Drugs[JOURNAL]
Sun
200 papers
Situational Prevention of Migraine Attacks: Can Early Treatment Change the Conversation?
Shah T, Ailani J
Drugs · 2025 Sep · PMID 40736648 · Publisher ↗
Migraine is a common disabling neurological condition for which head pain is the most common bothersome symptom. People with migraine also experience other associated symptoms, such as nausea, photophobia, phonophobia, f...
Migraine is a common disabling neurological condition for which head pain is the most common bothersome symptom. People with migraine also experience other associated symptoms, such as nausea, photophobia, phonophobia, fatigue, and neck pain, though these symptoms can start and end at different times during the attack. Associated symptoms can be as disabling as head pain, and-as they continue-can interfere with activities of daily living. Treating earlier in an attack before central sensitization may be more effective in preventing the development of other associated symptoms during a migraine attack. To understand how to discuss early treatment with patients with migraine, it is important to understand the phases of migraine and how to consider treatment during prodrome. Several studies have evaluated the treatment of migraine as early as prodrome. A study of ubrogepant used during prodrome showed improved prodrome symptoms and reduced the chances of proceeding to the headache phase of migraine compared with placebo. Another time to treat early is during situations known to cause migraine, such as menses. Data have shown that certain acute treatments taken regularly during the menstrual cycle and starting before the onset of migraine can be effective in reducing migraine attacks related to menses. In this article, we discuss the data available on treating migraine attacks early, both during prodrome phase and for the situational prevention of migraine during predictable triggers. Further clinical and real-world data are needed to continue to explore this concept and provide better management options for patients.
Acoltremon: First Approval.
Lee A
Drugs · 2025 Oct · PMID 40736647 · Publisher ↗
Acoltremon (TRYPTYR) is a TRPM8 thermoreceptor agonist formulated as eye drops, which has been developed by Alcon for the treatment of dry eye disease. As an insufficient aqueous layer is linked to dry eye disease, the s...
Acoltremon (TRYPTYR) is a TRPM8 thermoreceptor agonist formulated as eye drops, which has been developed by Alcon for the treatment of dry eye disease. As an insufficient aqueous layer is linked to dry eye disease, the stimulation of tear production by acoltremon can relieve symptoms associated with dry eye disease. During phase III trials in patients with dry eye disease, acoltremon demonstrated increased tear production with a low treatment discontinuation rate. This article summarizes the milestones in the development of acoltremon leading to this first approval for the treatment of the signs and symptoms of dry eye disease.
Senaparib: First Approval.
Lee A
Drugs · 2025 Nov · PMID 40736646 · Publisher ↗
Poly(ADP-ribose) polymerase (PARP) inhibitors have demonstrated benefits in progression-free survival for first-line maintenance therapy of ovarian cancer. Senaparib (; Sainapali Jiaonang) is a novel PARP inhibitor that...
Poly(ADP-ribose) polymerase (PARP) inhibitors have demonstrated benefits in progression-free survival for first-line maintenance therapy of ovarian cancer. Senaparib (; Sainapali Jiaonang) is a novel PARP inhibitor that potently inhibits PARP1 and PARP2, which is being developed by IMPACT Therapeutics for the treatment of advanced ovarian cancer and small-cell lung cancer. This article summarizes the milestones in the development of senaparib leading to this first approval for the maintenance treatment of adults with advanced epithelial ovarian, fallopian tube or primary peritoneal cancer who achieved a complete or partial response to first-line platinum-based chemotherapy.
Ensifentrine Plus a Long-Acting Muscarinic Antagonist in COPD: A Trifunctional Dual Bronchodilation Perspective.
Cazzola M, Page CP, Calzetta L … +3 more , Martinez FJ, Rogliani P, Matera MG
Drugs · 2025 Sep · PMID 40702257 · Full text
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...
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 β-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 FEV, 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.
Managing Pain in Fibromyalgia: Current and Future Options.
Clarke H, Peer M, Miles S … +1 more , Fitzcharles MA
Drugs · 2025 Sep · PMID 40679776 · Publisher ↗
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 t...
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.
