UK national guidance of clinical monitoring of bone and endocrine outcomes in individuals with DMD on vamorolone and switching from classic glucocorticoid to vamorolone UK national guidance of clinical monitoring of bone and endocrine outcomes in individuals with DMD on vamorolone and switching from classic glucocorticoid to vamorolone: Recommendations of the Endocrine & Bone Working Group of DMD Care UK, 2025 Background: Monitoring of bone and endocrine outcomes in individuals with Duchenne Muscular Dystrophy (DMD) on vamorolone should follow existing clinical recommendations for all individuals with DMD on classic glucocorticoids (prednisolone or deflazacort) as part of the recommendations of the Bone and Endocrine Working Group of DMD Care UK. Adrenal suppression (1,2), vertebral
Vamorolone (Agamree) - Duchenne muscular dystrophy 1 Published 13 January 2025 1 SMC2721 vamorolone oral suspension (Agamree®) Santhera Pharmaceuticals (Deutschland) GmbH 06 December 2024 The Scottish Medicines Consortium (SMC) has completed its assessment of the above product and advises NHS Boards and Area Drug and Therapeutic Committees (ADTCs) on its use in NHSScotland. The advice is summarised as follows: ADVICE: following a full submission assessed under the orphan medicine process vamorolone (Agamree®) is accepted for use within NHSScotland. Indication under review: treatment of Duchenne muscular dystrophy (DMD) in patients aged 4 years and older. In a randomised, double-blind, phase IIb study, treatment with vamorolone resulted in a significant improvement in the change in time
Vamorolone (Agamree) - Duchenne muscular dystrophy Skip to main contentSkip to FDA SearchSkip to footer links An official website of the United States government Here's how you know U.S. Food and Drug Administration Search MenuSearch FDASubmit search Home Drugs Drug Approvals and Databases Drugs@FDADrug Approval Package: AGAMREEShareTweetLinkedinEmailPrintCompany: Santhera Pharmaceuticals
Vamorolone for Duchenne Muscular Dystrophy Vamorolone for Duchenne Muscular Dystrophy Prepared by Michela Guglieri, John Walton Muscular Dystrophy Research Centre, Newcastle University and Newcastle upon Tyne Hospitals Trust Reviewed by members of the Corticosteroid Working Group and the Bone and Endocrine Working Group of DMD Care UK (www.dmdcareuk.org) Finalised on 29th January 2025 Vamorolone is a synthetic corticosteroid designed to treat Duchenne muscular dystrophy (DMD) and potentially other inflammatory conditions. This is a summary of current evidence on vamorolone in DMD, based on published data. A systematic review of current evidence of efficacy and safety in DMD has been recently published1. Indication Duchenne muscular dystrophy, age 4 years and older (based on NICE recommendations, 10th
Efficacy and Safety of Vamorolone Over 48 Weeks in Boys With Duchenne Muscular Dystrophy: A Randomized Controlled Trial. Vamorolone is a dissociative agonist of the glucocorticoid receptor that has shown similar efficacy and reduced safety concerns in comparison with prednisone in Duchenne muscular dystrophy (DMD). This study was conducted to determine the efficacy and safety of vamorolone over 48 weeks and to study crossover participants (prednisone to vamorolone; placebo to vamorolone). A randomized, double-blind, placebo-controlled and prednisone-controlled clinical trial of 2 doses of vamorolone was conducted in participants with DMD, in the ages from 4 years to younger than 7 years at baseline. The interventions were 2 mg/kg/d of vamorolone and 6 mg/kg/d of vamorolone for 48 weeks
Adrenal suppression from vamorolone and prednisone in Duchenne muscular dystrophy: results from the phase 2b clinical trial. Vamorolone, a novel "dissociative" steroid, demonstrated similar efficacy in muscle function relative to prednisone 0.75 mg/kg/day but improved linear growth and bone turnover markers in a randomized trial of pediatric Duchenne muscular dystrophy (DMD). To determine the frequency of adrenal suppression (AS) induced by vamorolone and prednisone in pediatric DMD, and to assess cortisol thresholds using a monoclonal antibody immunoassay. Post-hoc analysis of cortisol levels was performed on data from a randomized, double-blind, placebo- and prednisone-controlled 24-week trial of vamorolone with a 24-week crossover extension. Morning and ACTH-stimulated cortisol levels were
