Agamree (vamorolone) / ReveraGen, Santhera, Catalyst Pharma, Nxera Pharma, Ligand, Angelini Group - LARVOL DELTA

A structured approach to address variability in the global management of adrenal insufficiency in Duchenne muscular dystrophy: the 2025 PJ Nicholoff steroid protocol. (PubMed, Front Neurol) - "Management is increasingly complex because DMD treatment now includes multiple GC agents, including prednisone/prednisolone, deflazacort, and vamorolone, as well as daily, intermittent, and transition regimens that differ across countries and clinical settings. The updated protocol incorporates newer therapies such as vamorolone and emphasizes anticipatory education, written stress-steroid plans (including a weight-based approach), safe GC tapering, confirmation of hypothalamic-pituitary-adrenal axis recovery, and careful management of transitions between GC agents and regimens. This structured approach aims to support safer and more consistent adrenal suppression management for individuals with DMD receiving chronic GC therapy through six critical concepts."

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

Adrenal insufficiency in individuals with Duchenne muscular dystrophy treated with glucocorticoids: Insights from the past, current challenges, and future directions. (PubMed, J Neuromuscul Dis) - "Despite a rapidly evolving therapeutic landscape that now includes multiple disease modifying treatments including exon skipping medications, microdystrophin gene therapy, and givinostat, high dose glucocorticoids (GCs) initiated at a young age remain central to the standard of care for DMD...The approach described in this document also applies to individuals with related dystrophinopathies including Becker muscular dystrophy and female manifesting carriers treated with GCs, recognizing that GCs are not standard of care for these conditions and are prescribed on an individualized basis at some centers to severely affected individuals. Key themes discussed include best practices for individual and caregiver education about adrenal suppression, creation and implementation of stress steroid plans, safe transition between GC treatment regimens, and discontinuation of GC therapy."

Journal • Review • Becker Muscular Dystrophy • Duchenne Muscular Dystrophy • Endocrine Disorders • Gene Therapies • Genetic Disorders • Muscular Dystrophy • Nephrology • Renal Disease • Respiratory Diseases

Observational Study of Vamorolone in Children with Duchenne Muscular Dystrophy (ChiCTR) - P4 | N=45 | Not yet recruiting | Sponsor: West China Second Hospital, Sichuan University; West China Second Hospital, Sichuan University

New P4 trial • Becker Muscular Dystrophy • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Multi-bank virtual screening and comparative molecular dynamics of steroid mimetics targeting the GVR protein for Duchenne muscular dystrophy | Poster Board #1176 (ACS-Fall 2026) - "We performed comparative docking and MD simulations utilizing two control ligands—prednisone and vamorolone—against a diverse set of candidates sourced from the Asinex Steroid Mimetics and ChemDiv DMD Therapeutics libraries. These simulations provided insights into the time-dependent stability of protein-ligand interactions and the conformational flexibility of the GVR active site. Our results identified several high-affinity hits that exhibit superior binding energetics and structural persistence compared to traditional corticosteroids, suggesting promising new directions for DMD drug development."

Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Integrating Genetic Modifier Genotype With Serum Proteomics in Duchenne Muscular Dystrophy Clinical Trials Links LTBP4 Genetic Modifier to IL-23/CD93 Pathways in Muscle. (PubMed, Am J Med Genet A) - P2, P2a, P2b | "Participants in clinical trials (VBP15-002/003 [n = 48]; VBP15-004 [n = 121]; DNA available for n = 110) were genotyped for eight published genetic modifier loci, and associations of genotypes with baseline motor function defined via an age-adjusted linear model...In contrast, the DYNLT5 genotype was associated with proteosome and chaperonin pathways. Trial Registration: clinicaltrials.gov identifier: NCT02760264, NCT02760277, NCT03439670."

