|
|
- Crinecerfont: First Approval
-
Free pre-print version: Loading...
Rate this result:
What is this?
Please help us test our new pre-print finding feature by giving the pre-print link a rating.
A 5 star rating indicates the linked pre-print has the exact same content as the published article.
Abstract: Crinecerfont (CRENESSITY™) is a corticotropin releasing factor type 1 (CRF1) receptor antagonist developed by Neurocrine Biosciences for the treatment of classic congenital adrenal hyperplasia (CAH) in adult and paediatric patients. In patients with classic CAH, circulating levels of adrenocorticotropic hormone (ACTH), androstenedione and 17-hydroxyprogesterone are elevated, which traditionally has required supraphysiologic doses of glucocorticoids to manage. As a CRF1 receptor antagonist, crinecerfont acts by reducing systemic ACTH secretion to subsequently decrease elevated levels of steroid precursors and adrenal androgens, thereby reducing the dosage of glucocorticoids required to manage androgen levels in patients. This article summarizes the milestones in the development of crinecerfont leading to this first approval as an adjunctive treatment to glucocorticoid replacement to control androgens in adults and paediatric patients aged ≥ 4 years with classic CAH.
PubDate: 2025-04-17
- Association of Glucagon-Like Peptide-1 Receptor Agonist with Progression
to Liver Cirrhosis and Alcohol-Related Admissions in Patients with Alcohol
Use Disorder and Diabetes: A Retrospective Cohort Study-
Free pre-print version: Loading...
Rate this result:
What is this?
Please help us test our new pre-print finding feature by giving the pre-print link a rating.
A 5 star rating indicates the linked pre-print has the exact same content as the published article.
Abstract: Aim In recent years, use of glucagon-like peptide-1 receptor agonists (GLP-1RA) has exponentially increased due to their beneficial effects on weight loss and cardiovascular outcomes. Lately, some animal studies and observational data suggested that GLP-1RA may be useful in the treatment of alcohol use disorder (AUD). We aim to compare the risk of progression to liver cirrhosis and alcohol-related hospital admission after initiation of GLP-1RA versus dipeptidyl peptidase-4 inhibitors (DPP4i), as the active comparator, in patients with type 2 diabetes mellitus and AUD. Methods We conducted a retrospective propensity score-matched cohort study, utilizing new-user and active comparator design. The study used data from the Veterans Health Administration during fiscal years 2006 to 2021 encompassing adults with AUD who initiated either GLP-1RA or DPP4i prescriptions. Our two co-primary outcomes were progression to cirrhosis (compensated and decompensated cirrhosis) and alcohol-related hospital admission. Results The eligible cohort included 9965 GLP-1RA users and 19,688 DPP4i users. After propensity score matching, 7302 pairs were matched on 79 characteristics without residual imbalances. In the propensity score-matched cohort, progression to cirrhosis occurred in 6.6% of GLP-1RA users and 6.0% DPP4i users; odds ratio (OR): 1.1, 95% confidence interval (95% CI): 0.97–1.26. Alcohol-related hospital admission occurred in 1.4% of GLP-1RA users and in 1.7% of DPP4i users (OR: 0.85; 95% CI: 0.65–1.11). Conclusions Use of GLP-1RA in patients with AUD was not associated with beneficial effect on progression to cirrhosis or alcohol-related hospital admission.
PubDate: 2025-04-13
- Correction: Current and Emerging Therapies for Lysosomal Storage Disorders
-
Free pre-print version: Loading...
Rate this result:
What is this?
Please help us test our new pre-print finding feature by giving the pre-print link a rating.
A 5 star rating indicates the linked pre-print has the exact same content as the published article.
PubDate: 2025-04-12
- PI3K Inhibitors as Potential Therapeutic Agents for the Treatment of COPD
with Associated Atherosclerosis-
Free pre-print version: Loading...
Rate this result:
What is this?
Please help us test our new pre-print finding feature by giving the pre-print link a rating.
A 5 star rating indicates the linked pre-print has the exact same content as the published article.