Delgocitinib 20 mg/g Cream: A Review in Chronic Hand Eczema.
McGuigan A, Shirley M
Drugs · 2025 Sep · PMID 40658334 · Publisher ↗
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 moderat...
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.
Correction: Mirdametinib: First Approval.
Hoy SM
Drugs · 2025 Aug · PMID 40622462 · Full text
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Fitusiran: First Approval.
Lee A
Drugs · 2025 Aug · PMID 40579682 · Publisher ↗
Haemophilia A and B are inherited bleeding disorders caused by mutations in clotting factors. Small interfering RNA therapies may be utilised to restore coagulation by preventing the synthesis of antithrombin. Fitusiran...
Haemophilia A and B are inherited bleeding disorders caused by mutations in clotting factors. Small interfering RNA therapies may be utilised to restore coagulation by preventing the synthesis of antithrombin. Fitusiran (QFITLIA™) is a small interfering RNA therapy that targets antithrombin, which is being developed by Sanofi for the prophylactic management of haemophilia A and B with or without inhibitors. Fitusiran has the potential to be administered less frequently than other prophylactic treatments for haemophilia. This article summarizes the milestones in the development of fitusiran leading to this first approval for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and paediatric patients aged 12 years and older with haemophilia A or B with or without factor VIII or IX inhibitors.
Efficacy and Safety of Plecanatide in Chinese Patients with Functional Constipation: A Phase III Multicenter, Randomized, Double-Blind, Placebo-Controlled Trial.
Bai T, Xu Z, Zhen Y … +39 more , Liao A, Wang B, Zhao R, Zhu Y, Dai N, Liu S, Zhao H, Chen X, Huang K, Xu M, Zhou W, Xu B, Ye B, Zou D, Zhang H, Shi R, Zhang J, Ai Y, Fang X, Lin L, Zhang X, Zhang L, Wang J, Jiang Y, Cui J, Zhang M, Ding X, Zhou Z, Yan P, Li X, Jiang B, Liu Y, Ma Y, Yang S, Wang X, Wu Y, Wu J, Chen H, Hou X
Drugs · 2025 Aug · PMID 40571893 · Publisher ↗
BACKGROUND: Plecanatide is a novel guanylate cyclase-C agonist for the treatment of functional constipation (FC). Its efficacy may vary across different racial populations. OBJECTIVE: This study aimed to comprehensively...
BACKGROUND: Plecanatide is a novel guanylate cyclase-C agonist for the treatment of functional constipation (FC). Its efficacy may vary across different racial populations. OBJECTIVE: This study aimed to comprehensively evaluate the efficacy, safety, and pharmacokinetics of plecanatide in Chinese patients with FC. METHODS: This phase III, randomized, double-blind, placebo-controlled trial was conducted across 40 hospitals in China. A total of 648 patients with FC were randomly assigned in a ratio of 1:1 to receive either plecanatide 3 mg or placebo for 12 weeks, followed by a 2-week follow-up. The primary efficacy endpoint was the durable overall complete spontaneous bowel movement (CSBM) response rate. Data on adverse events were collected. A post hoc logistic regression analysis was performed to identify predictors of durable overall CSBM response. RESULTS: After 12 weeks of continuous treatment, the durable overall CSBM response rates were 23.5% in the plecanatide group and 10.2% in the placebo group (p < 0.001). Plecanatide significantly increased the mean weekly frequency of CSBM (1.89 vs 0.9) and SBM (2.33 vs 1.03) compared with placebo throughout the treatment period. In addition, all other secondary efficacy endpoints showed statistically significant improvements with plecanatide compared with placebo. The most common treatment-related emergent adverse event was diarrhea, which occurred in 4.3% of plecanatide-treated patients and 0.6% of placebo-treated patients (p = 0.002). Plasma concentrations of plecanatide and its metabolite SP-338 remained below the lower limit of quantification (0.500 ng/ml) at all assessed time points. Weekly CSBM response at week 2 (odds ratio 43.476; 95% confidence interval 18.274-103.432) and baseline stool consistency (odds ratio 0.550; 95% confidence interval 0.366-0.827) were identified as effective predictors of durable overall CSBM response. Even among plecanatide non-responders, a significant improvement in SBM frequency compared with placebo was observed over the 12-week treatment period. CONCLUSIONS: Plecanatide 3 mg was effective and well tolerated in the treatment of Chinese patients with FC. A weekly CSBM response at week 2 may serve as a predictor of 12-week durable overall efficacy. Patients who did not achieve the primary endpoint may still benefit from plecanatide treatment. CLINICALTRIALS: GOV: NCT0515132.
