Drugs[JOURNAL]
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200 papers
PCSK9 and ANGPTL3 Inhibitors in Homozygous Familial Hypercholesterolemia: A Meta-analysis of Randomized Clinical Trials.
Bytyçi I, Henein MY, Bytyqi S … +7 more , Shatri M, Gashi D, Dragusha E, Koliqi R, Elezi S, Hernandez AV, Banach M
Drugs · 2026 Feb · PMID 41501311 · Publisher ↗
OBJECTIVE: The aim of this meta-analysis was to compare the efficacy of PCSK9 and ANGPTL3 inhibitors in patients with homozygous familial hypercholesterolemia (HoFH). METHODS: We systematically searched selected electron...
OBJECTIVE: The aim of this meta-analysis was to compare the efficacy of PCSK9 and ANGPTL3 inhibitors in patients with homozygous familial hypercholesterolemia (HoFH). METHODS: We systematically searched selected electronic databases until 30 November 2024. Main end point was the effect of lipid lowering therapy on lipid profile: total cholesterol (TC), triglycerides (TG), low density lipoprotein cholesterol (LDL-C), high density lipoprotein cholesterol (HDL-C) and lipoproteins levels. The secondary end point was adverse clinical effects. RESULTS: A total of 12 trials involving 392 patients with HoFH, were finally included in the meta-analysis. At a median follow-up of 12 months, ANGPTL3 inhibitors achieved greater reductions in TC (- 49.9% versus - 21.2%; p < 0.001), LDL-C (- 50.77% versus - 17.88%; p < 0.001) and TG (- 48.9% versus - 8.2%; p < 0.001) compared with PCSK9 inhibitors, but had a smaller impact on HDL-C (- 28.9% versus + 5.2%; p = 0.001). Apolipoprotein B decreased more with ANGPTL3i (- 26.9% versus - 13.2%; p < 0.001), while lipoprotein(a) reductions were similar between groups, and apolipoprotein A remained unaffected with PCSK9i but slightly decreased with ANGPTL3i. In meta-regression, ANGPTL3i produced a greater LDL-C reduction in the negative LDL receptor (LDLR) genotype (- 34.5%; p = 0.04) and showed a trend toward significance in the defective genotype (- 23.1%; p = 0.07), with no significant difference in the heterozygous type. The rates of adverse events and discontinuations were not significantly different between the groups. CONCLUSIONS: PCSK9 inhibitors have lower efficacy in reducing lipid levels in HoFH compared with ANGPTL3 inhibitors, with the greatest difference seen in patients with the negative LDLR genotype. Further studies are needed to clarify efficacy across LDLR functional variants.
Biased Kappa-opioid Agonist Strategies in Pain Management: Approaches to Clinical Benefits with Reduced Adverse Effects.
Davis MP, Kiselev B
Drugs · 2026 Feb · PMID 41498917 · Publisher ↗
Mu-opioid receptor (MOR) agonists remain the cornerstone of pain management but are limited by respiratory depression, tolerance, dependence, and mood disturbances. Kappa-opioid receptor (KOR) agonists offer complementar...
Mu-opioid receptor (MOR) agonists remain the cornerstone of pain management but are limited by respiratory depression, tolerance, dependence, and mood disturbances. Kappa-opioid receptor (KOR) agonists offer complementary analgesic mechanisms with minimal abuse potential, yet clinical utility is restricted by dysphoria, sedation, and anxiety. This review critically evaluates emerging strategies that leverage both receptor systems through G-protein-biased KOR agonists and bifunctional KOR/MOR ligands. While preclinical evidence suggests that these approaches may provide additive analgesia with reduced adverse effects, significant challenges remain in translating these findings to clinical practice. The controversy surrounding G-protein bias measurement and the limited clinical data available highlight the need for cautious optimism regarding these novel therapeutic approaches.
Developments in Pharmacotherapy for Obstructive Sleep Apnoea: Unlocking the Potential for Targeted Treatment.
Eckert DJ
Drugs · 2026 Feb · PMID 41452574 · Full text
Obstructive sleep apnoea (OSA) is a common, chronic respiratory disorder associated with major co-morbidity and adverse safety consequences. New research has highlighted the heterogeneity of OSA pathogenesis and the impo...