Abstract: Chronic obstructive pulmonary disease (COPD) and cardiovascular disease (CVD) share a complex and multifactorial relationship characterized by overlapping risk factors, systemic inflammation, and intertwined pathophysiological mechanisms, with atherosclerosis emerging as a central inflammatory process connecting COPD and CVD, driven by systemic inflammation, oxidative stress, and endothelial dysfunction. While systemic inflammation is recognized as a critical link between these conditions, the precise pathways through which inflammation arises remain under investigation. There is therefore a need for therapeutic strategies to mitigate cardiovascular risks in patients with COPD. Among the pathways contributing to this interplay, the phosphoinositide 3-kinase (PI3K) signaling pathway has gained significant attention. Dysregulated PI3K signaling contributes to inflammation, oxidative stress, and endothelial dysfunction, which are key drivers of both COPD and CVD. Consequently, PI3K inhibitors have emerged as a promising therapeutic approach to mitigate inflammation and oxidative damage, offering a targeted strategy to address the shared pathological mechanisms underlying these diseases. A comprehensive understanding of the role of PI3K signaling and its inhibitors could facilitate the development of novel interventions to reduce cardiovascular risk in patients with COPD.
PubDate: 2025-04-11
- Acoramidis: First Approval
-
Free pre-print version: Loading...
Rate this result:
What is this?
Please help us test our new pre-print finding feature by giving the pre-print link a rating.
A 5 star rating indicates the linked pre-print has the exact same content as the published article.
Abstract: Disassociation of transthyretin (TTR) has been identified as a key step in the pathology of TTR amyloid cardiomyopathy, which is characterised by the presence of amyloid fibrils in cardiac tissue. Acoramidis (ATTRUBY™) is a small molecule TTR stabiliser being developed by BridgeBio Pharma, Inc. and is the first drug to demonstrate near-complete (> 90%) stabilisation of TTR. This article summarizes the milestones in the development of acoramidis leading to its first approval in the USA for the treatment of the cardiomyopathy of wild-type or variant TTR-mediated amyloidosis in adults to reduce cardiovascular death and cardiovascular-related hospitalization. In the EU, a positive opinion has been adopted for the treatment of wild-type or variant transthyretin amyloidosis in adult patients with cardiomyopathy.
PubDate: 2025-04-04
- The Potential of RNA Therapeutics in Treating Cardiovascular Disease
-
Free pre-print version: Loading...
Rate this result:
What is this?
Please help us test our new pre-print finding feature by giving the pre-print link a rating.
A 5 star rating indicates the linked pre-print has the exact same content as the published article.
Abstract: Despite significant advances in cardiology over the past few decades, cardiovascular diseases (CVDs) remain the leading cause of global mortality and morbidity. This underscores the need for novel therapeutic interventions that go beyond symptom management to address the underlying causal mechanisms of CVDs. RNA-based therapeutics represent a new class of drugs capable of regulating specific genetic and molecular pathways, positioning them as strong candidates for targeting the root causes of a wide range of diseases. Moreover, owing to the vast diversity in RNA form and function, these molecules can be utilized to induce changes at different levels of gene expression regulation, making them suitable for a broad array of medical applications, even within a single disease context. Several RNA-based therapies are currently being investigated for their potential to address various CVD pathologies. These include treatments aimed at promoting cardiac revascularization and regeneration, preventing cardiomyocyte apoptosis, reducing harmful circulating cholesterols and fats, lowering blood pressure, reversing cardiac fibrosis and remodeling, and correcting the genetic basis of inherited CVDs. In this review, we discuss the current landscape of RNA therapeutics for CVDs, with an emphasis on their classifications, modes of action, advancements in delivery strategies and considerations for their implementation, as well as CVD targets with proven therapeutic potential.
PubDate: 2025-04-02
- Efficiency and Safety of Noninvasive and Intravesical Therapy for Adult
Neurogenic Lower Urinary Tract Dysfunction: A Systematic Review and
Network Meta-analysis of Randomized Controlled Trials-
Free pre-print version: Loading...
Rate this result:
What is this?
