Agamree (vamorolone) / ReveraGen, Santhera, Catalyst Pharma, Xoma - LARVOL DELTA

From case to caution: hyponatremia in a patient with Duchenne muscular dystrophy on vamorolone and lessons for clinicians. (PubMed, Neuromuscul Disord) - "We report a patient with DMD who switched from daily deflazacort to vamorolone at age 13.0 years. Six months later, he developed fever, vomiting, and diarrhea, necessitating oral hydrocortisone stress dosing...Lisinopril and spironolactone were withheld. This case raised concern for an adrenal crisis, manifested by mineralocorticoid deficiency. It highlights the importance of stress dosing and checking electrolytes during illness when on vamorolone, especially if on cardiac medications, and education of patients, families, and multidisciplinary healthcare providers."

Journal • Cardiovascular • Duchenne Muscular Dystrophy • Endocrine Disorders • Genetic Disorders • Heart Failure • Muscular Dystrophy • Nephrology • Renal Disease

Rethinking Corticosteroid Therapy in Pediatric Neurology. (PubMed, J Inflamm Res) - "Several therapeutic innovations, including selective glucocorticoid receptor modulators (such as vamorolone) and intra-erythrocyte dexamethasone delivery, have shown promising safety profiles in Duchenne muscular dystrophy and ataxia telangiectasia, respectively. Beyond the well-established role of corticosteroids in infantile epileptic spasms syndrome, recent evidence suggests that corticosteroids may benefit a substantial proportion of patients with various forms of epilepsy that are resistant to conventional antiseizure medications. Further research is warranted to define the optimal use of corticosteroids with respect to dosing, formulation, timing, and route of administration in various pediatric neurological disorders."

Journal • Ataxia • CNS Disorders • Duchenne Muscular Dystrophy • Epilepsy • Genetic Disorders • Immunology • Inflammation • Movement Disorders • Muscular Dystrophy • Pediatrics • Primary Immunodeficiency

Results of a phase II open-label, multiple-dose study of vamorolone (VBP15-006) in 7- to < 18-year-old boys with duchenne muscular dystrophy. (PubMed, J Neurol) - P, P2 | "Vamorolone demonstrated a consistent safety profile in 7- to < 18-year-old boys with DMD. Switching to 6 mg/kg/day vamorolone appeared to mitigate adrenal insufficiency risk. There was no negative effect on growth, and catch-up growth occurred in previously CS-treated individuals switching to vamorolone."

Journal • P2 data • Duchenne Muscular Dystrophy • Endocrine Disorders • Genetic Disorders • Muscular Dystrophy • Nephrology • Renal Disease

Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy-SUMMIT (clinicaltrials.gov) - P=N/A | N=250 | Recruiting | Sponsor: Catalyst Pharmaceuticals, Inc. | Trial completion date: Nov 2030 ➔ Feb 2032 | Trial primary completion date: Sep 2030 ➔ Feb 2032

Trial completion date • Trial primary completion date • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Drug-induced urinary incontinence in pediatric patients: A disproportionality analysis of the FDA Adverse Event Reporting System. (PubMed, Medicine (Baltimore)) - "Montelukast was the most frequently reported drug (91 cases), followed by risperidone (74 cases)...The top 3 drugs with the strongest signals were glipizide (ROR = 141.1, 95% CI 37.4-532.4; PRR = 102.9), ropinirole (ROR = 125.7, 95% CI 49.8-317.2; PRR = 94.5), and sodium oxybate (ROR = 19.8, 95% CI 15.1-26.0; PRR = 18.9). Twenty-two drugs, such as montelukast, sodium oxybate, and sertraline, had a higher risk of UI in children than in adults. We also discovered unexpected associations, such as miglustat, tisagenlecleucel, and vamorolone. This study systematically identified a range of drugs, including several unexpected ones, associated with PUI. These findings enhance the understanding of the safety profile of drugs associated with PUI and provide important insights for optimizing clinical practice in pediatrics."