Obstructive sleep apnoea (OSA) is a common, chronic respiratory disorder associated with major co-morbidity and adverse safety consequences. New research has highlighted the heterogeneity of OSA pathogenesis and the importance of non-anatomical contributors beyond the primary cause-impaired pharyngeal anatomy. For > 40 years, continuous positive airway pressure (CPAP), which works downstream from the causes of OSA, has been the mainstay therapeutic approach. While efficacious when used, CPAP is associated with poor tolerance and acceptance which limits real-world clinical effectiveness. Non-CPAP therapies that target the primary anatomical cause such as oral appliances and upper airway surgery, reduce OSA severity by ~ 50%. Given that obesity increases the anatomical predisposition for OSA, new weight loss drugs can reduce OSA severity in the ~ 50% of patients who are obese. Thus, although these therapies can resolve OSA for certain patients, overall efficiency varies and is challenging to predict with currently available clinical tools. Recent translation of OSA pathogenesis research has unlocked new pharmacotherapy targets beyond impaired pharyngeal anatomy. These include drugs to activate the pharyngeal dilator muscles, reduce unstable respiratory control and promote deeper, more stable sleep and breathing. Accordingly, the sleep medicine field is undergoing a paradigm shift as OSA pharmacotherapy becomes a reality. This article outlines the latest OSA pathophysiology knowledge and accompanying recent major developments in OSA pharmacotherapy. Practical, readily available, clinical tools for OSA endotyping to help move beyond the current problematic one-size-fits-all, trial-and-error treatment model towards a targeted paradigm tailored to underlying mechanisms that include emerging pharmacotherapy are also included.
Zeprumetostat: First Approval.
Keam SJ
Drugs · 2026 Mar · PMID 41410728 · Publisher ↗
Zeprumetostat (), an oral, selective, small molecule inhibitor of enhancer of zeste homolog 2 (EZH2), a histone methyltransferase, is being developed by Jiangsu Hengrui Pharmaceutical Co., Ltd for the treatment of solid...
Zeprumetostat (), an oral, selective, small molecule inhibitor of enhancer of zeste homolog 2 (EZH2), a histone methyltransferase, is being developed by Jiangsu Hengrui Pharmaceutical Co., Ltd for the treatment of solid and haematological malignancies. In August 2025, zeprumetostat received conditional approval in China for use in adult patients with relapsed or refractory peripheral T-cell lymphoma (r/r PTCL) who have received ≥ 1 line of systemic therapy. This article summarizes the milestones in the development of zeprumetostat leading to this first approval for the treatment of r/r PTCL.
Rilzabrutinib: First Approval.
Lee A
Drugs · 2026 Mar · PMID 41359083 · Publisher ↗
Rilzabrutinib (WAYRILZ™) is a small-molecule, reversible, highly specific, covalent BTK inhibitor being developed by Sanofi for the treatment of multiple immune-related and inflammatory disorders, including immune thromb...
Rilzabrutinib (WAYRILZ™) is a small-molecule, reversible, highly specific, covalent BTK inhibitor being developed by Sanofi for the treatment of multiple immune-related and inflammatory disorders, including immune thrombocytopenia, warm autoimmune haemolytic anaemia, sickle cell disease, IgG4-related disease, asthma, chronic spontaneous urticaria, among others. As a BTK inhibitor, rilzabrutinib demonstrates multiple immunomodulatory effects, including reduced B cell activation and autoantibody production, inhibition of macrophage activity, and anti-inflammatory effects. In clinical trials, rilzabrutinib was associated with durable platelet responses in patients with previously treated immune thrombocytopenia. This article summarizes the milestones in the development of rilzabrutinib leading to its first approval for the treatment of adults with persistent or chronic immune thrombocytopenia who have had an insufficient response to previous treatment.
Safety and Tolerability of Glucagon-Like Peptide-1 Receptor Agonists: A State-of-the-Art Narrative Review.
Kunutsor SK, Seidu S
Drugs · 2026 Jan · PMID 41351656 · Publisher ↗
Glucagon-like peptide-1 receptor agonists (GLP-1RAs)-along with the dual glucose-dependent insulinotropic polypeptide (GIP)/GLP-1RA tirzepatide-are widely acknowledged for their efficacy in managing both type 2 diabetes...