Tiratricol: First Approval.
Lamb YN
Drugs · 2025 Aug · PMID 40549098 · Publisher ↗
Tiratricol (Emcitate) is an orally bioavailable small molecule being developed by Egetis Therapeutics for the treatment of monocarboxylate transporter 8 (MCT8) deficiency. Tiratricol, an analogue and metabolite of the th...
Tiratricol (Emcitate) is an orally bioavailable small molecule being developed by Egetis Therapeutics for the treatment of monocarboxylate transporter 8 (MCT8) deficiency. Tiratricol, an analogue and metabolite of the thyroid hormone triiodothyronine (T3), has thyromimetic effects but differs from T3 in that it can enter cells independent of MCT8. Tiratricol received its first approval on 12 February 2025 in the European Union, for the treatment of peripheral thyrotoxicosis in patients with MCT8 deficiency (Allan-Herndon-Dudley Syndrome), from birth. Tiratricol will be available as 350 µg dispersible tablets. Tiratricol is currently undergoing clinical development for MCT8 deficiency in several other countries including the USA. This article summarizes the milestones in the development of tiratricol leading to this first approval for MCT8 deficiency.
Limertinib: First Approval.
Lee A
Drugs · 2025 Aug · PMID 40549097 · Publisher ↗
Limertinib (Aoyixin) is an orally available, third-generation epidermal growth factor receptor (EGFR) tyrosine kinase inhibitor (TKI), which has been developed for the treatment of non-small cell lung cancer (NSCLC). In...
Limertinib (Aoyixin) is an orally available, third-generation epidermal growth factor receptor (EGFR) tyrosine kinase inhibitor (TKI), which has been developed for the treatment of non-small cell lung cancer (NSCLC). In clinical trials, limertinib demonstrated efficacy in patients who are positive for the T790M gatekeeper mutation. Limertinib received its first approval for the treatment of adults with locally advanced or metastatic NSCLC with disease progression following treatment with an EGFR TKI and are positive for the EGFR T790M mutation in China in January 2025. In April 2025, limertinib was also approved in China for first-line treatment for adult patients with locally advanced or metastatic NSCLC harbouring EGFR exon 19 deletions or exon 21 L858R mutations. This article summarizes the milestones in the development of limertinib leading to this first approval.
Recaticimab: First Approval.
Lamb YN
Drugs · 2025 Aug · PMID 40545518 · Publisher ↗
Recaticimab (), a humanized monoclonal immunoglobulin G1 antibody that targets proprotein convertase subtilisin/kexin type 9 (PCSK9), is being developed by Suzhou Suncadia Biopharmaceutical for the treatment of hyperchol...
Recaticimab (), a humanized monoclonal immunoglobulin G1 antibody that targets proprotein convertase subtilisin/kexin type 9 (PCSK9), is being developed by Suzhou Suncadia Biopharmaceutical for the treatment of hypercholesteremia and mixed dyslipidemia. Recaticimab received its first approval on 8 January 2025 in China, as an adjunct to diet, in combination with statins (with or without other lipid-lowering therapies) in adults with primary hypercholesterolemia (including heterozygous familial and non-familial hypercholesterolemia) and mixed dyslipidemia who have not achieved their low-density lipoprotein cholesterol (LDL-C) target despite receiving moderate or higher doses of statins, and for use as monotherapy in adults with non-familial hypercholesterolemia and mixed dyslipidemia to reduce LDL-C, total cholesterol, and apolipoprotein B levels. This article summarizes the milestones in the development of recaticimab leading to this first approval for hypercholesterolemia and mixed dyslipidemia.