Please help us test our new pre-print finding feature by giving the pre-print link a rating.
A 5 star rating indicates the linked pre-print has the exact same content as the published article.
Abstract: Background Neurogenic lower urinary tract dysfunction (NLUTD), a complex abnormality caused by multiple neurologic disorders, is a serious threat to patients’ prognosis and quality of life. However, benefit and safety for various treatments are inconsistent. The aim of the current study was to investigate the available trials of adult NLUTD treatments and provide valuable insights for clinical practice. Methods The data sources were Medline, Embase, and Cochrane databases up to 31 December 2023. A Bayesian network meta-analysis was conducted with randomized controlled trials in patients who were diagnosed with NLUTD, reporting clinical symptoms and urodynamic data. The main outcomes were incontinence episodes frequency (IEF) and maximum cystometric capacity (MCC). Secondary outcomes included frequency, maximum detrusor pressure, bladder compliance, volume of involuntary detrusor contraction, voided volume, incontinence, quality of life, and adverse events. Results A total of 54 articles were eligible, evaluating 28 treatments and 4478 patients for efficacy and safety. Compared with the control group, the oxybutynin instillation demonstrated a mean reduction in IEF of − 2.65 episodes (95% confidence interval [CI]: − 4.64 to − 0.67), with a surface under the cumulative ranking curve (SUCRA) value of 85.8%. Botulinum toxin trigone-combined injection resulted in a reduction of -2.30 episodes (95% CI: -3.23 to - 1.44; SUCRA 84.2%). Additionally, intravesical therapies significantly increased MCC: oxybutynin instillation (mean 227.75 mL, 95% CI 147.00 to 311.42, SUCRA 99.1%) and BTX300U (mean 147.88 mL, 95% CI 100.45–190.32, SUCRA 83.2%). Botulinum toxin injection emerged as the preferred option for improving most urodynamic outcomes and quality of life. However, the incidence of adverse events associated with intravesical injections was higher compared with oral medications and other noninvasive therapies. The three types of botulinum toxins (onabotulinum toxin, abobotulinum toxin, and incobotulinum toxin) demonstrated consistent efficacy in treating both IEF and MCC. Individual studies were sequentially excluded for analysis of network stability. Most results from the alternative networks were consistent with the original analysis, although specific trials influenced certain therapy rankings. Conclusions Overall, oxybutynin instillation and intravesical botulinum toxin injection demonstrated significant advantages in improving symptoms and urodynamic parameters. Our findings support intravesical treatment as a safe and effective option, provided that patients are fully informed about their treatment choices. Clinically, intravesical therapies, oral medications, nerve stimulation, and other treatments should be integrated into shared decision-making processes, while some options require further research to bolster the supporting evidence.
PubDate: 2025-04-01
- Iparomlimab and Tuvonralimab: First Approval
-
Free pre-print version: Loading...
Rate this result:
What is this?
Please help us test our new pre-print finding feature by giving the pre-print link a rating.
A 5 star rating indicates the linked pre-print has the exact same content as the published article.
Abstract: Iparomlimab and tuvonralimab (齐倍安®) is a bifunctional combination of anti-programmed death receptor-1 (PD-1)/anti-cytotoxic T lymphocyte-associated protein-4 (CTLA-4) monoclonal antibodies (mAbs) being developed by Qilu Pharmaceutical Co., Ltd for the treatment of advanced, solid, malignant tumours. In September 2024, iparomlimab and tuvonralimab was granted conditional approval (based on surrogate endpoints) for the treatment of patients with recurrent or metastatic cervical cancer who have failed previous platinum-based chemotherapy. This article summarizes the milestones in the development of iparomlimab and tuvonralimab leading to this first approval for the treatment of patients with recurrent or metastatic cervical cancer who have failed previous platinum-based chemotherapy.
PubDate: 2025-04-01
- Tafolecimab, a Third Monoclonal Antibody for PCSK9 as the Novel
Lipid-Lowering Drug Around the World: A Narrative Review-
Free pre-print version: Loading...
Rate this result:
What is this?
