Drugs[JOURNAL]
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200 papers
Antibody-Drug Conjugates in Non-small Cell Lung Cancer.
Zullo L, Bortolot M, Ogliari FR … +6 more , Saw SPL, van Geel R, Ter Heine R, Sadowska A, Remon J, Hendriks L
Drugs · 2026 Jul · PMID 42151668 · Full text
Antibody-drug conjugates (ADCs) are complex molecules composed of a monoclonal antibody, a linker and a cytotoxic payload. Their design enables the selective delivery of cytotoxic agents to tumoral cells through antibody...
Antibody-drug conjugates (ADCs) are complex molecules composed of a monoclonal antibody, a linker and a cytotoxic payload. Their design enables the selective delivery of cytotoxic agents to tumoral cells through antibody binding to a tumor-expressed antigen, followed by internalization, intracellular degradation and payload release, ultimately enabling the cytotoxic drug to exert its antitumor activity. ADCs have been evaluated in phase II and III trials in previously treated advanced non-small cell lung cancer (NSCLC), both in oncogene-addicted and in non-oncogene-addicted tumors, addressing resistance to standard therapies. Ongoing clinical trials are now expanding their use both in the first-line setting for advanced disease and in earlier disease stages. This narrative review summarizes the currently available data for ADC treatment in NSCLC, highlighting the need for improved patient selection to maximize benefit while limiting toxicity, incorporating clinical characteristics, pharmacogenomics and optimal treatment sequencing in the equation. Moreover, the understanding of resistance mechanisms and the development and validation of predictive biomarkers will be of utmost relevance to inform clinical practice.
Narsoplimab: First Approval.
Shirley M
Drugs · 2026 Jul · PMID 42144536 · Publisher ↗
Narsoplimab (narsoplimab-wuug; YARTEMLEA) is a recombinant human immunoglobulin G4 monoclonal antibody targeted against mannan-binding lectin-associated serine protease 2 (MASP-2) being developed by Omeros Corporation in...
Narsoplimab (narsoplimab-wuug; YARTEMLEA) is a recombinant human immunoglobulin G4 monoclonal antibody targeted against mannan-binding lectin-associated serine protease 2 (MASP-2) being developed by Omeros Corporation initially for the treatment of haematopoietic stem cell transplantation (HCT)-associated thrombotic microangiopathy (TA-TMA). MASP-2 inhibition by narsoplimab is thought to provide benefit in patients with TA-TMA by decreasing or preventing lectin complement pathway-mediated cellular injury. In December 2025, narsoplimab received its first approval, in the USA, for the treatment of adult and paediatric patients aged ≥ 2 years with TA-TMA. Additionally, a Marketing Authorisation Application for narsoplimab for the treatment of TA-TMA is currently under regulatory review in the EU. This article summarises the milestones in the development of narsoplimab leading to this first approval for TA-TMA.
Reframing the High-density Lipoprotein (HDL) Hypothesis in Light of Clinical Trials of Cholesterol Ester Transfer Protein (CETP) Inhibitors.
Chehade MJ, Haas MJ, Mooradian AD
Drugs · 2026 Jul · PMID 42142223 · Publisher ↗
High-density lipoprotein cholesterol (HDL-C) has been inversely associated with atherosclerotic cardiovascular disease (ASCVD) risk. This relationship is the foundation of the "HDL hypothesis," which states that increasi...
High-density lipoprotein cholesterol (HDL-C) has been inversely associated with atherosclerotic cardiovascular disease (ASCVD) risk. This relationship is the foundation of the "HDL hypothesis," which states that increasing plasma HDL-C levels would result in a decrease in cardiovascular events. The recent surge in clinical testing of cholesteryl ester transfer protein (CETP) inhibitors provides valuable data for this hypothesis. The CETP inhibitors increase plasma HDL-C along with variable effects on low-density lipoprotein cholesterol (LDL-C) and apolipoprotein B (apoB). The clinical outcomes of randomized controlled trials were heterogeneous, with several studies showing either neutral or adverse clinical outcomes despite robust increases in HDL-C values. These observations along with Mendelian randomization studies suggest that low HDL-C is a risk marker, not a causal mediator of ASCVD. New evidence points to HDL functionality, specifically cholesterol efflux potential and particle composition, instead of HDL-C concentration as a more relevant determinant of ASCVD risk. The favorable clinical outcomes observed with certain CETP inhibitors are more consistently explained by reductions in apoB-containing lipoproteins (including LDL-C and non-HDL-C), rather than increases in HDL-C itself. This distinction reframes the HDL hypothesis where HDL-C is merely a biomarker of ASCVD while apoB-containing lipoproteins are the more plausible causal treatment pathway. These findings have shifted the focus of the HDL hypothesis from the "quantity" to the "quality" of HDL.