Glucagon-like peptide-1 receptor agonists (GLP-1RAs)-along with the dual glucose-dependent insulinotropic polypeptide (GIP)/GLP-1RA tirzepatide-are widely acknowledged for their efficacy in managing both type 2 diabetes mellitus and obesity, with expanding indications in cardiometabolic risk reduction. While their glycemic, weight-lowering, and cardiovascular benefits are well established through randomized trials and meta-analyses, concerns remain regarding their safety and tolerability across diverse populations and clinical settings. Gastrointestinal (GI) adverse events-particularly nausea, vomiting, diarrhea, and constipation-are the most common side effects, generally emerging during dose escalation and resolving over time. However, accumulating evidence has identified additional GI complications, including cholelithiasis, cholecystitis, gastroparesis, and bowel obstruction, which may warrant caution in susceptible individuals. Injection-site reactions and worsening of pre-existing diabetic retinopathy are also relevant clinical concerns. Although rare, associations with nonarteritic anterior ischemic optic neuropathy, pancreatitis, medullary thyroid carcinoma, and acute kidney injury (AKI) have been reported, primarily through pharmacovigilance and case-based evidence. Importantly, large-scale randomized trials, meta-analyses, and observational studies suggest that GLP-1RAs do not significantly increase AKI risk and may even confer renal benefits in high-risk populations. There is no confirmed elevated risk of suicidality, but surveillance remains warranted. Safety data in special populations-such as pregnant or lactating women, pediatric patients, and those with advanced renal or hepatic impairment-remain limited and require further study. This state-of-the-art narrative review synthesizes current evidence from clinical trials, pharmacovigilance databases, and real-world cohorts to provide a comprehensive evaluation of the safety and tolerability of GLP-1RAs and tirzepatide. We present clinical strategies for adverse event mitigation, monitoring recommendations, contraindications, and practical considerations for treatment discontinuation or switching. Although these agents offer transformative therapeutic potential, their optimal use requires individualized care, careful patient selection, and ongoing safety surveillance. Future research should prioritize long-term safety in underrepresented populations and strategies to mitigate lean mass loss during therapy.
Brensocatib: First Approval.
Keam SJ
Drugs · 2026 Feb · PMID 41348269 · Publisher ↗
Brensocatib (BRINSUPRI™), an oral, small molecule, reversible inhibitor of dipeptidyl peptidase 1 (DPP1), is being developed by Insmed Incorporated under license from AstraZeneca for the treatment of neutrophil-mediated...
Brensocatib (BRINSUPRI™), an oral, small molecule, reversible inhibitor of dipeptidyl peptidase 1 (DPP1), is being developed by Insmed Incorporated under license from AstraZeneca for the treatment of neutrophil-mediated diseases, including non-cystic fibrosis bronchiectasis (NCFB), chronic rhinosinusitis without nasal polyps (CRSsNP) and hidradenitis suppurativa (HS). Brensocatib received its first approval on 12 August 2025 in the USA for the treatment of NCFB in adult and paediatric patients 12 years of age and older. Brensocatib received a positive opinion in the EU on 17 Oct 2025 and is also under regulatory review in the UK for this indication. This article summarizes the milestones in the development of brensocatib leading to this first approval for the treatment of NCFB.
Elamipretide: First Approval.
Shirley M
Drugs · 2026 Mar · PMID 41335372 · Publisher ↗
Elamipretide (Forzinity™) is a mitochondrial cardiolipin binder being developed by Stealth BioTherapeutics for the treatment of a range of disorders featuring mitochondrial dysfunction. In September 2025, elamipretide wa...
Elamipretide (Forzinity™) is a mitochondrial cardiolipin binder being developed by Stealth BioTherapeutics for the treatment of a range of disorders featuring mitochondrial dysfunction. In September 2025, elamipretide was granted accelerated approval in the USA for use to improve muscle strength in adult and pediatric patients with Barth syndrome weighing ≥ 30 kg. With this accelerated approval, elamipretide became the first disease-specific treatment approved for Barth syndrome, an ultra-rare X-linked recessive genetic disorder. Elamipretide is also under phase III clinical development for use in the treatment of dry age-related macular degeneration and mitochondrial myopathies. This article summarizes the milestones in the development of elamipretide leading to this first approval for Barth syndrome.
