GASTROENTEROLOGY

FDA approved 46 novel drugs in 2025, including key advances in metabolic, GI, hepatology and obesity treatments.

FDA Drug Approvals 2025: Advances in Metabolic and GI Therapies

In 2025, the FDA’s CDER approved 46 novel therapies, including two metabolic drugs: Lerochol, a PCSK9 inhibitor for lowering LDL cholesterol, and Redemplo, an siRNA therapy reducing triglycerides in familial chylomicronaemia syndrome. This follows 2024, which saw 50 approvals, including six advances in gastroenterology, hepatology, and obesity, such as Linzess for paediatric constipation, Rinvoq for Crohn’s disease, and Wegovy for NASH with fibrosis.

UROLOGY

A gene expression study identified transcripts enriched in urine that may support the development of non-invasive prostate cancer biomarkers.

Urinary Gene Expression Profiles for Prostate Cancer Biomarkers

A comparative gene expression study evaluated differences between prostate tissue and urine samples to identify potential candidates for non-invasive prostate cancer biomarkers. Researchers profiled 367 gene transcripts in 54 prostate tissue and 93 urine samples. Urine samples showed greater enrichment of prostate epithelial, vascular endothelial, and regulatory T-cell marker genes, while tissue samples were enriched for fibroblast and prostate basal cell markers. The findings provide a foundation for developing urine-based biomarkers, although further validation is required.

A study found that neoadjuvant hormonal therapy improved favorable postoperative outcomes but highlighted the importance of baseline risk factors in recurrence prediction.

Neoadjuvant Hormonal Therapy and Recurrence Risk in Prostate Cancer

A retrospective study of 1,099 prostate cancer patients evaluated the impact of neoadjuvant hormonal therapy (Neo-HT) before radical prostatectomy. Neo-HT increased the proportion of patients achieving favorable postoperative pathological and PSA outcomes. However, among patients with favorable postoperative findings, those receiving Neo-HT showed lower unadjusted 3-year biochemical progression-free survival (81.5% vs. 93.6%). After propensity score matching, no statistically significant differences were observed in oncological outcomes. The findings emphasize the importance of baseline disease characteristics in postoperative risk assessment.

A multi-dataset study identified an LHB-associated gene signature linked to reduced androgen receptor signaling and increased proliferative and EMT-related expression programs in prostate cancer.

LHB-Associated Transcriptomic Signature in Prostate Cancer

A multi-dataset transcriptomic study characterized an LHB-correlation-derived gene signature in prostate cancer. Using 501 TCGA-PRAD tumors and external datasets, researchers found that higher signature scores were associated with high-grade tumors, reduced androgen receptor (AR) signaling, and increased cell-cycle and epithelial–mesenchymal transition (EMT) programs. Single-cell analysis showed similar expression patterns in signature-high epithelial states. However, the signature does not measure circulating hormones or establish LHB-dependent biological mechanisms, and further functional validation is required.

PEDIATRICS

A Chinese neonatal cohort study evaluated sepsis incidence and mortality, finding that higher nSOFA scores were associated with increased mortality risk.

Neonatal Sepsis Incidence and nSOFA Mortality Prediction in China

A retrospective observational study in Huai’an, China, evaluated the incidence, mortality, associated risk factors, and predictive performance of the neonatal sequential organ failure assessment (nSOFA) score in neonatal sepsis. Among 7,960 hospitalized neonates from 59,056 live births, 1,225 cases of neonatal sepsis were identified. The nSOFA score demonstrated moderate-to-good performance in predicting sepsis-related mortality, with AUROC values ranging from 0.80 to 0.85. Higher nSOFA scores were associated with increased mortality risk, supporting its potential role in neonatal risk assessment.

OPHTHALMOLOGY

A prospective study evaluated funnel-shaped retinal detachment after open-globe injury, reporting retinal reattachment in all eyes following vitrectomy and improved visual outcomes.