Emerging Cardiorenal Protective Therapies in Lupus Nephritis.
Capon IM, Sandino-Pérez J, Galindo M … +1 more , Morales E
Drugs · 2026 May · PMID 42133281 · Publisher ↗
Lupus nephritis (LN) remains a major cause of morbidity and progression to chronic kidney disease in systemic lupus erythematosus. Beyond immunosuppression, emerging evidence highlights the critical role of nephron-prote...
Lupus nephritis (LN) remains a major cause of morbidity and progression to chronic kidney disease in systemic lupus erythematosus. Beyond immunosuppression, emerging evidence highlights the critical role of nephron-protective therapies in mitigating chronic damage and preserving renal function. This narrative review included a structured literature search in PubMed, EMBASE, and Web of Science for new cardio-renoprotective therapies in LN. Agents targeting metabolic, hemodynamic, and inflammatory pathways, including sodium-glucose cotransporter 2 inhibitors (SGLT2i), glucagon-like peptide-1 receptor agonists (GLP-1 RA), and nonsteroidal mineralocorticoid receptor antagonists (ns-MRAs), have shown renal and cardiovascular benefits in proteinuric and diabetic populations, and recent data suggest potential efficacy in LN. These therapies reduce intraglomerular pressure, proteinuria, oxidative stress, and tubular inflammation, complementing the immunomodulatory effects of standard regimens. In addition, endothelin receptor antagonists and novel anti-fibrotic or metabolic modulators are under investigation for synergistic cardiorenal protection. Early integration of these agents may delay progression to end-stage kidney disease, improve systemic vascular health, and reduce long-term treatment burden. Future randomized trials specifically designed in LN cohorts are warranted to define optimal timing, combinations, and safety in the context of immunosuppression. Nephron-protective therapy represents a paradigm shift in LN management, from solely controlling autoimmunity toward preserving long-term renal structure and function through multi-system, cardio-renal protection.
Diagnosis and Medication Treatment of Schizophrenia in Adolescents.
Findling RL, Shah D, Prajapati P … +3 more , Cordrey E, Nichols M, Stepanova E
Drugs · 2026 Jul · PMID 42129067 · Full text
Schizophrenia is a chronic mental health condition that most commonly develops in the third decade of life, but may emerge during adolescence, although in rare cases it can also present in childhood. Pre-adult onset is a...
Schizophrenia is a chronic mental health condition that most commonly develops in the third decade of life, but may emerge during adolescence, although in rare cases it can also present in childhood. Pre-adult onset is associated with substantial morbidity, functional impairment, and long-term disability. Accurately diagnosing schizophrenia in youth is particularly challenging, as its symptoms frequently overlap with or are misattributed to other psychiatric or developmental conditions. Conversely, misdiagnosis can also occur when psychotic symptoms are present in patients suffering from other conditions. Establishing an accurate and timely diagnosis is therefore critical to prevent errors in treatment and to optimize prognosis and functional outcomes. Antipsychotic medications are first-line interventions for schizophrenia in youth, with second-generation antipsychotics (SGAs) having the strongest evidence base in this population. Randomized controlled trials and open-label studies support the efficacy of risperidone, aripiprazole, olanzapine, quetiapine, paliperidone, lurasidone, and brexpiprazole in reducing psychotic symptoms in adolescents. In contrast, ziprasidone and asenapine have not demonstrated clear benefit in treatment of schizophrenia in youth. Similar to studies in adults, evidence from pediatric clinical trials suggests that clozapine remains the most effective option for treatment-resistant adolescent schizophrenia, although its use requires close monitoring for hematologic and other adverse effects. While SGAs are generally favored over first-generation antipsychotics (FGAs) in youth because of their reduced propensity for causing extrapyramidal side effects as well as a stronger evidence base to support their use, their safety profiles require careful consideration. Weight gain, metabolic syndrome, sedation, extrapyramidal symptoms, and endocrine effects have been reported in youth with schizophrenia. Such side effects can significantly impact adherence and long-term physical health, necessitating the need for ongoing monitoring and thoughtful agent selection tailored to each patient's individual clinical profile and risk factors. Overall, current evidence supports SGAs as the foundation of psychopharmacologic treatment for schizophrenia in pediatric-aged patients, with clozapine reserved for refractory cases. However, the relative paucity of pediatric-specific data compared with adult populations underscores the importance of continued research to guide clinical practice. In the meantime, accurate diagnosis, early intervention, and vigilant monitoring of both therapeutic response and side effects remain essential to optimizing outcomes for youth with schizophrenia.
