The therapeutic landscape of hemophilia, a rare inherited bleeding disorder, has rapidly evolved, introducing several novel modalities: extended half-life (EHL) coagulation factor concentrates, non-factor therapies, hemostasis-rebalancing agents, and gene therapy. These advances have improved clinical outcomes by reducing bleeding and treatment burden. However, individualizing treatment in routine practice has become more complex. This narrative review aims to provide a practical summary of available treatment options for pharmacologists, policymakers, and other readers who are not hemophilia specialists. A practical decision framework integrates available data with the clinical indication (regular prophylaxis, management of breakthrough bleeding, and perioperative care), patient-specific modifiers (age, lifestyle, inhibitor status, venous access, joint phenotype, comorbidities, and thrombosis risk). Drug therapies are discussed according to clinical evidence, including pivotal phase 3 trials, long-term extension studies, real-world data, and late-stage development programs. Factor replacement remains critical for hemostatic control during acute bleeding, surgery, and other interventions where predictable and measurable exposure is needed, whereas non-factor and rebalancing therapies provide low-burden prophylactic alternatives with distinct safety profiles and monitoring challenges. Direct comparisons between treatment classes are limited, making treatment individualization essential. Gene therapy has demonstrated more durable outcomes in hemophilia B than in hemophilia A. This review supports shared decision-making and individualized treatment selection in modern hemophilia care. While clinical trial evidence is limited by the scarcity of head-to-head comparisons and incomplete integration of real-world determinants, registry data collection will be essential.

Camizestrant (Etcamah[®]) is an orally available, potent selective estrogen receptor degrader (SERD) and complete estrogen receptor (ER) α antagonist being developed by AstraZeneca for the treatment of breast cancer. It received its first approval in the United Arab Emirates (UAE) in May 2025. At the time of writing, the drug had been subsequently approved in Saudi Arabia, Japan, the EU, Canada, the UK and the USA. In patients with ER-positive and human epidermal growth factor receptor 2 (HER2)-negative breast cancer with a detectable ESR1 mutation, switching to camizestrant while continuing the same CDK4/6 inhibitor significantly prolonged progression-free survival in comparison with continuing treatment with an aromatase inhibitor in combination with a CDK4/6 inhibitor. This article summarizes the milestones in the development of camizestrant leading to this first approval for the treatment of adults with locally advanced or metastatic hormone receptor positive (HR+), HER2-negative breast cancer with an ESR1 gene mutation that emerges during first-line hormonal therapy, in combination with CDK4/6 inhibitors.

Irritable bowel syndrome with constipation is a prevalent disorder of gut-brain interaction in children and adolescents, associated with chronic abdominal pain, altered bowel habits, reduced quality of life, and a significant healthcare burden. Despite a wide range of therapeutic approaches, treatment responses remain suboptimal, highlighting the need for effective, evidence-based pharmacological options. This review summarizes current and emerging pharmacological therapies for pediatric irritable bowel syndrome with constipation, with a focus on their mechanisms of action, efficacy, and safety. Traditional treatments such as dietary fiber, probiotics, antispasmodics, laxatives, prokinetics, antidepressants, antibiotics, and antihistamines are reviewed, emphasizing the limited and often inconsistent evidence supporting their use for pain relief and overall symptom improvement. Particular attention is given to newer agents approved for adults with irritable bowel syndrome with constipation, including secretagogues (linaclotide, plecanatide, and lubiprostone) and the retainagogue tenapanor, with a detailed discussion of their pharmacological properties and clinical trial data. Linaclotide is currently the only medication approved by the U.S. Food and Drug Administration for the treatment of irritable bowel syndrome with constipation in children aged 7 years or older. While other novel agents have shown promise in adults, pediatric data remain limited or unavailable. Overall, although pharmacological treatment options for pediatric irritable bowel syndrome with constipation are expanding, high-quality, pediatric specific clinical trials are still needed to guide safe and effective management.

Gonorrhoea, caused by Neisseria gonorrhoeae, is a common bacterial sexually transmitted infection and a major global health burden, with more than 80 million new cases estimated annually. There is currently no licensed vaccine, and increasing antimicrobial resistance has renewed the urgency of vaccine development. However, vaccines licensed to prevent invasive meningococcal disease, caused by the closely related bacterium Neisseria meningitidis, may provide cross-protection against N. gonorrhoeae. Observational studies have associated outer membrane vesicle-containing serogroup B meningococcal vaccines with reduced gonorrhoea rates, with estimated effectiveness of approximately 30%. The four-component meningococcal group B vaccine (4CMenB) contains antigens with gonococcal homologues and induces cross-reactive immune responses in both humans and animal models. These findings have supported the implementation of targeted 4CMenB immunisation programmes for people at high risk of gonorrhoea in the UK and Galicia, Spain. However, the recently reported GoGoVax trial and other randomised controlled trials in men who have sex with men (MSM) at high risk of infection have not demonstrated protection against gonorrhoea. Differences in study design, populations, exposure intensity, endpoints and residual confounding may explain the divergence between observational and randomised findings. Ongoing trials in other populations, together with post-implementation surveillance in the UK, will provide important evidence regarding the effectiveness of 4CMenB in different settings. Several gonococcus-specific vaccine candidates are also advancing through preclinical and clinical development. Future progress will benefit from validated correlates of protection, improved understanding of protective mucosal immunity across different anatomical sites and evaluation of vaccine efficacy across diverse populations and exposure settings.