Novel Potassium Binders in Reduction of Hyperkalemia and Optimization of RAAS Inhibitors Treatment in Patients with Chronic Kidney Disease or Heart Failure: A Systematic Review and Meta-analysis.
Huang N, Xu Y, Liu C … +6 more , Liu Y, Fan Y, Li Z, Zhang D, Mao H, Chen W
Drugs · 2025 Aug · PMID 40542996 · Full text
BACKGROUND: Renin-angiotensin-aldosterone system inhibitors (RAASi) and its combination therapy are essential supportive treatments for patients with chronic kidney disease (CKD) and heart failure (HF). Hyperkalemia (HK)...
BACKGROUND: Renin-angiotensin-aldosterone system inhibitors (RAASi) and its combination therapy are essential supportive treatments for patients with chronic kidney disease (CKD) and heart failure (HF). Hyperkalemia (HK) is the major safety concern associated with the treatments, which often leads to RAASi dose reduction or discontinuation, thereby compromising cardiovascular protective effects. Although novel potassium binders (NPBs) are recommended by current guidelines for the treatment of HK, systematic evidence is needed to guide their use in RAASi optimization and HK management. A systematic review and meta-analysis was conducted to evaluate the incidence of HK in patients with CKD or HF who received RAASi and the efficacy of NPBs in RAASi optimization. METHODS: PubMed, Medline, Embase, and the Cochrane Library were searched from January 1, 2011 to December 31, 2023. Any studies of adult patients with CKD or HF who received RAASi were included in systematic review of HK incidence and risk factors (part 1). Randomized controlled trials (RCTs) of NPBs in patients with CKD or HF were included in meta-analysis on the efficacy of novel potassium binders (NPBs) (part 2). The primary outcome was optimization of RAASi therapy with NPBs. A pooled analysis was conducted in part 1. Network meta-analyses using a random-effects model were performed in part 2. RESULTS: A total of 83 studies (24 with CKD, 54 with HF, and 5 with CKD and HF) were included in part 1 and 8 RCTs (2 with CKD, 4 with HF, and 2 with CKD and HF) were included in part 2. The pooled HK incidence was 10.7% overall at any criteria and in all patients who received RAASi. The highest incidence of HK was observed with the combination of angiotensin converting enzyme inhibitor (ACEi)/angiotensin ii receptor blockers (ARBs)/angiotensin receptor-neprilysin inhibitor (ARNi)+ mineralocorticoid receptor antagonists (MRAs) (24.4%), followed by the triple therapy ACEi/ARB/ARNi+ sodium glucose cotransporter-2 inhibitor (SGLT2i)+MRA (10.9%), and ACEi/ARB/ARNi + SGLT2i (2.2%). Novel potassium binders improved RAASi optimization by 38% compared with placebo (risk ratio, 1.38; 95% CI, 1.16-1.65). Additionally, NPBs decreased the incidence of HK by 28% and reduced the level of potassium by 0.71 mEq/L. The CKD population showed a higher optimization rate than the HF population (84% vs 29%). CONCLUSION: The RAASi treatment was associated with high prevalence of HK, especially in bigeminal and triple therapy. The NPBs were effective in RAASi optimization and HK management, especially among the CKD population.
Ivarmacitinib Sulfate: First Approval.
Keam SJ
Drugs · 2025 Sep · PMID 40542222 · Publisher ↗
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 M...
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.
Sipavibart: First Approval.
Keam SJ
Drugs · 2025 Aug · PMID 40488927 · Publisher ↗
Sipavibart (KAVIGALE), a recombinant human immunoglobulin (Ig)G1-based antibody, is being developed by AstraZeneca for the pre-exposure prophylaxis of COVID-19 in immunocompromised individuals. Sipavibart was approved in...