Bruton's Tyrosine Kinase Inhibitors in Multiple Sclerosis.
Lambe J, Fox RJ
Drugs · 2026 Jul · PMID 42126690 · Full text
Multiple sclerosis (MS) is a chronic, inflammatory disorder of the central nervous system (CNS) characterized by overlapping relapsing and progressive pathologies. Although they exist along a continuum, pathology in rela...
Multiple sclerosis (MS) is a chronic, inflammatory disorder of the central nervous system (CNS) characterized by overlapping relapsing and progressive pathologies. Although they exist along a continuum, pathology in relapsing MS (RMS) is primarily driven by the peripheral adaptive immune system, while progressive MS (PMS) pathology is underpinned by innate immune activity and other pathologic processes compartmentalized within the CNS. While currently approved therapies are highly effective in targeting aspects underpinning relapsing pathology, they have limited efficacy in PMS due to either inability to cross the blood brain barrier (BBB) or inability to modulate pathologies driving PMS. Bruton's tyrosine kinase (BTK) inhibitors are an emerging class of oral agents that represent a novel approach to MS treatment. These agents target BTK, an enzyme which plays a pivotal role in both adaptive and innate immune signaling. As small molecules, some BTK inhibitors can cross the BBB at biologically relevant concentrations. Therefore, BTK inhibitors may potentially modulate both relapsing and progressive MS pathology. Data from Phase 2 and Phase 3 trials have been promising in this regard. The unique pharmacological profile of each BTK inhibitor may underpin observed differences in efficacy and safety outcomes to date. In particular, tolebrutinib has demonstrated efficacy against disability progression in non-relapsing secondary progressive MS, while fenebrutinib has recently shown promise in both relapsing and primary progressive MS. However, adverse events include elevated liver enzymes, which can reach life-threatening levels. This review highlights the role of BTK in immune signaling and the rationale for BTK inhibition in MS, discusses the evolution of BTK inhibitors for MS and other indications, summarizes findings from Phase 2 and Phase 3 trials in RMS and PMS, and explores practical questions around the potential use of BTK inhibitors in real-world practice.
Incretin-Based Anti-obesity Medications in Polycystic Ovary Syndrome: The Evidence Map.
Jensterle M, Janez A
Drugs · 2026 Jul · PMID 42106472 · Publisher ↗
Polycystic ovary syndrome (PCOS) is a common, heterogeneous condition that is tightly linked to obesity, visceral adiposity and insulin resistance. Lifestyle intervention and off-label use of metformin provide only modes...