Imlunestrant: First Approval.
Keam SJ
Drugs · 2026 Jan · PMID 41335371 · Publisher ↗
Imlunestrant (Inluriyo™), an oral, selective, estrogen receptor (ER) degrader (SERD), is being developed by Eli Lilly and Company for the treatment of ER-positive (ER+), human epidermal growth factor receptor 2 (HER2) ne...
Imlunestrant (Inluriyo™), an oral, selective, estrogen receptor (ER) degrader (SERD), is being developed by Eli Lilly and Company for the treatment of ER-positive (ER+), human epidermal growth factor receptor 2 (HER2) negative (HER2-) breast cancer. In September 2025, imlunestrant was approved for the treatment of adults with ER+, HER2-, ESR1-mutated advanced or metastatic breast cancer with disease progression following at least one line of endocrine therapy in the USA. This article summarizes the milestones in the development of imlunestrant leading to this first approval for use in patients with ER+, HER2-, ESR1-mutated advanced or metastatic breast cancer.
Benefits and Liabilities of Benzodiazepines and Z-Drugs: What We Know and What We Don't Know.
Silberman EK
Drugs · 2026 Jan · PMID 41324787 · Publisher ↗
Benzodiazepines, first introduced in the 1960s as effective and safer alternatives to barbiturate sedative-hypnotics, are now widely viewed as having a high risk of overdose, abuse, addiction, and "dependency". These bel...
Benzodiazepines, first introduced in the 1960s as effective and safer alternatives to barbiturate sedative-hypnotics, are now widely viewed as having a high risk of overdose, abuse, addiction, and "dependency". These beliefs are contrary to extensive scientific evidence. Systematic research has shown that prescribed benzodiazepines are not prone to tolerance, dose-escalation, abuse, or addiction. Benzodiazepine abuse and overdose fatalities occur largely in the context of established polysubstance abuse. They are as effective as antidepressants and have fewer adverse effects but similar withdrawal syndromes. Major adverse effects are impairment of coordination, memory, and cognition, of most concern in older populations, and increased risk of motor vehicle accidents, especially when used along with alcohol. Most patients can be withdrawn from benzodiazepines without major difficulty using clinician supportiveness and flexible tapers. Reports of severe adverse reactions to benzodiazepines and of severe, prolonged difficulties withdrawing from them have not been subjects of systematic study and are poorly understood. In summary, the literature supports use of benzodiazepines on par with antidepressants for anxiety disorders, and for benzodiazepines and z-drugs for short-term mitigation of insomnia.
Paltusotine: First Approval.
Lee A
Drugs · 2026 Feb · PMID 41296264 · Publisher ↗
Paltusotine (PALSONIFY™) is a non-hormonal, orally active selective somatostatin receptor 2 (SSTR2) agonist that controls insulin-like growth factor-1 (IGF-1) levels, which are elevated in patients with acromegaly. It is...
Paltusotine (PALSONIFY™) is a non-hormonal, orally active selective somatostatin receptor 2 (SSTR2) agonist that controls insulin-like growth factor-1 (IGF-1) levels, which are elevated in patients with acromegaly. It is the first approved once daily, orally administered treatment available for the treatment of acromegaly. Paltusotine received its first approval in the USA in September 2025, based on results from the PATHFNDR-1 and PATHFNDR-2 phase III clinical trials in patients with acromegaly. This article summarizes the milestones in the development of paltusotine leading to this first approval for the treatment of adults with acromegaly who had an inadequate response to surgery and/or for whom surgery is not an option.
Pan-JAK Inhibition Across Chronic Hand Eczema and Other Cutaneous Diseases: An Updated Review on Topical Delgocitinib.
Termini D, Avallone G, Tavoletti G … +5 more , Beaziz J, Moltrasio C, Boero V, Marzano AV, Ferrucci SM
Drugs · 2026 Jan · PMID 41291177 · Publisher ↗
Chronic hand eczema is a highly prevalent inflammatory skin disorder associated with substantial impairments in quality of life and social interactions. Topical corticosteroids and calcineurin inhibitors are conventional...
