Gepotidacin accepted for priority review by the US FDA for the oral treatment of uncomplicated urogenital gonorrhoea

• Submission supported by positive phase III data in patients with uncomplicated urogenital gonorrhoea in EAGLE-1 trial¹
• Significant need for new antibiotics for gonorrhoea, a priority pathogen for the World Health Organization²
• If approved, gepotidacin would offer a new oral option to US patients currently relying on injectable treatments
• 11 December assigned as Prescription Drug User Fee Act (PDUFA) goal date for FDA decision

GSK plc (LSE/NYSE: GSK) today announced that the US Food and Drug Administration (FDA) has accepted for priority review a supplemental New Drug Application for gepotidacin as an oral option for the treatment of uncomplicated urogenital gonorrhoea in patients 12 years of age and older (weighing ≥45 kg). The US FDA has assigned a Prescription Drug User Fee Act action date of 11 December 2025. In March 2025, gepotidacin was approved by the US FDA under the licensing name Blujepa as oral treatment for female adult and paediatric patients 12 years of age and older (weighing ≥40 kg) with uncomplicated urinary tract infection (uUTI).³

Gonorrhoea is a common, sexually transmitted infection caused by Neisseria gonorrhoeae, which has been recognised by the World Health Organization as a priority pathogen² and an urgent public health threat by the US Centers for Disease Control and Prevention (CDC).⁴ It affects both men and women and if left untreated or inadequately treated, it can lead to infertility and other sexual and reproductive health complications. There were more than 600,000 cases of gonorrhoea reported in the United States in 2023 according to the CDC, making it the second most commonly reported sexually transmitted infection in the country.⁵ There is currently no vaccine licensed in the US for the prevention of gonorrhoea infection and the standard of care is injectable treatment which may not be suitable or available for all patients.⁶

The US application is based on results from the EAGLE-1 phase III trial recently published in The Lancet, showing that gepotidacin (oral, two doses of 3,000mg) was non-inferior, with 92.6% (187/202, [95% CI 88·0 to 95·8]) success rates at urogenital site when compared to 91.2% (186/204, [95% CI 86.4-94.7]) success rates for intramuscular ceftriaxone (500mg) plus oral azithromycin (1,000mg) combined therapy, a leading combination treatment regimen for gonorrhoea. Additionally, there were no failures at the urogenital site due to bacterial persistence of N. gonorrhoeae in either treatment arm. The safety and tolerability profile of gepotidacin in the EAGLE-1 trial was consistent with results seen in previous clinical trials, with no serious drug related adverse events observed in either the gepotidacin or the comparator arm. The most common reported adverse reactions were mild to moderate gastrointestinal events.¹

This is the second major indication filed in the US for gepotidacin, and review of regulatory submissions for the uUTI indication is also ongoing in the UK and Australia.

The development of gepotidacin has been funded in part with federal funds from the US Department of Health and Human Services, Administration for Strategic Preparedness and Response, Biomedical Advanced Research and Development Authority (BARDA), under Other Transaction Agreement number HHSO100201300011C and with federal funds awarded by the US Department of Defense’s Threat Reduction Agency under agreement number HDTRA1-07-9-0002.

About gepotidacin

Gepotidacin, discovered by GSK scientists, is a bactericidal, first-in-class triazaacenaphthylene antibiotic that inhibits bacterial DNA replication by a distinct binding site, a novel mechanism of action, and for most pathogens, provides well-balanced inhibition of two different Type II topoisomerase enzymes. This provides activity against Neisseria gonorrhoeae and most target uropathogens (such as Escherichia coli and Staphylococcus saprophyticus), including isolates resistant to current antibiotics. Due to this well-balanced inhibition for most pathogens, a single target-specific mutation may not significantly impact gepotidacin activity.

The US Prescribing Information is available here.

