Drugs最新文献

Immune checkpoint inhibitors (ICIs) produce often durable responses in many cancer types but are associated with autoimmune-like toxicities. These immune-related adverse events (irAEs) occur in multiple organ systems and often improve with steroids or therapy cessation. However, many irAEs have either partial or no improvement, leading to residual sequelae even after ICI discontinuation. Chronic irAEs, often defined as irAEs lasting > 3 months, may occur in up to 40-50% of patients treated with ICI therapy and have a wide range of manifestations, severity, and duration. These chronic events likely stem from either ongoing inflammation or sequalae of prior inflammatory-mediated damage. IrAEs impacting the endocrine glands and joints have particularly high rates of chronicity, while chronic events impacting the gastrointestinal (GI) tract, liver, and lungs are less common. In this review, we discuss updated studies surrounding chronic irAEs, providing an overview, followed by organ-specific considerations and consideration of future directions. Key challenges for the field include characterizing the chronic impact of irAEs, developing better treatment or prevention strategies (one possible solution being anti-cytokine therapy), and identifying which patients have active inflammation.

Famitinib (^®), an oral, multi-targeting tyrosine kinase inhibitor (TKI) with dual anti-tumour effects (inhibition of both cell proliferation and angiogenesis), is being developed by Jiangsu Hengrui Medicine Co., Ltd for the treatment of solid tumours. In May 2025, famitinib was granted conditional approval for use in combination with camrelizumab for the treatment of patients with recurrent or metastatic cervical cancer who have failed prior platinum-based chemotherapy and have not received prior bevacizumab in China. This article summarizes the milestones in the development of famitinib leading to this first approval for the treatment of recurrent or metastatic cervical cancer.

Mazdutide (Xinermei^®) is a dual glucagon receptor (GcgR) and glucagon-like peptide-1 receptor (GLP-1R) agonist being developed by Eli Lilly and Company along with Innovent Biologics for use in weight management in adults with obesity or overweight and for the treatment of type 2 diabetes (T2D). In June 2025, mazdutide received its first approval, in China, for use (in combination with diet control and increased physical activity) in long-term body weight management in adults with a body-mass index (BMI) of ≥ 28 kg/m² or with a BMI ≥ 24 kg/m² together with one or more weight-related comorbidity. Subsequently, in September 2025, mazdutide also received approval in China for use in glycaemic control in adults with T2D. Additionally, mazdutide is under clinical evaluation for use in the treatment of metabolic dysfunction-associated fatty liver disease, obstructive sleep apnoea and alcohol use disorder. This article summarises the milestones in the development of mazdutide leading to this first approval for long-term body weight management in adults with obesity or overweight.

Congenital adrenal hyperplasia (CAH) is a group of autosomal recessive endocrine disorders caused by impaired steroidogenesis and insufficient cortisol production from the adrenal cortex. The most common form of CAH is 21-hydroxylase deficiency, caused by mutations in the CYP21A2 gene. This enzyme deficiency causes failure to progress through the steroidogenic pathways, consequently there is an increased synthesis of steroid hormone precursors that result in adrenal androgens excess, leading to virilisation in female patients. Over the past two decades, significant progress has been made in optimising corticosteroid replacement strategies, with a focus on reducing androgen excess while minimising glucocorticoid exposure. Modified-release hydrocortisone formulations, such as Efmody^® and Plenadren^®, as well as continuous subcutaneous infusion therapies, have been developed to help better mimic physiological cortisol rhythms. In addition, corticotropin-releasing hormone-1 (CRH1) receptor antagonists have been investigated and have now been approved as a novel approach to reducing adrenocorticotropic hormone (ACTH)-driven adrenal hyperplasia and androgen excess. More recently, promising novel approaches have been developed to control androgen excess and reduce glucocorticoid exposure. This review provides an overview of current standard-of-care treatments for CAH and highlights recent advancements in therapeutic strategies. By addressing the limitations of traditional glucocorticoid replacement, these innovations have the potential to improve long-term outcomes and quality of life for patients with CAH.

Ifupinostat (; Betlin) is a first-in-class dual phosphoinositide 3-kinase α (PI3Kα)/histone deacetylase (HDAC) inhibitor being developed by BeBetter Med for the treatment of relapsed or refractory diffuse large B-cell lymphoma. On the 30th June 2025, ifupinostat received its first approval in China as a monotherapy for adults with relapsed or refractory diffuse large B-cell lymphoma who have received at least two lines of systemic therapy. Continued approval is dependent on the results of a confirmatory, randomized, controlled phase 3 trial. This article summarizes the milestones in the development of ifupinostat leading to this first approval as a monotherapy for adults with relapsed or refractory diffuse large B-cell lymphoma who have received at least two lines of systemic therapy.

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.

Taletrectinib [DOVBLERON ^® (China); IBTROZI^TM (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.

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.

Clesrovimab (ENFLONSIA™; clesrovimab-cfor) is a long-acting monoclonal antibody developed by Merck & Co., Inc. to prevent respiratory syncytial virus (RSV) disease in infants. It binds a highly conserved epitope at antigenic site IV of the RSV fusion protein, blocking viral entry into host cells and conferring passive immunity. Incorporation of a YTE triple amino acid substitution in the Fc region enhances binding to the neonatal Fc receptor, extending serum half-life and allowing a single, body weight-independent dose. Clesrovimab received its first US approval on 9 June 2025 for preventing RSV lower respiratory tract disease in neonates and infants born during, or entering, their first RSV season. This article summarises the milestones leading to this first approval.