Polycystic ovary syndrome (PCOS) is a common, heterogeneous condition that is tightly linked to obesity, visceral adiposity and insulin resistance. Lifestyle intervention and off-label use of metformin provide only modest and unsustained weight loss, insufficient to reverse obesity-driven pathophysiology in most women with PCOS and obesity. Incretin-based anti-obesity medications, including glucagon-like peptide-1 receptor agonists (GLP-1RAs) and dual glucose-dependent insulinotropic polypeptide/glucagon-like peptide-1 receptor agonists (dual GIP/GLP-1RAs), offer a biologically plausible way to target adipose dysfunction, hyperinsulinemia and chronic inflammation that drive PCOS in a large subset of patients. In this narrative, product-segmented review, we map the evidence for liraglutide, semaglutide and tirzepatide in PCOS across mechanistic, clinical, and safety domains, and highlight key evidence gaps that limit current practice. Liraglutide has the densest PCOS-specific evidence, demonstrating reproducible weight loss across small and heterogeneous cohorts, reductions in visceral adiposity and hepatic fat, improved glycemia and inflammatory markers, and early signals for androgen and fertility benefits in selected phenotypes. Semaglutide data remain sparse but conceptually rich, demonstrating weight-loss efficacy and mechanistic insights, alongside preliminary signals of increased likelihood of natural conception. Tirzepatide currently has no PCOS-specific evidence and cannot be recommended beyond extrapolation from obesity and diabetes trials. Across all agents, reproductive outcomes, periconceptional and pregnancy safety, adolescent use, long-term cardiovascular-kidney-metabolic trajectories, obstructive sleep apnea, musculoskeletal health and phenotype-stratified response remain major evidence gaps. We propose a multidimensional, metabolic high-risk PCOS phenotype as the most rational current target for incretin therapy, while emphasizing that well-designed, PCOS-specific trials are essential before these drugs can be viewed as PCOS-modifying therapies rather than powerful, but still adjunctive, weight-loss agents.
Large Vessel Vasculitis: Recent Advances in Pathophysiology and Targeted Therapies.
Reisch M, Thiel J, Bosch P
Drugs · 2026 Jun · PMID 42081084 · Full text
Large vessel vasculitides (LVV), including giant cell arteritis (GCA) and Takayasu arteritis (TAK), share common features such as inflammation of large sized arteries but differ in several key aspects, including age of o...
Large vessel vasculitides (LVV), including giant cell arteritis (GCA) and Takayasu arteritis (TAK), share common features such as inflammation of large sized arteries but differ in several key aspects, including age of onset and pathogenic mechanism. This narrative review gives an update of recent insights into pathogenesis of GCA and TAK, and discusses emerging targeted therapies based on these insights. It highlights omics-based signatures, ULK3 and SLAMF7 in GCA, EGR1 in TAK, alongside genetic and somatic risk factors such as clonal haematopoiesis (DNMT3A/TET2) linked to relapse and ischaemic vision loss in GCA, and the IL6R-p.Asp358Ala variant as a predictor of reduced interleukin (IL)-6 receptor blockade response. Common mechanisms include CD4⁺ T-cell, monocyte/macrophage, and B-cell infiltration with activation of IL-6, JAK/STAT/interferon, and IL-17 pathways. Giant cell arteritis is characterised by GM-CSF-driven macrophages and disrupted programmed cell death (PD)-1/PD-L1 checkpoint regulation, while TAK shows dominance of CD8⁺ T cells and tumour necrosis factor (TNF)-α signalling. Interleukin-6 receptor inhibitors (e.g., tocilizumab) show robust efficacy in GCA but with notable non-responders; the JAK inhibitor upadacitinib demonstrated efficacy in a Phase III study, whereas IL-17 blockade (secukinumab) yielded inconsistent results. In TAK, TNF inhibitors and tocilizumab are comparably effective; early data suggest Janus kinases (JAK) inhibitors promote remission, imaging improvement, and glucocorticoid sparing. Mavrilimumab (GM-CSF receptor blockade) is promising in GCA. Recent studies have increasingly focused on short-term glucocorticoid therapy in combination with biologic agents. Advances in biomarker research, including investigation of the IL-6 receptor and IL-17A gene polymorphisms, may enable more targeted therapeutic strategies.
Advances in Immune-Based Approaches for the Cure of HIV Infection.
Wagner JA, Sandel DA, Deitchman AN … +3 more , Rutishauser RL, Deeks SG, Peluso MJ
Drugs · 2026 Jun · PMID 42068526 · Full text
Despite significant advances in antiretroviral therapy, the need for a cure for HIV persists because of factors such as long-term antiretroviral therapy-related comorbidities, disease stigma, and inequities in access to...