Adverse events • Journal • Pediatrics • Urinary Incontinence • Urology

Santhera Receives Swissmedic Approval of AGAMREE (Vamorolone) for the Treatment of Duchenne Muscular Dystrophy (GlobeNewswire) - "The Swissmedic approval of AGAMREE was based on the data from the pivotal Phase 2b VISION-DMD study and assessment results from the European Medicines Agency. Following approval, the Company has been granted a 15-year exclusivity period under Swiss orphan drug status. Santhera retains exclusive distribution rights for AGAMREE in Switzerland and anticipates commercial launch in H2 2026, following the completion of national pricing and reimbursement procedures."

Approval • Launch Europe • Duchenne Muscular Dystrophy

Adrenal Suppression in Duchenne Muscular Dystrophy: Management Strategies Incorporating Novel Steroid Vamorolone. (PubMed, J Endocr Soc) - "It also emphasizes the importance of educating the DMD community and health care providers about the recognition and management of adrenal suppression and outlines critical concepts for clinicians managing adrenal suppression risk, tapering GCs, and transitioning from classic GC therapy to vamorolone. The key principles of managing adrenal suppression due to classic GCs and novel vamorolone therapy highlighted in this perspective are expected to enhance clinical practice, mitigate mortality, and optimize health outcomes for individuals with DMD."

Journal • Duchenne Muscular Dystrophy • Endocrine Disorders • Genetic Disorders • Muscular Dystrophy • Nephrology • Renal Disease

Endocrine and metabolic complications in a national cohort of Slovene children and adolescents with Duchenne muscular dystrophy: real-world criteria for transition to vamorolone therapy. (PubMed, Front Endocrinol (Lausanne)) - "Two patients were corticosteroid-naive, the rest treated with deflazacort (average treatment duration 5.4, range 1.1-11.3 yrs). We propose that ambulatory, non-corticosteroid naive patients with pathological fractures, markedly reduced bone density, short stature, or significant growth deceleration could be considered for the therapeutic transition. Accordingly, we established national clinical criteria to guide individualized therapeutic transitions, aimed at optimizing clinical outcomes and ensuring efficient allocation of healthcare resources."

Journal • Real-world evidence • Diabetes • Duchenne Muscular Dystrophy • Genetic Disorders • Metabolic Disorders • Muscular Dystrophy • Musculoskeletal Diseases • Obesity • Orthopedics • Pediatrics • Type 2 Diabetes Mellitus

GUARDIAN: A Study on Safety and Effectiveness of Long-term Treatment With Vamorolone in Boys With Duchenne Muscular Dystrophy (clinicaltrials.gov) - P4 | N=80 | Active, not recruiting | Sponsor: Santhera Pharmaceuticals | Recruiting ➔ Active, not recruiting

Enrollment closed • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Corticosteroid treatment in Duchenne muscular dystrophy. (PubMed, Arch Pediatr) - "Corticoids are started and maintained at the theoretical dose of 0.75 mg/kg/day of prednisone/prednisolone or 0.9 mg/kg/day of deflazacort, adjusting the dose to weight if the benefit-risk ratio favors treatment. The only reasons to discontinue treatment when the patient and family are adhering to it are the uncontrolled side effects. Thus, in many countries including France, as of late 2025, the only routinely prescribed symptomatic treatment for DMD remains conventional corticosteroids, which will be discussed in this article, followed by a description of vamorolone, a dissociative steroidal compound, and givinostat, a histone deacetylase inhibitor."

Journal • Review • Cardiomyopathy • Cardiovascular • Duchenne Muscular Dystrophy • Genetic Disorders • Infectious Disease • Muscular Dystrophy • Obesity • Osteoporosis • Respiratory Diseases • Rheumatology

Circulating protein biomarkers identified in two independent clinical trial cohorts of glucocorticoid-naive Duchenne muscular dystrophy patients. (PubMed, Sci Rep) - "Here, we quantified the relative abundance of 7,289 serum proteins using SomaScan proteomics in pre-treatment samples from 51 boys with DMD (aged 4 to <7) and 13 healthy controls from the VISION DMD (VBP15-004) trial...This larger-scale, multi-clinical trial-based cohort study in untreated DMD boys substantially expands the catalog of circulating biomarkers, highlighting early-stage pathological processes. These findings can help identify new therapeutic targets and develop clinically actionable biomarkers to assess disease progression and response to therapies."