Funnel-Shaped Retinal Detachment After Open-Globe Injury

A prospective study of 1,026 patients with open-globe injury identified 37 eyes with funnel-shaped retinal detachment (FS-RD) and vitreous loss. Most eyes had wounds larger than 10 mm (81.8%), zone II injuries (73%), and total vitreous loss (91.9%). Following vitrectomy, retinal reattachment was achieved in all eyes, although 86.5% remained dependent on silicone oil. BCVA improved from 3.41 ± 0.66 to 2.48 ± 0.86 LogMAR, while the proportion of eyes with no light perception decreased from 54.1% to 18.9%. The findings highlight favorable anatomical outcomes, although larger multicenter studies are needed for validation.

A study found that 29.7% of patients achieved spectacle independence after bilateral enhanced monofocal IOL implantation targeted for distance vision.

Spectacle Independence With Enhanced Monofocal IOLs

A single-center cross-sectional study evaluated spectacle independence in 74 patients who underwent bilateral implantation of enhanced monofocal intraocular lenses targeted for distance vision. One month after surgery, 22 patients (29.7%) reported performing daily activities, including driving, watching television, reading, and smartphone use, without glasses. Patients achieving spectacle independence had significantly lower refractive values in the eye with the lower measurement, suggesting that a low target refraction in one eye may be associated with a greater likelihood of glasses-free vision.

Add-on motugivatrep improved subjective ocular discomfort and ocular surface staining in patients with persistent dry eye symptoms after 2 weeks of treatment.

Motugivatrep for Persistent Symptoms in Dry Eye Disease

A retrospective observational study evaluated add-on 0.3% motugivatrep ophthalmic solution in 23 patients (41 eyes) with persistent dry eye symptoms despite conventional treatment. After 2 weeks, the mean OSDI score significantly decreased from 44.9 ± 25.3 to 25.5 ± 21.9 (mean change: −19.4; 95% CI, −27.3 to −11.6; p = 0.00018). Ocular surface staining also improved, while tear film breakup time showed no significant change. No adverse events were reported during the observation period. Further controlled studies are needed to confirm efficacy and safety.

NEUROLOGY

A case–control study found reduced salivary flow, increased plaque accumulation, and altered tongue microbiota in patients with Parkinson’s disease.

Parkinson’s Disease Linked to Altered Oral Microbiota

A case–control study compared oral health, salivary secretion, and microbial profiles in 20 patients with Parkinson’s disease (PD) and 20 matched controls. Patients with PD had lower unstimulated salivary flow, increased plaque accumulation, and higher prevalence of xerostomia and drooling. Tongue samples showed reduced microbial richness and Shannon diversity, with enrichment of Fusobacterium periodonticum and F. pseudoperiodonticum. These findings suggest that the tongue microbiome may be a potential marker of PD-associated oral dysbiosis, although further research is required.

A study identified socioeconomic disadvantage and logistical barriers associated with missed ophthalmology appointments, with a 16.9% overall no-show rate.

Factors Associated With Ophthalmology Appointment No-Shows

A retrospective mixed-methods study analyzed 62,678 adult ophthalmology appointments at New York Eye and Ear Infirmary of Mount Sinai during 2025. The overall no-show rate was 16.9%. Public insurance or self-pay status, greater neighborhood social vulnerability, afternoon appointments, younger age, and selected ophthalmic subspecialties were associated with higher odds of missed appointments. Follow-up calls with 67 glaucoma patients identified forgetfulness, health or family emergencies, and scheduling conflicts as common reasons for nonattendance. The findings support targeted interventions addressing socioeconomic and logistical barriers.

GYNAECOLOGY - IVF

A multicenter study found substantially higher risks of pre-eclampsia before 34 weeks in non-reduced triplet pregnancies compared with twin and singleton pregnancies.

Pre-eclampsia Risk in Triplet Pregnancies With and Without Reduction

A retrospective multicenter study evaluated pre-eclampsia (PE) risk in 1,020 triplet pregnancies, including 456 non-reduced pregnancies, 468 reduced to twins, and 96 reduced to singletons. Compared with singleton pregnancies, the risk of PE with delivery before 34 weeks was approximately 21-fold higher in non-reduced triplets and 12-fold higher in triplets reduced to twins. Triplets reduced to singletons showed a risk comparable to singleton pregnancies. Compared with twins, the risk was approximately 8-fold higher in non-reduced triplets and 4-fold higher in triplets reduced to twins. These findings highlight differences in early-onset PE risk according to pregnancy type and reduction status.