Sipavibart (KAVIGALE), a recombinant human immunoglobulin (Ig)G1-based antibody, is being developed by AstraZeneca for the pre-exposure prophylaxis of COVID-19 in immunocompromised individuals. Sipavibart was approved in December 2024 in Japan to prevent the onset of infection caused by SARS-CoV-2 in adults and adolescents aged ≥ 12 years weighing ≥ 40 kg where vaccination against infection caused by SARS-CoV-2 is not recommended or may not achieve a sufficient immune response. Sipavibart was also approved in the EU for the pre-exposure prophylaxis of COVID-19 in adults and adolescents aged ≥ 12 years weighing ≥ 40 kg who are immunocompromised due to a medical condition or receipt of immunosuppressive treatments in January 2025 and in Canada in March 2025. This article summarizes the milestones in the development of sipavibart leading to this first approval for the pre-exposure prophylaxis of COVID-19 in immunocompromised adults and adolescents.
QT Prolongation and Risk of Death with the Use of Methadone for Chronic Cancer and Noncancer Pain: Myths or Reality?
Mercadante S
Drugs · 2025 Aug · PMID 40445531 · Publisher ↗
The use of methadone has been associated with corrected QT (QTc) prolongation. However, conclusions about the dangers of methadone are limited by its dual use for narcotic abuse deterrence. All these observations can det...
The use of methadone has been associated with corrected QT (QTc) prolongation. However, conclusions about the dangers of methadone are limited by its dual use for narcotic abuse deterrence. All these observations can deter physicians from prescribing methadone in patients with chronic pain, particularly those with cancer pain. The aim of this review was to evaluate the existing data regarding the relevance of QT changes, the risk factors for QTc prolongation, as well as the risk for cardiac events and mortality, in patients receiving methadone for chronic pain. In total, 15 studies were evaluated. They differed greatly in design (prospective, retrospective), levels of QTc ranges, number of patients included, and methadone doses. Data suggest that the relevance of QTc prolongation induced by methadone seems to be minimal, also considering the range of dosages commonly used in both noncancer and cancer pain. Some risk factors for QTc prolongation have been identified. Information regarding a prior history or prolonged QTc interval, a family history of a prolonged QTc interval, or a family history of sudden, unexplained death is crucial. In this population and at clinical methadone dosages, serious cardiac events have not been described. Low doses of methadone commonly used in most chronic patients may not require QTc monitoring. When a patient is receiving various medications that could potentially prolong QTc, clinicians may consider obtaining a manually measured QTc. Early discussions with patients regarding goals of care, risks, and benefits will help avoid QTc measurements at regular intervals.
Mirdametinib: First Approval.
Hoy SM
Drugs · 2025 Jul · PMID 40419758 · Full text
Mirdametinib (GOMEKLI) is an oral small molecule inhibitor of mitogen-activated protein kinase kinases 1 and 2 (MEK1/2) developed by SpringWorks Therapeutics for the treatment of neurofibromatosis type 1 (NF1)-associated...
Mirdametinib (GOMEKLI) is an oral small molecule inhibitor of mitogen-activated protein kinase kinases 1 and 2 (MEK1/2) developed by SpringWorks Therapeutics for the treatment of neurofibromatosis type 1 (NF1)-associated plexiform neurofibromas (PN). It is the first therapy to be approved for the treatment of both adults and children with NF1-associated PN in the USA, where it is indicated for use in adult and paediatric patients 2 years of age and older with NF1 who have symptomatic PN not amenable to complete resection. A marketing authorisation application for mirdametinib in NF1-PN is currently under review in the EU. Clinical studies of mirdametinib for the treatment of paediatric low-grade glioma are ongoing. This article summarizes the milestones in the development of mirdametinib leading to this first approval.
Vimseltinib: First Approval.
Lee A
Drugs · 2025 Jul · PMID 40415163 · Publisher ↗
Vimseltinib (ROMVIMZA™) is an orally administered kinase inhibitor that targets colony-stimulating factor 1 receptor (CSF1R), which is being developed by Deciphera Pharmaceuticals. As CSF1R activity has been identified a...