Chronic inflammatory pouch disorders may affect up to 20% of patients within 5 years after ileal pouch-anal anastomosis and remains a major therapeutic challenge. Within this spectrum, chronic antibiotic-refractory pouchitis is associated with impaired quality of life, long-term pouch-related complications, and potential risk for pouch failure and excision. Current recommendations support the use of probiotics for primary and secondary prevention in selected patients. Increasingly, targeted advanced therapies, including biologics and small molecules approved for the treatment of ulcerative colitis and Crohn's disease, are being used to reduce antibiotic dependence and maintain disease control in patients with pouch inflammation. Vedolizumab is the only advanced therapy approved in Europe for chronic pouchitis, whereas evidence for tumor necrosis factor (TNF) antagonists, ustekinumab, and newer agents, such as interleukin (IL)-23p19 antagonists and Janus kinase (JAK) inhibitors is largely based on observational data from clinical practice and real-world experience. Fecal microbiota transplantation is biologically attractive but remains investigational, with early studies showing inconsistent efficacy. Dietary patterns may modify pouch inflammation; however, most evidence is observational. Overall, current management strategies for chronic pouchitis are limited by incomplete understanding of disease pathogenesis, and by persistent methodological gaps, including poorly standardized treatment outcomes and the lack of validated treat-to-target strategies for this population. Well designed prospective and controlled studies are needed to define appropriate antibiotic-sparing strategies, optimal treatment positioning, and long-term durability of response.

Metabolic dysfunction-associated steatotic liver disease (MASLD), formerly known as non-alcoholic fatty liver disease (NAFLD), is a prevalent condition affecting 25-30% of the global population and is closely linked to obesity, type 2 diabetes, and other cardiometabolic risk factors. Although liver biopsy remains the gold standard for diagnosis, its invasive nature has prompted a shift toward non-invasive biomarkers for disease assessment, staging, and monitoring. Multiple non-invasive biomarkers have been studied or are currently under investigation to assess both fibrosis and steatosis among patients with MASLD. This review aims to summarize the current literature on emerging and established biomarkers for MASLD, focusing on their roles in detecting inflammation, fibrosis, metabolic dysfunction, and steatosis, including imaging-based surrogate endpoints and composite biomarker panels.

Doravirine/islatravir is an oral two-drug fixed-dose combination of doravirine [a non-nucleoside reverse transcriptase inhibitor (NNRTI)] and islatravir [a nucleoside reverse transcriptase translocation inhibitor (NRTTI)]. Developed by Merck & Co., Inc., Rahway, NJ, USA, doravirine/islatravir [IDVYNSO[®] イドビンソ[®] (Japan); IDVYNSO™ (USA); IDVYNSO[®] (Canada)] was first approved on 6 March 2026 in Japan and subsequently approved on 21 April 2026 in the USA and 3 June 2026 in Canada for the treatment of HIV-1 infection in adults who are virologically suppressed (HIV-1 RNA < 50 copies/mL). Clinical development is underway in several other countries worldwide. This article summarizes the milestones in the development of doravirine/islatravir leading to these first approvals.

Anbenitamab (Ennituo[®]), a bispecific IgG monoclonal antibody targeting human epidermal growth factor receptor 2 (HER2), is being developed by Alphamab Oncology for the treatment of various solid tumours (including gastric cancer and breast cancer). Anbenitamab received its first approval in China on 27 May 2026. Anbenitamab, in combination with chemotherapy, is approved for the treatment of adult patients with locally advanced or metastatic HER2-positive gastric or gastroesophageal junction (GEJ) adenocarcinoma who have previously received at least one trastuzumab-containing therapy. This article summarizes the milestones in the development of anbenitamab leading to this first approval for gastric or GEJ cancer.

To summarise the literature regarding the safety of short-term use of the non-steroidal anti-inflammatory drug (NSAID) celecoxib.

Atacicept (atacicept-vymj; TRUTAKNA[™]) is a human recombinant fusion glycoprotein targeting B lymphocyte stimulator [BLyS; also known as B cell-activating factor (BAFF)] and A proliferation-inducing ligand (APRIL) being developed by Vera Therapeutics, under a license from Merck Serono. On 7 July 2026, atacicept received accelerated approval in the USA to reduce proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk for disease progression. In patients with IgAN, atacicept produced sustained reductions in proteinuria across the ORIGIN clinical development program and stabilized estimated glomerular filtration rates through 96 weeks. This article summarizes the milestones in the development of atacicept leading to this first approval for IgAN.

Pivekimab sunirine (pivekimab sunirine-pvzy; DECNUPAZ™) is first-in-class CD123-directed antibody-drug conjugate (ADC) developed by ImmunoGen for the treatment of haematological malignancies, including blastic plasmacytoid dendritic cell neoplasm (BPDCN) and acute myeloid leukaemia (AML). CD123 is an attractive therapeutic target because it is highly expressed on malignant cells and leukaemic stem cells, but has limited expression on normal haematopoietic stem cells. On 27 May 2026, pivekimab sunirine received its first approval in the USA for the treatment of adult patients with BPDCN. This article summarizes the milestones in the development of pivekimab sunirine leading to this first approval.