Drugs最新文献

Atrasentan (VANRAFIA^®), a potent, highly selective, orally administered endothelin type A (ET_A) receptor antagonist, is in development by Novartis for the treatment of glomerular disease. In April 2025, atrasentan was approved in the USA (under accelerated approval based on a reduction in proteinuria) to reduce proteinuria in adults with primary IgA nephropathy (IgAN) at risk of rapid disease progression, generally a urine protein-to-creatinine ratio (UPCR) ≥ 1.5 g/g. It has not been established whether atrasentan slows kidney function decline in patients with IgAN. This article summarizes the milestones in the development of atrasentan leading to this first approval for the reduction of proteinuria in adults with primary IgAN at risk of rapid disease progression.

Linvoseltamab (Lynozyfic™) is a human B cell maturation antigen (BCMA)×cluster of differentiation (CD) 3 bispecific antibody that binds to both BCMA and CD3 to direct T cells against malignant B cells. Linvoseltamab is being developed by Regeneron Pharmaceuticals, Inc. for multiple indications including multiple myeloma and received its first approval on 28 Apr 2025 in the EU. This article summarises the milestones in the development of linvoseltamab leading to this first approval in the EU as monotherapy for the treatment of adult patients with relapsed/refractory multiple myeloma who have received ≥ 3 prior therapies, including a proteasome inhibitor, an immunomodulatory agent and an anti-CD38 monoclonal antibody, and have demonstrated disease progression on the last therapy.

Pain relief is a key element of fibromyalgia (FM) treatment. Current guidelines recommend antidepressant (i.e. serotonin-norepinephrine reuptake inhibitors) and anticonvulsant medications (gabapentin/pregabalin), drugs that provide only modest relief, with limitations primarily driven by side effects. In contrast, traditional analgesic drugs, although not sufficiently tested in FM, are commonly used by patients. This dearth of effective treatments has led to isolated, mostly small studies of less familiar drug treatments for FM-related pain. Although no single drug has emerged with appreciable effect, some agents such as cannabinoids and naltrexone, amongst others, have shown some pain modulatory effects. In the absence of drugs in the pipeline, non-pharmacological interventions such as behavioural interventions, neuromodulation techniques and faecal transplantation have been studied. This narrative review will focus on drugs and interventions that have been examined in recent years to modulate pain in FM.

Burning mouth syndrome (BMS) is a chronic pain condition characterized by a persistent burning sensation in the oral mucosa in the absence of visible clinical signs. Its management remains a significant clinical challenge due to the unclear and multifactorial nature of its etiopathogenesis. Differentiating between primary (idiopathic) and secondary (associated with identifiable underlying conditions) BMS is critical for guiding treatment. Current management strategies range from addressing underlying systemic or local factors to utilizing established systemic or topical pharmacologic options (such as benzodiazepines, capsaicin, anticonvulsants, antidepressants), new off-label treatments (including low-dose naltrexone), supplements (such as alpha lipoic acid and phytotherapeutics), alongside non-pharmacological approaches aimed at addressing pain symptoms and enhancing pain-coping skills (such as nerve blocks, cognitive behavioral therapy, and transcranial magnetic stimulation). This review synthesizes the current evidence supporting both established and newly investigated therapies and discusses future research directions to improve outcomes for individuals affected by this chronic pain condition. Ultimately, the best management approach should be based on the most robust evidence-based findings, tailored to the underlying etiopathogenetic mechanisms, and individualized to address patient contributing factors.

Telisotuzumab vedotin (telisotuzumab vedotin-tllv; EMRELIS^™), an antibody-drug conjugate (ADC) directed against c-mesenchymal-epithelial transition factor (c-MET) protein, is being developed by AbbVie for the treatment of solid tumours. On 14 May 2025, telisotuzumab vedotin received accelerated approval in the USA for the treatment of adult patients with locally advanced or metastatic non-squamous non-small cell lung cancer (NSCLC) with high c-Met protein overexpression, as determined by an FDA-approved test, who have received a prior systemic therapy. This article summarizes the milestones in the development of telisotuzumab vedotin leading to this first approval for NSCLC.

Luvometinib (^®), a highly selective orally administered mitogen-activated protein kinase kinase 1/2 (MEK1/2) inhibitor, is being developed by Shanghai Fosun Pharmaceutical (Group) Co., Ltd (Fosun Pharma) for the treatment of rare malignancies and other rare diseases associated with abnormal mitogen-activated protein kinase (MAPK) activation. In May 2025, luvometinib was approved in china for the treatment of adult patients with Langerhans cell histiocytosis (LCH) and histiocytic neoplasms and for the treatment of paediatric patients aged ≥ 2 years with neurofibromatosis type 1 (NF1) who have symptomatic plexiform neurofibromas (PN) not amenable to complete resection. This article summarizes the milestones in the development of luvometinib leading to this first approval for the treatment of adult patients with LCH/histiocytic tumours and children and adolescents aged ≥ 2 years with NF1 with symptomatic, inoperable PN.