Despite significant advances in antiretroviral therapy, the need for a cure for HIV persists because of factors such as long-term antiretroviral therapy-related comorbidities, disease stigma, and inequities in access to care. Most cure efforts focus on inducing durable HIV remission (antiretroviral therapy-free viral control) either by augmenting immune function or reducing the HIV reservoir. In this review, we highlight immune-based cure interventions currently under investigation with a particular focus on those that have demonstrated the ability to induce durable HIV remission after analytic treatment interruption, here termed "post-intervention control". While current cure interventions are generally complex, expensive, and not easily scalable, they provide critical "proof of principle" that a cure for HIV is possible. Continuing to make studies of HIV cure a funding priority is important, we believe, as continued optimization of cure interventions should eventually lead to a cure that is simple, safe, effective, affordable, and scalable. In addition, we highlight critical features in clinical trial design and pharmacokinetics/pharmacodynamics that should be considered prior to clinical trial implementation.
Etripamil: First Approval.
Brown MB
Drugs · 2026 Jul · PMID 42068525 · Publisher ↗
Etripamil (CARDAMYST) is an intranasal L-type calcium channel blocker (CCB) developed by Milestone Pharmaceuticals for the treatment of symptomatic paroxysmal supraventricular tachycardia (PSVT) and is under investigatio...
Etripamil (CARDAMYST) is an intranasal L-type calcium channel blocker (CCB) developed by Milestone Pharmaceuticals for the treatment of symptomatic paroxysmal supraventricular tachycardia (PSVT) and is under investigation for atrial fibrillation with rapid ventricular rate (AF-RVR). It received its first approval on the 12 December 2025 in the USA for the conversion of acute symptomatic PSVT episodes to sinus rhythm in adults. Additional global regulatory filings of etripamil for PSVT in adults, a phase II trial of etripamil in pediatrics, and a phase III trial for AF-RVR are in progress. This article summarizes the milestones in the development of etripamil leading to this first approval for PSVT.
Sonrotoclax: First Approval.
Blair HA
Drugs · 2026 Jul · PMID 42047966 · Publisher ↗
Sonrotoclax (Baiyueda), a small-molecule B-cell lymphoma-2 (BCL-2) inhibitor, is being developed by BeOne Medicines for the treatment of various B-cell malignancies. On 6 January 2026, sonrotoclax received conditional ap...
Sonrotoclax (Baiyueda), a small-molecule B-cell lymphoma-2 (BCL-2) inhibitor, is being developed by BeOne Medicines for the treatment of various B-cell malignancies. On 6 January 2026, sonrotoclax received conditional approval in China for adult patients with chronic lymphocytic leukaemia (CLL)/small lymphocytic leukaemia (SLL) who have previously received at least one systemic therapy, including a Bruton's tyrosine kinase (BTK) inhibitor, and adult patients with relapsed or refractory mantle cell lymphoma (MCL) who have previously received at least two systemic therapies, including a BTK inhibitor. This article summarizes the milestones in the development of sonrotoclax leading to this first approval for the second-line treatment of relapsed/refractory CLL/SLL and MCL.
Malaria Prevention: Progress to Date.
Grobusch MP, Schnyder JL, Schlagenhauf P … +2 more , de Jong HK, Hanscheid T
Drugs · 2026 Jul · PMID 42045630 · Full text
Chemoprophylaxis (in travellers) and seasonal chemoprophylaxis and preventive treatment (in endemic areas) are important but not exclusive elements of malaria prevention, which hinges on vector repelling in travellers an...
Chemoprophylaxis (in travellers) and seasonal chemoprophylaxis and preventive treatment (in endemic areas) are important but not exclusive elements of malaria prevention, which hinges on vector repelling in travellers and more comprehensive vector control measures in endemic areas. In malaria-endemic countries, a comprehensive strategy combining drug-based prevention, vaccines and vector control is essential to reduce disease burden. Chemoprophylaxis in endemic settings primarily involves intermittent preventive treatment in vulnerable groups and mass drug administration in communities, rather than continuous daily chemoprophylaxis (as recommended for non-immune travellers). Vaccination has become a groundbreaking addition to the malaria control portfolio in endemic countries. The World Health Organization-endorsed RTS,S/AS01 (Mosquirix) and R21/Matrix-M vaccines rolled out in many highly malaria-endemic sub-Saharan African countries, in addition to other malaria control tools, provide overall moderate protection in young children but significantly reduce severe disease and death, and are considered safe. For travellers, malaria vaccines are not available to date. Effort is being put into the development of monoclonal antibodies against malaria as a preventive treatment strategy both to provide protection for the immunocompromised or unvaccinated high-risk individuals before exposure and to disrupt seasonal malaria transmission with a single application. The evolution of malaria preventive tools is a dynamic process, with numerous novel developments on the horizon. For travel medicine indications, further harmonisation of recommendations on the one hand, but also more sophisticated personalisation of recommendations, is envisaged.