The PART trial compares partial prostate ablation with radical treatment for localized intermediate-risk prostate cancer, assessing oncological outcomes and quality of life.

PART Trial: Partial Prostate Ablation vs Radical Treatment for Prostate Cancer

The UK-wide PART randomized controlled trial evaluates whether partial prostate ablation (PA), using high-intensity focused ultrasound (HIFU) or irreversible electroporation (IRE), provides non-inferior oncological treatment success compared with radical treatment (RT) for localized intermediate-risk prostate cancer. The study will compare treatment effectiveness, urinary and sexual side effects, health-related quality of life, serious adverse events and cost-effectiveness. The trial aims to generate prospective evidence to support treatment decision-making and evaluate whether partial ablation can offer an alternative to radical treatment.

PULMONOLOGY

Study identifies nasal polyposis and earlier asthma onset as predictors of response to IL-5 inhibitors in severe eosinophilic asthma.

IL-5 Inhibitors in Severe Eosinophilic Asthma: Predictors of Treatment Response

A retrospective cohort study evaluated real-world clinical and phenotypic predictors of treatment response to interleukin-5 (IL-5) pathway inhibitors in adults with severe eosinophilic asthma. The study included 128 patients treated with mepolizumab or benralizumab for at least 12 months between June 2019 and June 2025. Nasal polyposis and earlier age at asthma onset were independently associated with treatment response, while obesity and total IgE levels were not independently associated with response. The findings support phenotype-oriented approaches to biologic selection and personalized asthma management.

Study examines associations between different types of screen time and developmental outcomes among children aged 0–6 years in Thailand.

Screen Time and Child Development: Associations with Developmental Delays in Thailand

A cross-sectional study investigated the associations between different types of screen time and developmental outcomes among children aged 0–6 years in Thailand. The study included 465 caregiver–child pairs, with 78.9% of children exceeding recommended screen time limits. Higher total and recreational screen time were associated with increased odds of suspected developmental delays, particularly receptive language and gross motor delays. Educational and communication-based screen time were associated with lower odds of delays in selected developmental domains. The findings highlight the importance of limiting recreational screen exposure and encouraging age-appropriate, interactive digital activities.

DENTISTRY

Randomized clinical trial compares 24-month partial pulpotomy outcomes using White MTA Angelus and NeoPutty in mature mandibular molars.

Partial Pulpotomy with MTA and NeoPutty: 24-Month Clinical Outcomes

A randomized clinical trial compared the clinical and radiographic outcomes of partial pulpotomy using White MTA Angelus and NeoPutty in mature mandibular molars with deep carious lesions and varying degrees of pulpitis. The study included 139 teeth, all of which completed the 24-month follow-up. Success rates were 90.6% for White MTA Angelus and 92.0% for NeoPutty, with no statistically significant difference between the groups. Haemostasis time was significantly associated with treatment outcome, whereas preoperative pulpal diagnosis was not.

Systematic review evaluates telemedicine-based parent training and its effects on communication, adaptive behaviour and caregiver well-being in autism.

Telemedicine Parent Training for Autism: Effects on Children and Caregivers

A systematic review evaluated the effectiveness of telemedicine-based parent training interventions for children with autism spectrum disorder (ASD) and their caregivers. Nineteen studies involving 616 children were included. Telemedicine interventions, including real-time video coaching, self-guided online modules and hybrid models, were associated with reduced parenting stress, improved parental self-efficacy and enhanced caregiver competence. Improvements were also observed in children's communication abilities, interaction and adaptive behaviours. The findings highlight telemedicine as a potential approach to expanding access to professional support, particularly for families in remote areas.

FDA Highlights 2025 Drug ...

FDA Highlights 2025 Drug Approvals with Breakthroughs in Metabolic and GI Disorders