About the EAGLE clinical programme

The EAGLE-1 trial (NCT04010539) is part of a comprehensive global phase III clinical programme for gepotidacin in adults and adolescents including:

EAGLE-1 (non-inferiority urogenital gonorrhoea trial) compared the efficacy and safety of gepotidacin (oral, two doses of 3,000mg) to intramuscular ceftriaxone (500mg) plus oral azithromycin (1,000mg) in approximately 600 patients with uncomplicated urogenital gonorrhoea. The data were presented at ESCMID in April 2024⁷ and published in The Lancet in April 2025.¹

EAGLE-2 and EAGLE-3 (non-inferiority uUTI trials) compared the efficacy and safety of gepotidacin (1,500mg administered orally twice daily for five days) to nitrofurantoin (100mg administered orally twice daily for five days). The data were first presented at European Congress of Clinical Microbiology and Infectious Diseases (ECCMID) in 2023.⁸

GSK in infectious diseases

GSK has pioneered innovation in infectious diseases for over 70 years, and the Company’s pipeline of medicines and vaccines is one of the largest and most diverse in the industry, with a goal of developing preventive and therapeutic treatments for multiple disease areas or diseases with high unmet needs globally. Our expertise and capabilities in infectious disease strongly position us to help prevent disease and mitigate the challenge of antimicrobial resistance (AMR).

About GSK

GSK is a global biopharma company with a purpose to unite science, technology, and talent to get ahead of disease together. Find out more at gsk.com.

Cautionary statement regarding forward-looking statements

GSK cautions investors that any forward-looking statements or projections made by GSK, including those made in this announcement, are subject to risks and uncertainties that may cause actual results to differ materially from those projected. Such factors include, but are not limited to, those described in the “Risk Factors” section in GSK’s Annual Report on Form 20-F for 2024, and GSK’s Q2 Results for 2025.

References

1. Ross J et al, “Oral gepotidacin for the treatment of uncomplicated urogenital gonorrhoea (EAGLE-1): a phase 3 randomised, open-label, non-inferiority, multicentre study” in The Lancet, 2025; 405: 1608–20; https://doi.org/10.1016/ S0140-6736(25)00628-2
2. WHO. bacterial priority pathogens list, 2024: Bacterial pathogens of public health importance to guide research, development and strategies to prevent and control antimicrobial resistance. Available at: https://www.who.int/publications/i/item/9789240093461 Last accessed: August 2025
3. GSK. Blujepa approved by US FDA for treatment of uncomplicated urinary tract infections. Available at: https://www.gsk.com/en-gb/media/press-releases/blujepa-gepotidacin-approved-by-us-fda-for-treatment-of-uncomplicated-urinary-tract-infections/ Last accessed: August 2025
4. CDC. Antibiotic Resistance Threats Report. Available at: https://www.cdc.gov/antimicrobial-resistance/media/pdfs/covid19-impact-report-508.pdf Last accessed: August 2025
5. CDC. National Overview of STIs in 2023. Available at: https://www.cdc.gov/sti-statistics/annual/summary.html. Last accessed: August 2025
6. CDC. STI treatment guideline. Available: https://www.cdc.gov/std/treatment-guidelines/default.htm Last accessed: August 2025
7. GSK. EAGLE 1 phase III data show potential for gepotidacin as a new oral treatment option for uncomplicated urogenital gonorrhoea (GC) amid growing resistance to existing treatments. Available at: https://www.gsk.com/en-gb/media/press-releases/eagle-1-phase-iii-data-show-potential-for-gepotidacin-as-a-new-oral-treatment-option-for-uncomplicated-gc/ Last accessed: August 2025.
8. GSK. Gepotidacin’s positive phase III data shows potential to be the first in a new class of oral antibiotics for uncomplicated urinary tract infections in over 20 years. Available at: https://www.gsk.com/en-gb/media/press-releases/gepotidacin-s-positive-phase-iii-data-shows-potential-to-be-the-first-in-a-new-class-of-oral-antibiotics-for-uncomplicated-urinary-tract-infections/ Last accessed: August 2025.