Pixavir Marboxil: First Approval.
Kang C
Drugs · 2026 Jul · PMID 42033572 · Publisher ↗
Pixavir marboxil (Yilikang; ) is an oral cap-snatching endonuclease inhibitor being developed by TaiGen Biotechnology for the treatment of influenza virus infections. Pixavir marboxil recently received approval in China...
Pixavir marboxil (Yilikang; ) is an oral cap-snatching endonuclease inhibitor being developed by TaiGen Biotechnology for the treatment of influenza virus infections. Pixavir marboxil recently received approval in China for the treatment of uncomplicated influenza A and B in previously healthy adults and adolescents aged ≥ 12 years. This article summarizes the milestones in the development of pixavir marboxil leading to this first approval for uncomplicated influenza A and B infections.
Antifungal Treatment Regimens as Primary Therapy for Invasive Aspergillosis: A Systematic Review and Network Meta-analysis.
Gu Q, Chen Y, Ding P … +3 more , He J, Zhang H, Chen D
Drugs · 2026 Jun · PMID 42012594 · Publisher ↗
BACKGROUND: Invasive aspergillosis (IA) is an invasive fungal disease associated with high mortality. Triazoles are the primary therapy for IA, while amphotericin B, echinocandins, and antifungal combined therapy are com...
BACKGROUND: Invasive aspergillosis (IA) is an invasive fungal disease associated with high mortality. Triazoles are the primary therapy for IA, while amphotericin B, echinocandins, and antifungal combined therapy are commonly considered as alternative treatment options although their efficacy and safety remain subjects of debate. We employed Bayesian network meta-analysis (NMA) to synthesize evidence and evaluate the efficacy and safety of different primary antifungal treatment regimens for IA. METHODS: A literature search was done in PubMed, EMBASE, Cochrane, and Web of Science databases for clinical studies on primary treatment for patients with IA published before 28 March 2024. We included randomized controlled trials (RCTs) and cohort studies involving patients with proven or probable IA who underwent primary treatment. The primary treatment regimens comprised different types or dosages of monotherapy or combination therapy involving triazoles, echinocandins, and amphotericin B. The primary outcome was cumulative all-cause mortality. Secondary outcomes included all-cause mortality at day 42, day 84, or end of treatment (EOT); the overall response at day 42, day 84, or EOT; invasive aspergillosis-associated mortality on day 42, day 84, and EOT; the incidence of adverse events (AEs), serious adverse events (SAEs), and treatment-related AEs; and treatment discontinuation due to AEs. RESULTS: The primary network meta-analysis for cumulative all-cause mortality (5 RCTs, 6 regimens, n = 1293) revealed no statistically significant differences among treatment regimens compared with voriconazole (VOR; surface under the cumulative ranking curve [SUCRA] 56.3%), including VOR combined with anidulafungin (ANI; risk ratio [RR] 0.74, 95% credible interval [CrI] 0.43-1.28; SUCRA 84.3%; moderate confidence), isavuconazole (ISA; RR 0.79, 95% CrI 0.45-1.40; 79.4%; moderate confidence), and posaconazole (POS; RR 1.20, 95% CrI 0.72-2.02; 34.6%; moderate confidence). Sensitivity analyses confirmed the robustness of these findings. Regarding safety, no significant differences were observed in SAEs for ISA (RR 0.91, 95% CrI 0.75-1.11; 85.7%; moderate confidence), POS (RR 1.03, 95% CrI 0.87-1.23; 40.8%; low confidence), and VOR combined with ANI (RR 1.09, 95% CrI 0.88-1.36; 20.8%; moderate confidence) compared with VOR (52.6%). Integrated efficacy-safety analysis suggested a favorable risk-benefit profile for isavuconazole. CONCLUSIONS: ISA, POS, and VOR are the preferred antifungal agents for primary treatment of IA, with ISA demonstrating a higher likelihood of an optimal efficacy-safety balance. The combination of VOR and ANI may be considered for a subset of severe cases. REGISTRATION: PROSPERO identifier number CRD42024561215.