Vimseltinib (ROMVIMZA™) is an orally administered kinase inhibitor that targets colony-stimulating factor 1 receptor (CSF1R), which is being developed by Deciphera Pharmaceuticals. As CSF1R activity has been identified as a contributing factor for tenosynovial giant cell tumour (TGCT), treatments targeting CSF1R have been investigated. Vimseltinib received its first approval in February 2025 in the USA for the treatment of TGCT and is under development for the treatment of chronic graft versus host disease. This article summarizes the milestones in the development of vimseltinib leading to this first approval for the treatment of adult patients with symptomatic TGCT for which surgical resection will potentially cause worsening functional limitation or severe morbidity.
A Clinical Pharmacological Perspective on Intraperitoneal Chemotherapy.
Rietveld PCS, Guchelaar NAD, Sassen SDT … +3 more , Koch BCP, Mathijssen RHJ, Koolen SLW
Drugs · 2025 Jul · PMID 40411722 · Full text
Peritoneal metastases (PM), frequently observed in malignancies such as ovarian, colorectal, pancreatic, and gastric cancers, present a significant therapeutic challenge due to poor prognosis and limited effectiveness to...
Peritoneal metastases (PM), frequently observed in malignancies such as ovarian, colorectal, pancreatic, and gastric cancers, present a significant therapeutic challenge due to poor prognosis and limited effectiveness to systemic chemotherapy. The peritoneal-plasma barrier reduces effective drug transfer from plasma to the peritoneal cavity, reducing cytotoxic effects on PM. Intraperitoneal (IP) chemotherapy offers a locoregional approach, enabling high local drug concentrations that can enhance therapeutic efficacy while limiting systemic toxicity. The three major methods for IP administration-hyperthermic intraperitoneal chemotherapy (HIPEC), pressurized intraperitoneal aerosol chemotherapy (PIPAC), and catheter-based IP (CBIP) chemotherapy-each provide unique pharmacokinetic (PK) advantages for PM treatment. This review provides a comprehensive update on the pharmacological rationale of IP chemotherapy, focusing on drug characteristics that support extended IP retention and effective tumor targeting. The effects of administration variables are discussed, highlighting their role in optimizing IP drug exposure. Additionally, recent PK data on commonly used drugs in IP therapy, including platinum-based agents, taxanes, and novel nanoparticle formulations, will be evaluated. While PK rationale supports the administration of IP chemotherapy, further efficacy results from ongoing clinical trials are still awaited. Innovations in nanoparticle-based formulations and controlled-release systems offer substantial potential for improving both drug retention and targeted delivery, enhancing treatment precision and minimizing systemic toxicity. Continued exploration in these areas, along with optimization of IP administration protocols, is vital for advancing patient outcomes, refining therapeutic strategies, and maximizing the benefits of IP chemotherapy in clinical practice.
Rebound Pain After Peripheral Nerve Block: A Review.
Yin W, Luo D, Mi H … +4 more , Ren Z, Li L, Fan Z, Lin J
Drugs · 2025 Aug · PMID 40402360 · Publisher ↗
Peripheral nerve block (PNB) is now a commonly used analgesic treatment in clinical anesthesia owing to ongoing advancements in ultrasound imaging technology, which provides clear images of the nerves. Multimodal analges...
Peripheral nerve block (PNB) is now a commonly used analgesic treatment in clinical anesthesia owing to ongoing advancements in ultrasound imaging technology, which provides clear images of the nerves. Multimodal analgesia based on peripheral nerve blocks is replacing the conventional opioid-based analgesic strategy. However, after the nerve block effect is removed, some patients experience rebound pain (RP), which exacerbates suffering. The benefits of PNB as a perioperative analgesic may be completely negated if RP is discovered and treated too late, even if it can be promptly managed with analgesics. The definitions, clinical signs, risk factors, pathophysiology, and prevention of RP after PNBs are reviewed in this article. At present, the mechanism of RP after PNB is still unclear, but different types of RP may share similar mechanisms in the pain transmission pathway. In this review, we have determined the characteristics of RP and tried to identify the high-risk factors. Among the many means of preventing and reducing the incidence of RP identified, a single block with adjuvant dexamethasone is a reliable regimen, but for the time being, the application of a catheter would be a more reliable method of reducing RP. This review also provides recommendations for the proper use of nerve blocks as supplemental analgesics under clinical anesthesia.
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