Chronic hand eczema is a highly prevalent inflammatory skin disorder associated with substantial impairments in quality of life and social interactions. Topical corticosteroids and calcineurin inhibitors are conventional treatments often limited by the achievement of suboptimal disease control and concerns regarding long-term safety. Delgocitinib 2% cream, the first topical pan-Janus kinase inhibitor approved in Europe for adult patients with moderate-to-severe chronic hand eczema, represents a significant therapeutic advance. By simultaneously targeting multiple cytokine pathways implicated in chronic hand eczema pathogenesis, delgocitinib offers a corticosteroid-sparing alternative with minimal systemic absorption. Pivotal phase III clinical trials (DELTA 1, 2, and 3) demonstrated significant reductions in disease severity, itch, and pain compared with baseline, with a favorable safety profile sustained over long-term treatment. Recent investigations in adolescent and Asian populations are further expanding the therapeutic profile of topical delgocitinib across different age groups and ethnic backgrounds. Both the cream and ointment formulations have been assessed in clinical settings, with distinct utility depending on anatomical site and barrier integrity. Beyond chronic hand eczema, topical delgocitinib has shown efficacy in atopic dermatitis, and preliminary evidence is emerging in other inflammatory skin diseases, including vitiligo, lichenoid dermatoses, and hair disorders. This review provides an updated overview of the biological rationale for pan-Janus kinase inhibition, summarizes the most recent outcomes of topical delgocitinib in chronic hand eczema and atopic dermatitis management, and outlines its potential new applications in the landscape of immune-mediated skin diseases.
Tolebrutinib: First Approval.
Blair HA
Drugs · 2026 Jan · PMID 41284222 · Publisher ↗
Tolebrutinib (CENRIFKI) is an orally available, brain-penetrant small molecule inhibitor of Bruton's tyrosine kinase (BTK) being developed by Sanofi for the treatment of multiple sclerosis (MS). On 26 August 2025, tolebr...
Tolebrutinib (CENRIFKI) is an orally available, brain-penetrant small molecule inhibitor of Bruton's tyrosine kinase (BTK) being developed by Sanofi for the treatment of multiple sclerosis (MS). On 26 August 2025, tolebrutinib received its first approval in the United Arab Emirates (UAE) for the treatment of non-relapsing secondary progressive multiple sclerosis (nrSPMS) and to slow disability accumulation independent of relapse activity in adults. The drug is under regulatory review in the USA and the EU. This article summarizes the milestones in the development of tolebrutinib leading to this first approval for MS.
Aztreonam-Avibactam: A Review in the Treatment of Serious Bacterial Infections Caused by Aerobic Gram-Negative Organisms.
Shirley M
Drugs · 2026 Jan · PMID 41258612 · Publisher ↗
Aztreonam-avibactam (Emblaveo), a fixed-dose combination of the monobactam antibacterial agent aztreonam and the broad-spectrum β-lactamase inhibitor avibactam, has been developed as a treatment, administered intravenous...
Aztreonam-avibactam (Emblaveo), a fixed-dose combination of the monobactam antibacterial agent aztreonam and the broad-spectrum β-lactamase inhibitor avibactam, has been developed as a treatment, administered intravenously, for serious bacterial infections caused by aerobic Gram-negative organisms. In the EU, aztreonam-avibactam is indicated in adults for the treatment of complicated intra-abdominal infection (cIAI); hospital-acquired pneumonia (HAP), including ventilator-associated pneumonia (VAP); and complicated urinary tract infection (cUTI), including pyelonephritis; as well as for the treatment of other infections due to aerobic Gram-negative organisms in adult patients with limited treatment options. Adding to existing data on aztreonam and avibactam individually, the approval of aztreonam-avibactam was supported by a clinical development programme which included pharmacokinetic and pharmacodynamic modelling and probability of target attainment analyses together with evaluation of the combination in a range of clinical trials. Based on all available data, aztreonam-avibactam is a valuable addition to the treatment options for adults with serious bacterial infections caused by aerobic Gram-negative organisms. Furthermore, with in vitro data showing that the drug combination retains potent activity against metallo-β-lactamase (MBL)-producing Enterobacterales and Stenotrophomonas maltophilia, and noting the limited effective treatment options available for infections caused by such bacteria, aztreonam-avibactam is likely to be particularly useful in regions with a high prevalence of MBLs.