Efficacy and Safety of Vamorolone in Duchenne Muscular Dystrophy: A 30-Month Nonrandomized Controlled Open-Label Extension Trial. Vamorolone is a synthetic steroidal drug with potent anti-inflammatory properties. Initial open-label, multiple ascending dose-finding studies of vamorolone among boys with Duchenne muscular dystrophy (DMD) found significant motor function improvement after 6 months treatment in higher-dose (ie, ≥2.0 mg/kg/d) groups. To investigate outcomes after 30 months of open-label vamorolone treatment. This nonrandomized controlled trial was conducted by the Cooperative International Neuromuscular Research Group at 11 US and non-US study sites. Participants were 46 boys ages 4.5 to 7.5 years with DMD who completed the 6-month dose-finding study. Data were analyzed from July
Efficacy and Safety of Vamorolone vs Placebo and Prednisone Among Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial. Corticosteroidal anti-inflammatory drugs are widely prescribed but long-term use shows adverse effects that detract from patient quality of life. To determine if vamorolone, a structurally unique dissociative steroidal anti-inflammatory drug, is able to retain not previously treated with corticosteroids. The study included 4 groups: placebo; prednisone, 0.75 mg/kg per day; vamorolone, 2 mg/kg per day; and vamorolone, 6 mg/kg per day. Study outcomes monitored (1) efficacy, which included motor outcomes (primary: time to stand from supine velocity in the vamorolone, 6 mg/kg per day, group vs placebo; secondary: time to stand from supine velocity [vamorolone, 2 mg/kg
Efficacy and safety of vamorolone in Duchenne muscular dystrophy: An 18-month interim analysis of a non-randomized open-label extension study. Treatment with corticosteroids is recommended for Duchenne muscular dystrophy (DMD) patients to slow the progression of weakness. However, chronic corticosteroid treatment causes significant morbidities. Vamorolone is a first-in-class anti-inflammatory investigational drug that has shown evidence of efficacy in DMD after 24 weeks of treatment at 2.0 or 6.0 mg/kg/day. Here, open-label efficacy and safety experience of vamorolone was evaluated over a period of 18 months in trial participants with DMD. A multicenter, open-label, 24-week trial (VBP15-003) with a 24-month long-term extension (VBP15-LTE) was conducted by the Cooperative International Neuromuscular
Vamorolone trial in Duchenne muscular dystrophy shows dose-related improvement of muscle function. To study vamorolone, a first-in-class steroidal anti-inflammatory drug, in Duchenne muscular dystrophy (DMD). An open-label, multiple-ascending-dose study of vamorolone was conducted in 48 boys with DMD (age 4-<7 years, steroid-naive). Dose levels were 0.25, 0.75, 2.0, and 6.0 mg/kg/d in an oral suspension formulation (12 boys per dose level; one-third to 10 times the glucocorticoid dose in DMD). The primary goal was to define optimal doses of vamorolone. The primary outcome for clinical efficacy was time to stand from supine velocity. Oral administration of vamorolone at all doses tested was safe and well tolerated over the 24-week treatment period. The 2.0-mg/kg/d dose group met the primary
Muscle miRNAome shows suppression of chronic inflammatory miRNAs with both prednisone and vamorolone Corticosteroids are highly prescribed and effective anti-inflammatory drugs but the burden of side effects with chronic use significantly detracts from patient quality of life, particularly in children. Developing safer steroids amenable to long-term use is an important goal for treatment of chronic inflammatory diseases such as Duchenne muscular dystrophy (DMD). We have developed vamorolone (VBP15), a first-in-class dissociative glucocorticoid receptor (GR) ligand that shows the anti-inflammatory efficacy of corticosteroids without key steroid side effects in animal models. miRNAs are increasingly recognized as key regulators of inflammatory responses. To define effects of prednisolone
