FDA Approves New Indications for First-in-Class Oral Small Molecules and Monoclonal Antibody Therapies

Innovative Mechanism! FDA Approves Another "First-in-Class" Oral Small Molecule

GSK recently announced that the U.S. FDA has approved the oral antibiotic Blujepa (gepotidacin) for the treatment of Neisseria gonorrhoeae susceptible to the drug.Neisseria gonorrhoeae) strains in adolescent and adult patients aged 12 years and above with uncomplicated urogenital gonorrhea. These patients weigh at least 45 kg and are unsuitable for, intolerant of, or unwilling to accept standard first-line treatment.According to the press release, Blujepa is the first entirely new class of antibiotic approved for the treatment of gonorrhea in over 30 years.

The marketing application of Blujepa is primarily based on the positive results from the Phase 3 clinical trial, EAGLE-1. Analysis shows,AcceptTwiceThe genitourinary cure rate of patients treated with oral Blujepa was 92.6%.(187/202, 95% CI: 88.0-95.8), compared with the cure rate of 91.2% (186/204, 95% CI: 86.4-94.7) for the regimen of intramuscular ceftriaxone combined with oral azithromycin, met the criteria for non-inferiority. No treatment failures in the urogenital tract due to persistent Neisseria gonorrhoeae were observed in either group, and the safety profile was consistent with previous clinical outcomes. No serious drug-related adverse events were observed, and common adverse reactions were mild to moderate gastrointestinal symptoms.

Blujepa is an oral "first-in-class" antibiotic with a unique mechanism of action.It can evenly inhibit two different type II topoisomerases, thereby exerting antibacterial activity against most target pathogens, including strains resistant to existing antibiotics. Because it equally inhibits the activity of both enzymes, bacteria would need to develop mutations in both enzymes simultaneously to significantly reduce sensitivity to Blujepa, making the likelihood of resistance development low. In March this year, Blujepa was approved by the U.S. FDA for the treatment of uncomplicated urinary tract infections (uUTI) in women and children aged 12 years and older (body weight ≥40 kg).

FDA Approves Amgen's Monoclonal Antibody Therapy Again

Amgen announced that the U.S. FDA has approved the antibody Uplizna (inebilizumab) for the treatment of adult patients with generalized myasthenia gravis (gMG) who have acetylcholine receptor antibodies (anti-AChR) and anti-muscle-specific tyrosine kinase antibodies (anti-MuSK).Uplizna is a CD19-targeted humanized monoclonal antibody., which can specifically and continuously eliminate key cells associated with the disease (CD19-positive B cells that produce autoantibodies, including plasmablasts and some plasma cells).This approval allows gMG patients to potentially achieve long-term disease control with Uplizna administered twice a year after receiving two initial doses.

This approval is mainly based on the data from the MINT study.In the study,Patients using baseline steroids began tapering at week 4 and reduced prednisone to 5 mg daily by week 24; by week 26, 87.4% of patients in the Uplizna group and 84.6% in the placebo group had tapered their steroid dose to 5 mg or less per day.At Week 26, patients in the Uplizna group showed a 1.9-point improvement difference in the Myasthenia Gravis Activities of Daily Living (MG-ADL) score compared to placebo (-4.2 vs. -2.2; p<0.0001).

FDA Approves Small Molecule for Expanded Indication

BioCryst Pharmaceuticals today announced that the U.S. FDA has approved its once-daily oral therapy Orladeyo (berotralstat) granule formulation for the prophylactic treatment of hereditary angioedema (HAE) in children aged 2 to <12 years. According to the press release,Orladeyo is the first targeted oral preventive therapy approved for HAE patients aged 2 years and older, with its granule formulation offering a more acceptable administration method for children.

Orladeyo is an innovative oral specific plasma kallikrein inhibitor.The therapy was approved by the FDA in 2020 for the prevention of HAE attacks in adults and patients aged 12 years and older. This approval was primarily based on the positive interim data from the APeX-P clinical trial. The study results showed that Orladeyo was well-tolerated in this age group, with a safety profile consistent with previous adult and adolescent trials. It demonstrated a sustained reduction in the number of monthly attacks in patients early in the treatment, with no new safety risks identified. The most common treatment-emergent adverse events (TEAEs) were nasopharyngitis.

References:

[1] Blujepa (gepotidacin) approved by US FDA as oral option for treatment of uncomplicated urogenital gonorrhoea (uGC). Retrieved December 12, 2025 from https://www.gsk.com/en-gb/media/press-releases/blujepa-gepotidacin-approved-by-us-fda-as-oral-option-for-treatment-of-uncomplicated-urogenital-gonorrhoea-ugc/

[2] BioCryst Announces FDA Approval of ORLADEYO® (berotralstat) Oral Pellets, First and Only Oral Prophylactic Treatment for Patients with HAE Aged 2 to <12 Years. Retrieved December 12, 2025 from https://www.globenewswire.com/news-release/2025/12/12/3204568/29446/en/BioCryst-Announces-FDA-Approval-of-ORLADEYO-berotralstat-Oral-Pellets-First-and-Only-Oral-Prophylactic-Treatment-for-Patients-with-HAE-Aged-2-to-12-Years.html

[3] FDA APPROVES UPLIZNA® FOR ADULTS WITH GENERALIZED MYASTHENIA GRAVIS. Retrieved December 12, 2025 from https://www.prnewswire.com/news-releases/fda-approves-uplizna-for-adults-with-generalized-myasthenia-gravis-302639699.html

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