Targeting HER2 in Metastatic Colorectal Cancer: Current Therapies, Biomarker Refinement, and Emerging Strategies.
Vaghi C, Tosi F, Mauri G … +16 more , Bonazzina E, Amatu A, Bencardino K, Piscazzi D, Roazzi L, Villa F, Maggi M, Monti L, Bombelli A, Marrapese G, Ghezzi S, Patelli G, Ros J, Elez E, Sartore-Bianchi A, Siena S
Drugs · 2026 Jan · PMID 41247589 · Full text
HER2 overexpression/amplification is a well-established oncogenic driver across several tumor types and has emerged as a therapeutically actionable biomarker in 3-5% of metastatic colon and rectal cancers (mCRCs). Based...
HER2 overexpression/amplification is a well-established oncogenic driver across several tumor types and has emerged as a therapeutically actionable biomarker in 3-5% of metastatic colon and rectal cancers (mCRCs). Based on increasing clinical evidence, several anti-HER2 therapeutic regimens have been incorporated into treatment guidelines. In this review, we provide a comprehensive overview of both established and emerging HER2-targeted strategies in mCRC, examining the mechanisms of action, toxicity profiles, and clinical efficacy of individual anti-HER2 drugs and their associations. As accurate and standardized HER2 testing remains critical, we also discuss current methodologies and challenges in HER2 screening for mCRC. In addition, we present an in-depth analysis of the relationship between HER2 amplification levels, co-occurring genomic alterations, and treatment outcomes across clinical trials, underscoring the importance of quantitative HER2 assessment and comprehensive tumor profiling. These data support the refinement of therapeutic strategies through more precise diagnostic approaches and improved patient selection, considering factors such as gene-dosage, toxicity, and coexisting mutations. Finally, the emerging role of anti-HER2 rechallenge strategies and the advent of novel HER2-directed therapies-including bispecific antibodies, novel tyrosine kinase inhibitors, antibody-drug conjugates, and immune-based therapies-are discussed as new opportunities to improve outcomes in this molecular subset of patients.
Donidalorsen: First Approval.
Syed YY
Drugs · 2026 Feb · PMID 41222832 · Publisher ↗
Donidalorsen (DAWNZERA™) is a first-in-class, RNA-targeted antisense oligonucleotide (ASO), developed by Ionis Pharmaceuticals for the prevention of hereditary angioedema (HAE) attacks. It received its first approval on...
Donidalorsen (DAWNZERA™) is a first-in-class, RNA-targeted antisense oligonucleotide (ASO), developed by Ionis Pharmaceuticals for the prevention of hereditary angioedema (HAE) attacks. It received its first approval on 21 August 2025 in the USA for the prophylaxis of HAE attacks in adult and paediatric patients aged 12 years and older. Donidalorsen is currently under regulatory review in the EU and in phase III development in several other countries. This article summarizes the key milestones in the development of donidalorsen leading to its first approval for HAE.
Taletrectinib: First Approval.
Hoy SM
Drugs · 2026 Feb · PMID 41222831 · Publisher ↗
Taletrectinib [DOVBLERON (China); IBTROZI (USA)] is an oral, potent, next-generation proto-oncogene tyrosine-protein kinase-1 (c-Ros oncogene-1; ROS1) inhibitor developed by Nuvation Bio China Ltd., a Nuvation Bio Inc....
Taletrectinib [DOVBLERON (China); IBTROZI (USA)] is an oral, potent, next-generation proto-oncogene tyrosine-protein kinase-1 (c-Ros oncogene-1; ROS1) inhibitor developed by Nuvation Bio China Ltd., a Nuvation Bio Inc. company, for the treatment of advanced ROS1-positive non-small cell lung cancer (NSCLC). Taletrectinib received its first approval on 20 December 2024 in China for the treatment of adults with locally advanced or metastatic ROS1-positive NSCLC who have previously been treated with ROS1 inhibitors. Subsequently, taletrectinib was approved on 3 January 2025 in China and on 11 June 2025 in the USA for the treatment of adults with locally advanced or metastatic ROS1-positive NSCLC, and then on 19 September 2025 in Japan for the treatment of adults with unresectable advanced and/or recurrent ROS1-positive NSCLC. Additional global filings for taletrectinib are underway. This article summarizes the milestones in the development of taletrectinib leading to these first approvals.