Please help us test our new pre-print finding feature by giving the pre-print link a rating.
A 5 star rating indicates the linked pre-print has the exact same content as the published article.
Abstract: Tafolecimab, a fully human monoclonal antibody targeting proprotein convertase subtilisin/kexin type 9 (PCSK9), represents a significant therapeutic advancement in the management of hypercholesterolemia and has been approved for use in the Chinese population. Elevated low-density lipoprotein cholesterol (LDL-C) is a major risk factor for atherosclerotic cardiovascular disease, and traditional treatments often fall short due to challenges such as statin intolerance. Clinical trials have demonstrated that tafolecimab can effectively reduce LDL-C levels, achieving reductions of over 60% in many patients. It also improves other lipid parameters, including lipoprotein(a) [Lp(a)], non–high-density lipoprotein cholesterol (non–HDL-C), and apolipoprotein B (ApoB). It has a favorable safety profile, primarily characterized by mild to moderate adverse events. The long-acting formulation of tafolecimab enables less frequent dosing, thereby promoting compliance. As cardiovascular diseases continue to escalate globally, tafolecimab holds promise not only for patients in China but also for broader international applications, representing a critical advancement in the evolving landscape of lipid-lowering therapies.
PubDate: 2025-04-01
- The Tailored Biosimilar Approach: Expectations and Requirements
-
Free pre-print version: Loading...
Rate this result:
What is this?
Please help us test our new pre-print finding feature by giving the pre-print link a rating.
A 5 star rating indicates the linked pre-print has the exact same content as the published article.
Abstract: Regulatory approval of biosimilar medicines currently requires a combination of physicochemical and functional testing, pharmacokinetic data, and clinical efficacy studies (CES). In this article, we discuss the tailored biosimilar approach, which represents an evolution in regulatory thinking by moving away from the default requirement for CES in biosimilar approval. We explore how physicochemical and functional data can be predictive for clinical performance and address the limitations of CES for regulatory decision-making. We argue that, in most cases, the combination of a robust package of physicochemical and functional testing, with appropriately designed pharmacokinetic studies provides sufficient evidence to establish biosimilarity. Additionally, we provide our perspective on the requirements, expectations, and exceptions for future biosimilar approvals, outlining specific scenarios where additional clinical evidence may be necessary. These include cases where the mechanism of action is unknown or poorly characterized, when product heterogeneity cannot be adequately characterized, or where relevant safety or immunogenicity concerns arise with the reference product or biosimilar candidate. Finally, we aim to clarify the remaining concerns surrounding the tailored biosimilar approach, providing insights into the potential to streamline biosimilar development and regulatory approval.
PubDate: 2025-04-01
- Cosibelimab: First Approval
-
Free pre-print version: Loading...
Rate this result:
What is this?
Please help us test our new pre-print finding feature by giving the pre-print link a rating.
A 5 star rating indicates the linked pre-print has the exact same content as the published article.
Abstract: Cosibelimab (UNLOXCYT; cosibelimab-ipdl) is an anti-PD-L1 antibody developed by Checkpoint Therapeutics for the treatment of advanced cancers, including metastatic or locally advanced cutaneous squamous cell carcinoma (mCSCC or laCSCC). Cosibelimab is the first approved anti-PD-L1 therapy in the USA for the treatment of adults with mCSCC or laCSCC who are not candidates for curative surgery or curative radiation. In contrast to other anti-PD-L1 antibodies, cosibelimab can induce antibody-dependent cell mediated cytotoxicity as it includes a functional Fc domain. This article summarizes the milestones in the development of cosibelimab leading to this first approval for this indication.
PubDate: 2025-04-01
- Classic Psychedelics for the Treatment of Depression: Potential Benefits
and Challenges-
Free pre-print version: Loading...
Rate this result:
What is this?
Please help us test our new pre-print finding feature by giving the pre-print link a rating.
A 5 star rating indicates the linked pre-print has the exact same content as the published article.