The corticosteroid compounds prednisolone and vamorolone do not alter the nociception phenotype and exacerbate liver injury in sickle cell mice Clinicians often hesitate prescribing corticosteroids to treat corticosteroid-responsive conditions in sickle cell disease (SCD) patients because their use can be associated with complications (increased hospital readmission, rebound pain, strokes , avascular necrosis, acute chest syndrome). Consequently, SCD patients may receive suboptimal treatment for corticosteroid-responsive conditions. We conducted a preclinical trial of dissociative (vamorolone) and conventional (prednisolone) corticosteroid compounds to evaluate their effects on nociception phenotype, inflammation, and organ dysfunction in SCD mice. Prednisolone and vamorolone had
Phase 1 trial of vamorolone, a first-in-class steroid, shows improvements in side effects via biomarkers bridged to clinical outcomes Glucocorticoid drugs are highly effective anti-inflammatory agents, but chronic use is associated with extensive pharmacodynamic safety concerns that have a considerable negative impact on patient quality of life. Vamorolone (VBP15) is a first-in-class steroidal -in-human Phase 1 clinical trials (86 healthy adult males), with single ascending doses (0.1-20.0 mg/kg), and multiple ascending doses (1.0-20 mg/kg/day; 14 days treatment). Vamorolone was well-tolerated at all dose levels. Vamorolone showed pharmacokinetic and metabolism profiles similar to prednisone. Biomarker studies showed loss of side effects of traditional glucocorticoid drugs (bone fragility
A Study on Safety and Effectiveness of Long-term Treatment With Vamorolone in Boys With Duchenne Muscular Dystrophy This study aims to assess safety and effectivness of long-term treatment with vamorolone in boys with Duchenne Muscular Dystrophy (DMD) who have completed prior studies with vamorolone. All subjects in this study have completed previous studies with vamorolone and continued to receive vamorolone under special programs: Compassionate Use Program \[CUP\], Named Patient Program \[NPP\] or Expanded Access Protocol \[EAP\]. All subjects will continue treatment with vamorolone under Guardian protocol instead. The primary objective of this study is to evaluate the safety of long-term treatment with vamorolone in boys with Duchenne Muscular Dystrophy regarding vertebral fractures
Efficacy and safety of different doses of vamorolone in boys with Duchenne muscular dystrophy: a systematic review and network meta-analysis PROSPERO International prospective register of systematic reviews Print | PDF PROSPERO This information has been provided by the named contact for this review. CRD has accepted this information in good faith and registered the review in PROSPERO
Efficacy and Safety of Vamorolone in Duchenne Muscular Dystrophy: A Systematic Review and Meta-Analysis PROSPERO International prospective register of systematic reviews Print | PDF PROSPERO This information has been provided by the named contact for this review. CRD has accepted this information in good faith and registered the review in PROSPERO. The registrant confirms that the information
Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy. The goal of this observational study is to follow patients being treated with the FDA approved drug AGAMREE® in male patients 2 years of age or older with Duchenne's Muscular Dystrophy for long term safety and quality of life. This is a multi-center, observational, prospective
Effect and safety of vamorolone in Duchenne muscular dystrophy: a systematic review and meta-analysis. PROSPERO International prospective register of systematic reviews Print | PDF PROSPERO This information has been provided by the named contact for this review. CRD has accepted this information in good faith and registered the review in PROSPERO. The registrant confirms that the information
Investigating the impact of different doses of Vamorolone in patients with Duchenne muscular dystrophy a systemic review and meta-analysis PROSPERO International prospective register of systematic reviews Print | PDF PROSPERO This information has been provided by the named contact for this review. CRD has accepted this information in good faith and registered the review in PROSPERO