GSK Receives FDA Orphan Drug Designation for Kidney Disease Treatment

GSK's kidney disease treatment has received FDA orphan designation. The company, a global biopharma firm, focuses on addressing unmet treatment needs for patients with various conditions, including respiratory and inflammatory diseases. Its vaccine portfolio protects against diseases like meningitis, shingles, and flu. Specialty medicines address respiratory disease and HIV, while general medicines treat asthma, COPD, and skin diseases. The company is also advancing oligonucleotide therapeutics for chronic hepatitis B and steatotic liver disease.

GlaxoSmithKline (GSK) has received UK approval for its oral antibiotic Blujepa (gepotidacin), marking a significant milestone in the fight against antibiotic-resistant urinary tract infections (UTIs). The UK Medicines and Healthcare products Regulatory Agency (MHRA) has granted approval for Blujepa to treat uncomplicated UTIs in females aged 12 and older, weighing at least 40 kg [1].

Blujepa's unique mechanism of action involves blocking two bacterial enzymes essential for replication, making it effective against many drug-resistant infections, including E. coli [2]. The approval comes nearly three decades after the last new oral antibiotic was introduced [3]. The UK approval follows the U.S. Food and Drug Administration's (FDA) approval in March and supports GSK's broader strategy to expand its infectious disease portfolio [1].

The drugmaker's focus on developing new treatments for UTIs is driven by the increasing prevalence and severity of these infections. According to the MHRA, UTIs affect about half of all women in the UK, and the risk of complications, such as sepsis and kidney damage, is rising due to antibiotic resistance [3]. Blujepa's effectiveness was demonstrated in two late-stage Phase 3 trials involving over 3,100 patients, where it was at least as effective as the current standard treatment, nitrofurantoin [3].

GSK expects Blujepa, along with two other medicines in development, to generate peak yearly sales of more than 2 billion pounds ($2.7 billion) and aims to reach overall annual sales of over 40 billion pounds by 2031 [2]. The company plans to work with UK health authorities and the National Institute of Health and Care Excellence (NICE) to make Blujepa available for eligible patients [2].

References:
[1] https://seekingalpha.com/news/4489875-uk-clears-gsks-new-oral-antibiotic-for-utis
[2] https://www.reuters.com/business/healthcare-pharmaceuticals/uk-approves-gsks-antibiotic-female-urinary-tract-infections-2025-08-27/
[3] https://stocktwits.com/news-articles/markets/equity/gsk-secures-uk-approval-for-first-new-oral-antibiotic-in-30-years-to-treat-resistant-urinary-tract-infections/chsCM1iRdl1