Abstract: There has been a recent resurgence in research on psychedelics as therapeutic agents for psychiatric conditions. This leading article outlines the studies to date of classic psychedelic treatments for treatment-resistant depression and major depression, including psilocybin, ayahuasca, dimethyltryptamine (DMT), and O-methyl-bufotenine (5-Me-O DMT). We discuss the potential of expanding treatment options for depression based on the data available, as well as the difficulties and limitations of research on psychedelics that make assessing that potential more challenging.
PubDate: 2025-03-24
- Current Drug Treatment for Acute and Recurrent Pericarditis
-
Free pre-print version: Loading...
Rate this result:
What is this?
Please help us test our new pre-print finding feature by giving the pre-print link a rating.
A 5 star rating indicates the linked pre-print has the exact same content as the published article.
Abstract: Pericarditis is the most frequent pericardial disease and presents with a relatively benign course when treated according to guideline-directed therapies at first presentation. Recurrence is the most frequent complication and may occur more frequently after a first episode, in patients with autoimmune etiology, in patients who received glucocorticoids, or after rapid (i.e., within 1 month) tapering of anti-inflammatory drugs. The therapeutic armamentarium for pericarditis includes high-dose nonsteroidal anti-inflammatory drugs (NSAIDs) that are tapered rapidly once symptoms are controlled. Colchicine is necessary to both relieve symptoms and reduce the rate of recurrences and is continued for at least 3–6 months. Low- to moderate-dose glucocorticoids are reserved for patients with a first recurrence for which NSAIDs and colchicine failed and/or who have an autoimmune disorder, with a slow tapering. Interleukin-1 blockers—anakinra, rilonacept, and goflikicept—are used as a third-line option in patients who cannot come off glucocorticoids or as second-line therapy after NSAIDs and colchicine in patients with contraindications to glucocorticoids or in those with high-risk features (i.e., multiple episodes, markedly elevated inflammatory markers, or extensive abnormalities at pericardial imaging) in whom treatment with glucocorticoids is unlikely to succeed.
PubDate: 2025-03-22
- Zanidatamab: First Approval
-
Free pre-print version: Loading...
Rate this result:
What is this?
Please help us test our new pre-print finding feature by giving the pre-print link a rating.
A 5 star rating indicates the linked pre-print has the exact same content as the published article.
Abstract: Zanidatamab (ZIIHERA®; zanidatamab- hrii), a bi-specific antibody targeting two non-overlapping epitopes of the human epidermal growth factor receptor 2 (HER2) protein, is being developed by Jazz Pharmaceuticals and BeiGene Ltd under license agreements from Zymeworks Inc., the developer of the molecule, for the treatment of HER2-expressing solid tumours. This article summarizes the milestones in the development of zanidatamab leading to this first accelerated approval for use in adults with previously treated, unresectable or metastatic HER2+ (IHC3+) biliary tract cancer (BTC), as detected by an FDA-approved test.
PubDate: 2025-03-20
- Correction: From Prospective Evaluation to Practice: Model-Informed Dose
Optimization in Oncology-
Free pre-print version: Loading...
Rate this result:
What is this?
Please help us test our new pre-print finding feature by giving the pre-print link a rating.
A 5 star rating indicates the linked pre-print has the exact same content as the published article.
PubDate: 2025-03-20
- Renal Safety Assessment of Lipid-Lowering Drugs: Between Old Certainties
and New Questions-
Free pre-print version: Loading...
Rate this result:
What is this?
Please help us test our new pre-print finding feature by giving the pre-print link a rating.
A 5 star rating indicates the linked pre-print has the exact same content as the published article.