Zoliflodacin: First Approval.
McGuigan A
Drugs · 2026 Jun · PMID 42002647 · Publisher ↗
Zoliflodacin (NUZOLVENCE), a first-in-class, single-dose, oral spiropyrimidinetrione bacterial type II topoisomerase inhibitor, has been developed by Innoviva Specialty Therapeutics in collaboration with the Global Antib...
Zoliflodacin (NUZOLVENCE), a first-in-class, single-dose, oral spiropyrimidinetrione bacterial type II topoisomerase inhibitor, has been developed by Innoviva Specialty Therapeutics in collaboration with the Global Antibiotic Research and Development Partnership (GARDP) and the U.S. National Institute of Allergy and Infectious Diseases (NIAID) for the treatment of uncomplicated urogenital gonorrhea (uUGC). In December 2025, zoliflodacin was approved by the US FDA for the treatment of uUGC due to Neisseria gonorrhoeae in adults and pediatric patients 12 years of age and older weighing at least 35 kg. This article summarizes the milestones in the development of zoliflodacin leading to this first approval for the treatment of uUGC.
The Role of β-Blockers in the Evolving Treatment Landscape of Resistant Hypertension.
Masi S, Pathak A, Bruno RM … +5 more , Brouwers S, Narkiewicz K, Taddei S, Tsioufis K, Kreutz R
Drugs · 2026 Jun · PMID 42002646 · Full text
Resistant hypertension (RHTN) is estimated to affect approximately 15% of all patients with hypertension, although its reported prevalence varies according to the blood pressure (BP) thresholds used for diagnosis, the cr...
Resistant hypertension (RHTN) is estimated to affect approximately 15% of all patients with hypertension, although its reported prevalence varies according to the blood pressure (BP) thresholds used for diagnosis, the criteria applied to exclude secondary or pseudoresistant hypertension, and the characteristics of the study populations. Regardless of its current prevalence, RHTN is expected to be encountered with increasing frequency in routine clinical practice, driven by the rising prevalence of conditions that are directly or indirectly linked with its increased risk, such as obesity, diabetes mellitus, and chronic kidney disease (CKD). Consequently, recent clinical trials and hypertension guidelines have devoted substantial attention to RHTN, also considering its association with an increased risk of cardiovascular events and renal failure. In this narrative review, we summarize mechanistic insights and evidence from randomized clinical trials and real-world studies, as well as recommendations from current international guidelines, to provide an updated perspective on the management of RHTN. Among multiple systems, activation of the sympathetic nervous system (SNS) and the renin-angiotensin-aldosterone system represents a central pathophysiological mechanism in RHTN, promoting sodium retention and vascular dysfunction. β-blockers counteract SNS overactivity, a target mechanism not addressed by other first-line therapies, and have compelling indications in most comorbidities associated with RHTN, including coronary artery disease, heart failure, and atrial fibrillation. They can be used across diverse patient populations, including those with advanced CKD, and their side effects can be readily monitored and managed. Evidence from randomized trials and real-world studies supports their efficacy, tolerability, and safety even in high-risk populations. Emerging strategies, including a quadruple single-pill combination containing a β-blocker, may enhance adherence, optimize BP control, and simplify RHTN management.
Developments in Pharmacotherapy for Acromegaly: Current and Emerging Approaches.
Tritos NA, Biller BMK
Drugs · 2026 Jun · PMID 41964795 · Publisher ↗
Pituitary surgery is the primary therapy for most patients with acromegaly. Medical therapy has an important, albeit adjunctive role in the management of patients with persistent disease after surgery. However, primary m...