Journal • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy • CD93 • IL23A • IL6

Prednisone, not vamorolone, suppresses novel serum bone and cartilage biomarkers associated with growth failure in children with Duchenne muscular dystrophy. (PubMed, Sci Rep) - P2b | "Our clinical trial studies suggest that the 10 novel, corticosteroid-related biomarkers likely reflect the induction of apoptosis in terminal hypertrophic chondrocytes and osteoblasts by traditional corticosteroids. Vamorolone may spare chondrocytes and osteoblasts from this apoptosis, consistent with maintenance of normal growth in vamorolone-treated children.Trial registration The clinical trial registration (clinicaltrials.gov) is NCT03439670 ( https://www.clinicaltrials.gov/study/NCT03439670 )."

Biomarker • Clinical • Journal • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy • ACAN • COL1A2 • COL2A1 • COL6A1

Vamorolone for Duchenne Muscular Dystrophy: A Cross-Trial Efficacy Comparison With Classic Corticosteroids From the FOR-DMD Trial. (PubMed, Neurology) - No abstract available

Journal • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Expanding vamorolone treatment access for Canadians with Duchenne muscular dystrophy. (PubMed, Can J Neurol Sci) - No abstract available

Journal • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Pharmacodynamic Response of Circulating Proteins to Different Corticosteroid Types and Regimens in Duchenne Muscular Dystrophy (ICNMD 2026) - P3 | "Moreover, most newer therapies are used in combination with steroids (prednisone, deflazacort, and vamorolone). We studied the dose-response of serum proteins between intermittent and daily prednisone, and differential response to daily prednisone vs deflazacort in a large-scale, untargeted fashion. We validated some serum proteins previously reported as differentially responsive to prednisone vs deflazacort, but also identified newer differentially responsive biomarkers. Our work may help to better understand the mechanism of action of steroids, the predictive potential of these biomarkers and their importance to accelerated regulatory pathways, help refine therapies, define synergistic interactions between novel treatments and steroids, and potentially use as surrogate/secondary outcomes in trials."

PK/PD data • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy

Considerations for Assessing Efficacy in Steroid-Naïve Interventional Trials in Duchenne Muscular Dystrophy (ICNMD 2026) - "Define subgroups that could increase statistical power compared to an all-comers analysis. We used data from a randomized, double blinded, placebo-controlled registrational trial of vamorolone (VBP15-004), a dissociative steroid approved for DMD in boys over 2 years old, as well as data from the placebo arm of another published interventional study... While some steroid-naïve boys with DMD may show a marginal improvement in some motor outcomes in early age bins, none of the motor outcomes showed consistent strength improvement until <7.5 yrs in this typical age range of interest for interventional trials. The ≥ 5-second TTSTAND and 300m-400m subgroups were found to provide greater statistical power for a corticosteroid effect than an all-comers analysis. These findings may be important both for steroid-naïve interventional trial designs and steroid add-on trials."

Clinical • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Genetic Modifier LTBP4 Stratification of Proteomics Points to Interacting IL-23/IL-6/IL-17D Pathways in Severity of DMD (ICNMD 2026) - "For validated modifiers, we stratified serum profiles at baseline by genotype to identify candidate cellular responses associated with clinical severity. Participants in clinical trials (VBP15-002/003 n=48; VBP15-004 n=131) were genotyped for genetic modifier loci (n=110 genotyped)... Significant associations with improved baseline motor outcomes in young steroid naïve DMD children were found for LTBP4 and DYNLT5 loci IL-23, IL-6 and IL-17D interacting pathways may participate in disease progression."

IO biomarker • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy • ADRB2 • CD40 • IL23A • IL6 • SPP1 • TNFRSF10A

'Weak by Structure'-Limb Muscle Fibre Cytoarchitecture Remodelling During Critical Illness and Effects of Chaperone Co-Inducer BGP-15 and Dissociative Glucocorticoid VBP-15. (PubMed, Cells) - "VBP-15 reversed atrophy at day 10 in soleus but not in EDL fibres. Our study is the first to quantify myofibrillar remodelling in limb muscle fibres during ICU intervention in 3D and provides exploratory assessment of BGP-15 and VBP-15 treatments on aberrant remodelling in CIM."