Biomarker • Journal • Duchenne Muscular Dystrophy • Genetic Disorders • Inflammation • Muscular Dystrophy

Exposure-response of serum biomarkers to vamorolone, a dissociative corticosteroidal anti-inflammatory drug, in 4- to <7-year children. (PubMed, Steroids) - "The serum proteome pharmacodynamics of the response to vamorolone was defined."

Biomarker • Journal • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy • AKT1 • CCL2 • CCL21 • CCL22 • CXCL12 • IL12B • IL17A • IL23A • KLK11 • KLK7 • KLK8

Health Canada Approves AGAMREE (vamorolone) as the First Treatment for Duchenne Muscular Dystrophy in Canada (PRNewswire) - "The Health Canada approval of AGAMREE was based on the data from the pivotal Phase 2b VISION-DMD study as supplemented with safety information collected from four open-label studies, including extension studies. In these trials, AGAMREE was administered at doses ranging from 2 to 6 mg/kg/day, extending for a period of up to 48 months."

Canada approval • Duchenne Muscular Dystrophy

A Study to Assess Vamorolone in Becker Muscular Dystrophy (BMD) (clinicaltrials.gov) - P2 | N=46 | Completed | Sponsor: ReveraGen BioPharma, Inc. | Active, not recruiting ➔ Completed

Trial completion • Becker Muscular Dystrophy • Duchenne Muscular Dystrophy • Muscular Dystrophy

Mineralocorticoid receptor antagonism of vamorolone: Evidence from LIONHEART and VISION-DMD clinical trials. (PubMed, Steroids) - "In LIONHEART, 30 healthy adult males were randomized 1:1:1 to vamorolone 20 mg/kg, eplerenone 200 mg, or no treatment arms. A fludrocortisone challenge was administered between -9 h and 24 h after treatment...In VISION-DMD, boys with DMD aged 4-7 years were treated with vamorolone 2 or 6 mg/kg/d for 48 weeks or with prednisone 0.75 mg/kg/d or placebo for 24 weeks followed by vamorolone 2 or 6 mg/kg/d for 20 weeks following a 4-week washout...The VISION-DMD results showed vamorolone-specific increases in renin serum levels, as well as klotho, and calcium carrier proteins fetuin A and B, consistent with an MR antagonist effect. The available data confirm the MR antagonistic effect of vamorolone in humans."

Journal • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Test-Retest Reliability of Motor Function and Myometry Outcomes From the Vamorolone Trials in Duchenne Muscular Dystrophy. (PubMed, Neurol Genet) - "We found NSAA, RWV, and CLIMBV to be the most reliable. This should be considered in the context of sensitivity to drug effect, and clinical meaningfulness of changes observed: all 5 motor outcomes were sensitive to drug effect of vamorolone and prednisone, whereas myometry was not."

Journal • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Clinical studies of vamorolone: An anti-inflammatory corticosteroid with unique structure/activity relationships: not metabolized by HSD11B's, and a MR antagonist (ASBMR 2025) - No abstract available

Clinical • Orthopedics

Duchenne muscular dystrophy: Evolving therapeutic strategies and multidimensional evaluation approaches. (PubMed, Brain Dev) - "In this study, a comprehensive review of the current literature was conducted focusing on mutation-specific therapies (exon skipping and gene therapy), non-mutation-specific pharmacological interventions (steroids, vamorolone, and histone deacetylase inhibitors), and emerging modalities such as cell-based therapy...Vamorolones and givinostat are alternative drugs with improved safety profiles...Multidimensional stage-specific evaluations are essential to capture functional changes and their therapeutic effects. Ongoing research on gene-, pharmacological-, and cell-based therapies, coupled with robust outcome assessments, may further improve patient care and QOL."