Pituitary surgery is the primary therapy for most patients with acromegaly. Medical therapy has an important, albeit adjunctive role in the management of patients with persistent disease after surgery. However, primary medical therapy can be appropriate as an option in select patients. Medical therapies in current use for acromegaly are somatostatin receptor ligands (SRLs) (octreotide long-acting release [LAR], octreotide acetate, lanreotide depot, octreotide subcutaneous (SC) depot, pasireotide LAR, oral octreotide, paltusotine), dopamine agonists (cabergoline) and growth hormone receptor antagonists (pegvisomant). These are often efficacious and generally well tolerated. However, a particular pharmaceutical agent may not meet the needs of individual patients because of intolerance, contraindications to their use, lack of sustained efficacy, or decreased quality of life. Several investigational drugs are in development towards addressing unmet needs of patients with acromegaly, including new formulations of SRLs (lanreotide prolonged-release formulation, Debio 4126, pasireotide SC depot), novel SRLs (somatoprim, HTL0030310), monoclonal antibodies against growth hormone, and new growth hormone receptor antagonists. Current and emerging therapies are offering renewed hope for disease control. More studies including comparator agents, identification of accurate biomarkers and models predictive of clinical effectiveness may further improve the care of patients with acromegaly.
Correction: Avutometinib and Defactinib: First Approval.
Blair HA
Drugs · 2026 May · PMID 41954872 · Publisher ↗
Abstract loading — click title to view on PubMed.
Targeting Inflammation in Bronchiectasis.
Mac Aogáin M, Gilmour A, Chalmers JD … +1 more , Chotirmall SH
Drugs · 2026 Jun · PMID 41954871 · Publisher ↗
Bronchiectasis is defined by chronic infection, dysregulated inflammation and impaired mucociliary clearance underpinning progressive structural lung injury. While airway infection remains a clinical hallmark, numerous s...
Bronchiectasis is defined by chronic infection, dysregulated inflammation and impaired mucociliary clearance underpinning progressive structural lung injury. While airway infection remains a clinical hallmark, numerous studies demonstrate that excessive neutrophil-dominated inflammation is a key determinant of disease severity, exacerbation risk and quality of life. Recent developments have transformed our understanding of inflammatory drivers uncovering distinct inflammatory endotypes defined by dominant microbial species, pattern-recognition receptor activation, inflammasome signalling, Th17-associated cytokine networks and failures of mucosal immunity. The emerging roles of viral-bacterial interactions, fungi, pathobionts and the broader microbiome challenge the conventional infection-only paradigm and highlight gaps in current therapeutic strategies. Such developments underpin the rationale behind anti-inflammatory strategies in bronchiectasis, ranging from suppression of neutrophil-driven injury through direct neutrophil elastase or upstream dipeptidyl peptidase-1 (DPP-1) inhibition, to immunomodulatory macrolides, toward therapies aimed at recalibrating epithelial and mucosal homeostasis. While several antibacterial and anti-infective trials have produced mixed results, this is likely to reflect unresolved heterogeneity in microbiome composition and host immune signalling. In contrast, emerging anti-inflammatory strategies show strong positive signals, reinforcing the need for better endotyping and biomarker-guided patient selection. Here we synthesize recent mechanistic and clinical insights to propose a more integrated framework for understanding and ultimately targeting airway inflammation in bronchiectasis.
Lerodalcibep: First Approval.
Zhuang-Yan A, Blair HA
Drugs · 2026 Jul · PMID 41945308 · Publisher ↗
Lerodalcibep (lerodalcibep-liga; LEROCHOL), a subcutaneously administered third-generation proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitor, is being developed by LIB Therapeutics for the treatment of hype...
Lerodalcibep (lerodalcibep-liga; LEROCHOL), a subcutaneously administered third-generation proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitor, is being developed by LIB Therapeutics for the treatment of hypercholesterolaemia. Lerodalcibep received its first approval on 12 December 2025 in the USA, as an adjunct to diet and exercise, to reduce low-density lipoprotein cholesterol (LDL-C) in adults with hypercholesterolaemia, including heterozygous familial hypercholesterolaemia. Lerodalcibep is in preregistration for hypercholesterolaemia in the EU. Lerodalcibep is administered as a once-monthly subcutaneous injection. This article summarizes the milestones in the development of lerodalcibep leading to this first approval for the treatment of adults with hypercholesterolaemia.
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