Journal • Critical care • Muscular Atrophy • Myositis • CEACAM1

Post-marketing safety surveillance and signal characterization of the novel dissociative steroid Vamorolone in Duchenne muscular dystrophy: a comparative disproportionality analysis based on FAERS data. (PubMed, Front Pharmacol) - "To comprehensively evaluate the real-world post-marketing safety profile of the novel dissociative steroid Vamorolone and perform a comparative analysis against the traditional glucocorticoid deflazacort. Nevertheless, known risks and unexpected hypothesis-generating signals-such as notable HPA axis suppression and early-onset psychiatric disturbances-remain critical safety concerns. Clinicians should interpret these differential reporting patterns with caution and rigorously monitor neurobehavioral and adrenal health during the first three months, with further external validation warranted."

Journal • P4 data • CNS Disorders • Duchenne Muscular Dystrophy • Genetic Disorders • Infectious Disease • Mental Retardation • Muscular Dystrophy • Musculoskeletal Diseases • Orthopedics • Osteoporosis • Psychiatry • Rheumatology • Urinary Incontinence

Patient Characteristics and Use of Vamorolone in Adult Patients With DMD in Germany and Austria (ICNMD 2026) - "Six patients were corticosteroid (CS) naïve, 16 had prior prednisone and three prior deflazacort treatment. This first real-world analysis demonstrates clinical efficacy of vamorolone regarding stabilization or even mild improvement in adult DMD patients who were CS-naïve, had discontinued previous CS treatment, or switched from other CS. This data further highlights the importance of continued corticosteroid treatment beyond loss of ambulation."

Clinical • Cushing’s Disease • Duchenne Muscular Dystrophy • Endocrine Disorders • Genetic Disorders • Muscular Dystrophy • Obesity • Osteoporosis

Genetic Modifier Associations With Steroid Safety Outcomes in Duchenne Muscular Dystrophy (ICNMD 2026) - "Traditional corticosteroids like prednisone and deflazacort can result in weight gain, adrenal suppression, growth stunting, and reduced bone biomarkers, while vamorolone, a newly approved dissociative steroid, has shown an improved safety profile, especially on bone health. Al though based on a small sample size and needing additional studies for confirmation, we provide initial evidence of the association of genetic modifiers at an early age with common steroid safety concerns including for weight gain, which is a common reason for steroids prescribed at sub-recommended doses. These findings may be important for clinical trials and care."

Clinical • CNS Disorders • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy • CDX2

Meta-analysis of clinical trials assessing the safety of pharmacological treatments for muscular degeneration in Duchenne muscular dystrophy. (PubMed, BMC Pharmacol Toxicol) - "Precise ADR estimates can support decision-making in DMD care, helping clinicians balance safety and efficacy, address family concerns, and promote adherence by contextualizing treatment risks."

Journal • Retrospective data • Dermatology • Duchenne Muscular Dystrophy • Genetic Disorders • Hematological Disorders • Muscular Dystrophy

Unified Chemoenzymatic Approach to 16α-Methyl Glucocorticoids: Stereocontrolled Synthesis of (+)-Dexamethasone, (+)-Mometasone, (+)-Flumethasone, (+)-Halometasone, and (+)-Vamorolone. (PubMed, JACS Au) - "Herein, we report a unified stereocontrolled synthesis of five 16α-methyl glucocorticoid pharmaceuticals, namely, (+)-dexamethasone, (+)-mometasone, (+)-flumethasone, (+)-halometasone, and (+)-vamorolone, in 9.9-26.8% overall yields starting from commercially available 9α-hydroxyandrost-4-ene-3,17-dione (9α-OH-AD), featuring a telescoped in-flow synthesis consisting of an acidic resin-mediated dehydration and an enzyme-catalyzed C1,2-dehydrogenation, an Au-(I)-catalyzed hydration of enyne to enone, and a conjugate addition-dihydroxylation sequence-enabled construction of the chiral C16α-methyl and C17α,21-dihydroxyacetone moieties. This established chemoenzymatic platform provides a versatile and generic access to other valuable 16α-methyl glucocorticoids."