Delgocitinib is a pan-Janus kinase (JAK) inhibitor that targets all four JAK isoforms. Delgocitinib 20 mg/g cream (Anzupgo^® 20 mg/g), a non-steroidal therapy, is the first topical treatment indicated in the EU for moderate to severe chronic hand eczema (CHE) in adults for whom topical corticosteroids are inadequate or inappropriate. In two 16-week, double-blind, vehicle-controlled, multinational phase III trials, twice-daily delgocitinib 20 mg/g cream improved the signs and symptoms of CHE and improved health-related quality of life in adults with moderate to severe disease; efficacy was maintained with as-needed treatment in a subsequent 36-week open-label extension study. Moreover, an active-controlled trial demonstrated that delgocitinib 20 mg/g cream had superior efficacy and apparent improved tolerability versus oral alitretinoin in adults with severe CHE. Delgocitinib 20 mg/g cream was well tolerated locally in clinical trials, with low systemic exposure with topical application. In summary, current data show that delgocitinib 20 mg/g cream is an efficacious and well tolerated treatment for moderate to severe CHE in adults. As the first topical treatment specifically approved for CHE, it presents a valuable new non-steroidal option in patients for whom topical corticosteroids are inadequate or inappropriate.

Diabetic kidney disease (DKD), a severe microvascular complication of diabetes mellitus (DM), is the predominant cause of end-stage renal disease. Patients with DM frequently experience dyslipidemia, which can exacerbate DKD progression. Consequently, initiating aggressive lipid-lowering therapy in the early stages of DKD is as important as controlling blood glucose and reducing urinary protein. Statins have been the cornerstone of lipid management, but their use is often limited by adverse effects and potential risks of accelerating DKD progression with prolonged administration. As such, identifying optimal lipid management agents for patients with DKD remains an urgent clinical priority. As a pivotal enzyme in lipid metabolism, proprotein convertase subtilisin/kexin type 9 (PCSK9) plays a role in lipid regulation and is intricately linked to various biological processes, including inflammation, programmed cell death (apoptosis, autophagy, pyroptosis, and ferroptosis), and tumor immunity. Emerging evidence suggests that PCSK9 is involved in the occurrence and development of DKD. This article reviews the possible pathways through which PCSK9 is involved in DKD from the aspects of inflammation, oxidative stress, and programmed cell death and how PCSK9 inhibitors may have the potential to improve DKD while reducing cholesterol levels. Therefore, we propose that PCSK9 inhibitors can be a potential priority choice for lipid-lowering in patients with DKD.

Ivarmacitinib sulfate (ivarmacitinib; ^®), a selective Janus kinase 1 (JAK1) inhibitor, is being developed by Jiangsu Hengrui Pharmaceuticals Co, Ltd Co, Ltd for the treatment of immune-mediated inflammatory diseases. In March 2025, ivarmacitinib was approved for use in adult patients with active ankylosing spondylitis (AS) who have responded poorly to or are intolerant to ≥ 1 tumour necrosis factor (TNF) inhibitors in China. In March 2025, ivarmacitinib was also approved in China for use in adult patients with moderate to severe active rheumatoid arthritis (RA) who have responded poorly to or are intolerant to ≥ 1 TNF inhibitors. In April 2025, ivarmacitinib was approved in China for use in adult patients with moderate to severe atopic dermatitis (AD) who have had an inadequate response or intolerance to topical or other systemic treatments. This article summarizes the milestones in the development of ivarmacitinib leading to this first approval for the treatment of adult patients with active AS.

Chronic obstructive pulmonary disease (COPD) management has evolved with the emergence of advanced pharmacological strategies, notably dual bronchodilation and bifunctional agents. Among these innovations, the selective inhaled phosphodiesterase (PDE)3/4 inhibitor ensifentrine represents a novel therapeutic class that combines bronchodilatory and anti-inflammatory properties within a single molecular entity. Dual bronchodilation, traditionally achieved through the combination of long-acting muscarinic antagonists (LAMAs) and long-acting β₂-agonists, has demonstrated superior efficacy compared with monotherapies, including enhanced pulmonary function, reduced symptom burden, and decreased exacerbation frequency. Ensifentrine, recently approved by the US Food and Drug Administration, exerts bronchodilation via PDE3 inhibition and suppresses inflammation by inhibiting PDE4, offering complementary benefits when combined with LAMAs. Clinical trials, including ENHANCE-1 and ENHANCE-2, have shown that ensifentrine significantly improves FEV₁, reduces exacerbations, and lowers inflammatory biomarkers, with a favorable safety profile. Notably, preclinical and clinical data suggest synergism between ensifentrine and muscarinic antagonists, underpinning the rationale for a combination approach. This has led to the conceptualization of "trifunctional dual bronchodilation," describing a regimen that simultaneously targets distinct bronchodilatory mechanisms and provides robust anti-inflammatory effects. Such a strategy may support corticosteroid stewardship by reducing inhaled corticosteroid use, particularly in patients with low eosinophil counts or corticosteroid resistance. However, limitations remain, including the absence of head-to-head trials against existing triple therapy, high cost, and reliance on nebulized delivery. Future research should evaluate long-term outcomes, optimal placement within treatment algorithms, and potential benefits of dry powder or metered-dose formulations. Overall, ensifentrine, especially in combination with a LAMA, may redefine maintenance therapy in COPD, offering a steroid-sparing alternative grounded in mechanistic synergy and clinical efficacy.