Abstract: Cardiovascular disease (CVD) is the leading cause of morbidity and mortality in patients with chronic kidney disease (CKD). Quantitative and qualitative changes in plasma lipoprotein profiles are frequently associated with CKD and represent a significant risk factor for CVD in patients with CKD. Guidelines from the European Society of Cardiology and the European Atherosclerosis Society classify CKD as a condition with high or very high cardiovascular risk and set specific low-density lipoprotein cholesterol targets. Conventional lipid-lowering therapies (LLTs), such as statins, ezetimibe, and fibrates, can control CKD-associated dyslipidemia and, to some extent, prevent major atherosclerotic events in patients with CKD, but their use in clinical practice presents challenges because of the potential renal safety concerns. In recent years, novel therapies with the ability to lower both low-density lipoprotein cholesterol and triglycerides have been introduced to the market (e.g., proprotein convertase subtilisin/kexin type 9 inhibitors, bempedoic acid, lomitapide, volanesorsen) to improve our ability to control lipid abnormalities. However, their impact on kidney functionality has not been fully elucidated. The aim of this review was to examine the renal safety profiles of various LLTs, with special reference to novel medications, and to highlight important considerations and guidance for the use of these medications in overt CKD or in patients with some degree of renal function impairment. We underscore the lack of a comprehensive understanding of kidney safety, particularly for novel LLT therapies, and strongly emphasize the importance of future dedicated research to fully assess the safety and efficacy of these agents in patients with kidney abnormalities.
PubDate: 2025-03-19
- Stapokibart: First Approval
-
Free pre-print version: Loading...
Rate this result:
What is this?
Please help us test our new pre-print finding feature by giving the pre-print link a rating.
A 5 star rating indicates the linked pre-print has the exact same content as the published article.
Abstract: Stapokibart (Kangyueda®; 康悦达®) is a humanised IgG4 monoclonal antibody targeted against the interleukin (IL)-4 receptor alpha subunit (IL-4Rα). By binding IL-4Rα, stapokibart blocks the binding by (and subsequent signalling of) IL-4 and IL-13, two type 2 cytokines. Stapokibart is being developed by KeyMed Biosciences for the treatment of atopic dermatitis and other type 2 inflammatory diseases. In September 2024, stapokibart received its first approval, in China, for use in the treatment of moderate-to-severe atopic dermatitis in adults whose disease is poorly controlled by, or not suitable for, topical medications. Subsequently, stapokibart additionally received approval in China for use in the treatment of chronic rhinosinusitis with nasal polyps (December 2024) and for the treatment of seasonal allergic rhinitis (February 2025). Stapokibart is also under clinical evaluation for use in the treatment of moderate-to-severe asthma and chronic obstructive pulmonary disease, and prurigo nodularis. This article summarises the milestones in the development of stapokibart leading to this first approval for moderate-to-severe atopic dermatitis.
PubDate: 2025-03-17
- Safety of Anti-osteoarthritis Medications: A Systematic Literature Review
of Post-marketing Surveillance Studies-
Free pre-print version: Loading...
Rate this result:
What is this?
Please help us test our new pre-print finding feature by giving the pre-print link a rating.
A 5 star rating indicates the linked pre-print has the exact same content as the published article.
Abstract: Background Several meta-analyses of phase 3 randomized controlled trials (RCTs) were published in 2019, reassessing the safety of most anti-osteoarthritis (OA) medications, mainly on the basis of data from full safety reports. The current systematic review (SR) intends to provide complementary insights into the safety of anti-OA medications, using evidence from post-marketing safety surveillance studies. Methods The review protocol was registered with PROSPERO database (registration no. CRD42021227872). We followed the Cochrane methodology for SRs of interventions and comprehensively searched the Medline, CENTRAL, Scopus and TOXLINE databases from inception to November 2023, to include all post-marketing safety surveillance studies on any anti-OA medications. The outcomes of this SR were any adverse events (AEs) reported in the included studies. Results The literature search yielded 16,990 studies, of which 59 articles were ultimately included in the review. Most studies investigated non-steroidal anti-inflammatory drugs (NSAIDs, 27 studies, 28 reports) and intra-articular hyaluronic acid (IAHA, 16 studies). Symptomatic