Journal

Santhera Announces Orphan Drug and Priority Review Designations for AGAMREE (Vamorolone) in South Korea (GlobeNewswire) - "Santhera Pharmaceuticals...notes that AGAMREE has been granted Orphan Drug Designation (ODD) and Global Innovative Products on Fast Track (GIFT) designation by South Korea’s Ministry of Food and Drug Safety (MFDS) for the treatment of Duchenne muscular dystrophy (DMD)...Nxera plans to submit a Marketing Authorization Application in South Korea during 2026...In the pivotal Phase 2b VISION-DMD study, AGAMREE met its primary endpoint, demonstrating a statistically significant improvement in Time to Stand (TTSTAND) velocity versus placebo at 24 weeks (p = 0.002)..."

Breakthrough therapy • Korea filing • Orphan drug • Duchenne Muscular Dystrophy

Regimen-dependent glucocorticoid effects improve muscle performance without altering CNS physiology in mdx mice. (PubMed, J Physiol) - "Daily glucocorticoid regimens triggered endocrine and metabolic side effects, whereas weekly dosing reduced these effects. Prednisolone and vamorolone produced broadly similar systemic side-effect profiles, but dosing frequency was the primary determinant of their severity."

Journal • Preclinical • Cardiovascular • Duchenne Muscular Dystrophy • Genetic Disorders • Immunology • Mood Disorders • Muscular Dystrophy • Psychiatry

Vamorolone for Duchenne Muscular Dystrophy: A Cross-Trial Efficacy Comparison With Classic Corticosteroids From the FOR-DMD Trial. (PubMed, Neurology) - P2b, P3 | "Vamorolone demonstrated numerically similar TTSTAND velocity changes to prednisone and deflazacort at 1 year; however, interpretations of differences are limited by 95% CIs crossing minimally important difference thresholds. Further evidence of the growth-protective effect of vamorolone was observed; however, all treatments increased BMI. Vamorolone provides a linear growth-protective classic corticosteroid alternative."

Clinical • Journal • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Optimizing Care for Growth and Puberty in Duchenne Muscular Dystrophy: A Survey of Clinical Practice in the OPTIMIZE DMD Consortium. (PubMed, Muscle Nerve) - "Compared with the 2018 Care Considerations, monitoring practices for growth and puberty remain largely consistent, while variability persists in management approaches. These findings provide important insights to inform future guidance and identify priorities for further education and research in endocrine management for individuals with DMD."

Journal • Duchenne Muscular Dystrophy • Endocrine Cancer • Endocrine Disorders • Genetic Disorders • Muscular Dystrophy

Vamorolone Safety, Pharmacokinetics, and Exploratory Efficacy in Duchenne Muscular Dystrophy: A Phase II, Nonrandomized, Multiple-Dose Study in 2-<4-Year-Old Boys. (PubMed, Neurology) - P, P2 | "Vamorolone was well tolerated in 2-<4-year-old boys with DMD, with no new safety concerns identified. A dose-dependent PK profile was observed, consistent with previous studies. Exploratory evidence suggested dose-dependent improvements in gross motor function. These findings are consistent with potential therapeutic benefit of vamorolone for young boys with DMD."

Journal • P2 data • PK/PD data • Duchenne Muscular Dystrophy • Gastroenterology • Gastrointestinal Disorder • Genetic Disorders • Infectious Disease • Muscular Dystrophy

A prospective, non-randomized, open-label, controlled cohort study comparing the safety of Vamorolone and prednisone in the treatment of children with Duchenne muscular dystrophy (ChiCTR) - P=N/A | N=124 | Sponsor: Children's Hospital of Fudan University; Children's Hospital of Fudan University

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

Santhera Receives Positive CHMP Opinion to Expand AGAMREE (vamorolone) Use in Pediatric DMD Patients Aged Two and Older in the EU (The Manila Times) - "In the pivotal Phase 2b VISION-DMD study, AGAMREE met its primary endpoint, demonstrating a statistically significant improvement in Time to Stand (TTSTAND) velocity versus placebo at 24 weeks (p = 0.002)."

CHMP • Duchenne Muscular Dystrophy

Treatment advances for Duchenne muscular dystrophy. (PubMed, Curr Opin Pediatr) - "This review summarizes the mechanism of action, key safety considerations and available evidence on motor function impact that these novel medications have demonstrated in DMD."

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