Journal • Review • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy

Design and development of glucocorticoid receptor modulators. (PubMed, Trends Pharmacol Sci) - "A novel GRM, the dissociated steroid vamorolone, received marketing approval in 2024, confirming that altering the transrepression-transactivation profile is a valid strategy. Here, we review GR-mediated gene regulation and the transrepression-transactivation profile of GCs in relation to their anti-inflammatory efficacy and AEs. We highlight technological advances driving the design/development of novel GRMs, such as selective GR agonists and modulators (SEGRAMs), and provide insights into their mechanism of action."

Journal • Review

Vamorolone Versus Traditional Glucocorticoids in Duchenne Muscular Dystrophy: A Review and Meta-Analysis of Efficacy and Safety. (PubMed, J Pharm Technol) - "Search terms included (Vamorolone) AND (Corticosteroids OR Glucocorticoids OR Prednisone) AND (Duchenne Muscular Dystrophy OR related terms). Safety outcomes showed enhanced height percentile (MD = 16.28, 95% CI = 6.31-26.25, P = .001) and bone biomarkers (osteocalcin: MD = 15.68, P < .00001; P1NP: MD = 158.34, P < .00001), with no significant body mass index-z score difference. Vamorolone offers comparable efficacy with traditional glucocorticoids in DMD, with improved motor function and safer profiles in growth and bone health, suggesting its potential to transform DMD management."

Journal • Retrospective data • Review • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Pharmacological Insights on USFDA-Approved Novel Drug Therapies in the Year 2023. (PubMed, Curr Drug Discov Technol) - "The novel drug therapies approved by the USFDA hold significant potential to enhance the patient's care by providing advanced treatment modalities. This manuscript, reporting the comprehensive description of therapeutic aspects of the mentioned new drug therapies, underscores the commitment of the pharmaceutical sector to address the unmet medical needs and reshape the landscape of the healthcare service system by instilling optimism among patients and healthcare providers."

FDA event • Journal • Pediatrics • Rare Diseases

A phase II open-label, multiple dose study to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and exploratory efficacy of vamorolone in boys ages 2 to <4 years and 7 to <18 years with Duchenne Muscular Dystrophy (DMD) (clinicaltrialsregister.eu) - P2 | N=54 | Sponsor: Santhera Pharmaceuticals (Schweiz) AG

New P2 trial • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

The Long-Term Effects of Duchenne Muscular Dystrophy and its Treatment on Bone Strength (ESPE-ESE 2025) - "In an effort to attenuate the adverse effects of CS therapy in DMD, regimens alternative to daily CS have been implemented (such as prednisone 10 days on/off); however, this saltatory approach comes at a cost to muscle strength. Recently, a novel steroid (vamorolone) has been studied in DMD...In an effort to improve the convenience of osteoporosis treatment and minimize first exposure adverse events, denosumab has been tried in this setting...Overall, it is recognized that DMD is the ideal setting to consider not only prevention of first-ever fractures but also osteoanabolic agents, given the limitations of bisphosphonate monotherapy in low bone turnover states such as DMD. Strategic planning for primary osteoporosis prevention is currently underway by members of The OPTIMIZE DMD Consortium, an international working group dedicated to education, advocacy and research in endocrine and bone health care for people with DMD."

Acute Respiratory Distress Syndrome • Duchenne Muscular Dystrophy • Endocrine Disorders • Gene Therapies • Genetic Disorders • Muscular Dystrophy • Musculoskeletal Diseases • Nephrology • Orthopedics • Osteoporosis • Pediatrics • Renal Disease • Respiratory Diseases • Rheumatology

New perspectives - Glucocorticoids/vamorolone (ESPE-ESE 2025) - No abstract available

Duchenne Muscular Dystrophy

Santhera shares updates on commercial rollout of AGAMREE (GlobeNewswire) - "Santhera Pharmaceuticals...announces the latest progress on the commercial rollout of AGAMREE for the treatment of Duchenne Muscular Dystrophy (DMD)....Funding from National Health Service (NHS) England is now available for AGAMREE for the treatment of DMD, enabling prescribing in England to commence."

Reimbursement • Duchenne Muscular Dystrophy