Elinzanetant: First Approval.
Lee A
Drugs · 2026 Jan · PMID 41222830 · Full text
Elinzanetant (Lynkuet™) is a non-hormonal, small-molecule neurokinin 1 (NK) and 3 (NK) antagonist being developed by Bayer for the treatment of vasomotor symptoms (VMS), which received its first approval in the UK in Jul...
Elinzanetant (Lynkuet™) is a non-hormonal, small-molecule neurokinin 1 (NK) and 3 (NK) antagonist being developed by Bayer for the treatment of vasomotor symptoms (VMS), which received its first approval in the UK in July 2025. As a neurokinin-targeted therapy, elinzanetant modulates the activity of hyperactive kisspeptin/neurokinin B/dynorphin neurones to reduce the frequency and severity of VMS, which was demonstrated during the OASIS clinical trials. This article summarizes the milestones in the development of elinzanetant leading to this first approval for the treatment of moderate to severe VMS associated with menopause.
Can Gene Therapy Transform the Treatment Landscape of Posterior Segment Eye Diseases? A Comprehensive Review of Recent Advancements.
Chen KY, Chan HC, Chan CM
Drugs · 2025 Dec · PMID 41138048 · Full text
Posterior segment eye diseases (PSEDs) encompass a diverse group of conditions affecting the retina, choroid, optic nerve, and vitreous humor, often leading to progressive and irreversible vision loss. Age-related macula...
Posterior segment eye diseases (PSEDs) encompass a diverse group of conditions affecting the retina, choroid, optic nerve, and vitreous humor, often leading to progressive and irreversible vision loss. Age-related macular degeneration (AMD), diabetic retinopathy (DR), retinitis pigmentosa (RP), and inherited retinal diseases (IRDs) are among the most clinically significant PSEDs with a substantial global burden and economic impact. Conventional treatments for PSEDs have limitations that necessitate the development of novel therapies that address the underlying molecular drivers of the disease. Gene therapy has emerged as a promising approach, offering the potential for durable and curative outcomes through precise genetic manipulation. Advancements in gene therapy strategies, including gene augmentation, gene editing, RNA-based therapies, and optogenetics, have led to significant progress in preclinical studies and clinical trials across various PSED subtypes. US Food and Drug Administration (FDA) approval of voretigene neparvovec (Luxturna) for RPE65-associated IRDs validated the clinical viability of ocular gene therapy, while ongoing trials for AMD, DR, and other IRDs continue to expand the therapeutic landscape. Innovations in viral and non-viral delivery systems, such as dual AAV vectors, lipid nanoparticles, and novel biomaterials, have enhanced the efficiency and specificity of gene delivery to the retina. However, challenges persist, including immune responses to viral vectors, limited transduction efficiency in certain cell types, and anatomical barriers posed by the blood-retinal barrier. Future directions in ocular gene therapy include the development of precision genome editing techniques, such as prime editing, miRNA-based regulation, and combinatorial approaches integrating gene therapy with stem cell transplantation or neuroprotective agents. As the field continues to evolve, addressing these challenges and optimizing gene therapy strategies will be crucial in translating the transformative potential of ocular gene therapy into clinical reality for patients with PSEDs.
Dordaviprone: First Approval.
Blair HA
Drugs · 2026 Jan · PMID 41138047 · Publisher ↗
Dordaviprone (MODEYSO) is a protease activator being developed by Chimerix for the treatment of various cancers, including glioma, glioblastoma, endometrial cancer, ovarian cancer, acute myeloid leukaemia, acute lymphobl...
Dordaviprone (MODEYSO) is a protease activator being developed by Chimerix for the treatment of various cancers, including glioma, glioblastoma, endometrial cancer, ovarian cancer, acute myeloid leukaemia, acute lymphoblastic leukaemia, myelodysplastic syndrome and colorectal cancer. On 6 August 2025, dordaviprone received accelerated approval in the USA for the treatment of adult and paediatric patients 1 year of age and older with diffuse midline glioma harbouring an H3 K27M mutation with progressive disease following prior therapy. This article summarizes the milestones in the development of dordaviprone leading to this first approval for glioma.
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