DNDi) and supported by key partners.

“This is a very encouraging outcome for the treatment of neonatal sepsis and the care of babies who are hardest-hit by rising antimicrobial resistance (AMR),” said Neonatal Sepsis Project Leader for GARDP, Sally Ellis. “The study provides crucial evidence of the correct dosage of fosfomycin for newborns.”

A paper on the outcome of the trial,  which was recently published in the global paediatric journal, Archives of Diseases in Childhood, indicated that fosfomycin offered significant potential as part of a combination antibiotic regimen for newborns, which is safe, easily administered, and affordable, GARDP said.

The paper comes after recent data showed that Sub-Saharan Africa has the highest overall burden of AMR in the world, and that babies in particular are most affected. The GRAM study, published in The Lancet in January 2022, revealed that children under five years old made up over half of the 255,000 people in Sub-Saharan Africa who died in 2019 because of AMR.

Neonatal sepsis is a life-threatening condition that requires prompt detection and treatment. Newborns – babies under 28 days old – are particularly vulnerable as their underdeveloped immune systems struggle to fight infections. This is complicated by drug resistance, as up to 40% of bacterial infections are resistant to standard treatments. Many newborns die if they don’t get timely treatment, while others may have significant long-term consequences, GARDP explained.

“Currently there are very limited antibiotics in the pipeline. Safe and affordable antibiotic combinations effective against bacteria causing sepsis in babies are needed to improve survival.  Our results are significant as they provide evidence that fosfomycin is safe and can now be taken forward into further clinical trials focusing on improving mortality outcomes for sepsis in babies,” said clinical investigator on the trial, Christina Obiero of the KEMRI-Wellcome Trust Research Programme in Kilifi, Kenya.

Fosfomycin is an antibiotic that is used in some countries to treat serious bacterial infections but has rarely been used for treatment of serious infection in babies admitted to hospitals. It is an off-patent antibiotic and thus potentially inexpensive, and has been identified as ‘critically important’ by the World Health Organization (WHO), GARDP said.

The results of the NeoFosfo trial will be followed by a large GARDP-led international clinical trial, NeoSep, which will enrol three thousand babies, including in Kenya and South Africa. It will obtain robust evidence of the safety and efficacy of new antibiotic combinations including fosfomycin, compared to other WHO-recommended and existing antibiotic combinations, for the treatment of neonatal sepsis.

It is expected that results obtained from this trial could inform WHO and local antibiotic treatment guidelines, as partners aim to demonstrate which combinations are safe and effective in treating neonatal sepsis.