slow-acting drugs for osteoarthritis (SYSADOAs) were assessed in seven studies (one of which also assessed NSAIDs), and corticosteroid injections in four studies, while opioids and “herbal mixtures and other compounds” each were investigated respectively in three and two studies. Most of the studies were cohort studies (n = 44), others were case reports or case series (n = 12), RCTs (n = 2 reports of the same trial), or a case–control study (n = 1). The most commonly reported AEs with NSAIDs from cohort (sample sizes varied between 129 and 22,938 patients), RCT (21,645 patients with OA), and case–control (174 cases and 926 control patients with OA) studies were gastrointestinal (GI) and/or cardiovascular (CV) AEs, with specific AEs varying with individual NSAIDs. Where comparisons between NSAIDs were made, the overall literature shows a better or similar safety profile for celecoxib (at a daily dose of 200 mg, where dosage was reported) compared with other NSAIDs in regards to GI, CV and renal events. Other anti-OA medications with most common AEs reported from cohort studies were: IAHA (injection site pain); diacerein (GI AEs and reddish urine); avocado–soybean unsaponifiables (GI AEs); non-pharmaceutical-grade glucosamine and chondroitin (allergic reactions, GI disorders); opioids (hip fracture associated with long-term tramadol use among older adults; GI and nervous system disorders with hydrocodone); corticosteroid injections (increased risk of OA progression); herbal mixtures and other compounds (GI AEs). There were case reports or case series of specific AEs with various anti-OA medications that require further investigations in well-designed cohort studies before any definitive conclusions can be reached. Conclusions This SR confirms previous evidence on the safety of anti-OA medications from meta-analyses of phase 3 RCTs. Beyond the evidence here reported, the limitations of this research highlight the urgent need of a reporting guideline for post-marketing safety surveillance studies. Importantly, real-life safety surveillance of anti-OA medications should be strengthened with large cohort studies with control groups, and results should be disaggregated by disease populations for drugs common to several conditions.
PubDate: 2025-03-17
- Olezarsen: First Approval
-
Free pre-print version: Loading...
Rate this result:
What is this?
Please help us test our new pre-print finding feature by giving the pre-print link a rating.
A 5 star rating indicates the linked pre-print has the exact same content as the published article.
Abstract: Olezarsen (TRYNGOLZA™) is a first-in-class, GalNAc3-conjugated antisense oligonucleotide being developed by Ionis Pharmaceuticals for the treatment of familial chylomicronaemia syndrome (FCS) and severe hypertriglyceridaemia. It binds to apoC-III mRNA, promoting its degradation and lowering serum apoC-III protein levels. This action lowers triglyceride levels by enhancing the clearance of plasma triglyceride-rich lipoproteins such as chylomicrons and very-low-density lipoproteins. GalNAc3 conjugation improves hepatocyte uptake, which helps localise oligonucleotide treatment to the liver; this enables lower dosing, reduced injection volume and frequency, potentially reducing the risk of adverse events such as thrombocytopenia. Olezarsen received its first approval in the USA on 19 December 2024 under priority review as an adjunct to diet to reduce triglycerides in adults with FCS. This article summarizes the milestones in the development of olezarsen leading to this first approval for FCS.
PubDate: 2025-03-13
- Revumenib: First Approval
-
Free pre-print version: Loading...
Rate this result:
What is this?
Please help us test our new pre-print finding feature by giving the pre-print link a rating.
A 5 star rating indicates the linked pre-print has the exact same content as the published article.
Abstract: Revumenib (Revuforj®) is an oral, first-in-class menin inhibitor developed by Syndax Pharmaceuticals for the treatment of KMT2A-rearranged (KMT2Ar) acute leukaemia, NPM1-mutated (NPM1m) acute myeloid leukaemia (AML) and solid tumours. The interaction between menin and the KMT2A protein complex leads to aberrant gene expression, driving leukaemogenic transcription. By blocking this interaction, revumenib promotes differentiation and exerts antileukaemic activity in KMT2Ar acute leukaemias and other menin inhibition-sensitive leukaemias. Revumenib received its first approval on 15 November 2024 in the USA for the treatment of relapsed or refractory (R/R) acute leukaemia with a KMT2A translocation in adult and paediatric patients 1 year and older. This article summarizes the milestones in the development of revumenib leading to this first approval.
PubDate: 2025-03-12
|
Subscription journal
