Promising long-term findings for AGAMREE in Duchenne muscular dystrophy are reported by Santhera.
Early vamorolone treatment demonstrates increased safety and long-lasting efficacy in muscular dystrophy by AGAMREE
Positive topline results from a long-term analysis of AGAMREE (vamorolone) in individuals with Duchenne muscular dystrophy (DMD), including initial data from the ongoing phase 4 GUARDIAN research, have been released by Santhera Pharmaceuticals.
Up to 110 corticosteroid-naïve patients who started treatment between the ages of four and seven and continued through different access programs were included in the analysis. AGAMREE was administered to patients for a maximum of eight years, with a median follow-up of five years. In real-world situations, the majority continued to take greater doses (4–6 mg/kg/day).
Over time, patients’ motor function was preserved, and their effectiveness was on par with that of conventional corticosteroids. Subgroup analyses compared to prednisone or deflazacort showed no differences. Crucially, AGAMREE demonstrated a distinct safety profile with a markedly lower incidence of cataracts, normal development patterns, and spinal fractures. There were no documented glaucoma instances or new warning signs.
Expert Insights
Prof Eugenio Mercuri said:
“These data provide important evidence that long-term treatment with vamorolone provides durable efficacy, with a substantial reduction in the risk of spine fractures and of improvement in height, in contrast to what is observed with conventional steroids.”
Prof Francesco Muntoni added:
“These preliminary data are encouraging. In particular, it is reassuring to see that children continue to grow in height without an apparent impact on treatment efficacy.”
Prof Craig McDonald said:
“These data now clearly show the benefits of treating early and maintaining treatment at effective doses with vamorolone.”
Shabir Hasham, CMO of Santhera, commented:
“We are truly delighted to offer the DMD community a corticosteroid treatment option that can provide long-term benefit and significantly reduce the occurrence of some of the most debilitating side effects.”
Why AGAMREE Matters in Duchenne
AGAMREE represents a next-generation steroid alternative designed to retain anti-inflammatory benefits while minimizing steroid-related growth and bone complications — a major barrier in DMD treatment.
Key Clinical Outcomes
- Sustained motor function over the years
- Safer bone and growth profile
- Lower cataract & fracture rates
- No new safety risks observed
Patient-Centric Advantage
Growth preservation is a major milestone for families and clinicians, addressing one of the biggest steroid-related concerns in DMD children.
Future Trends & Industry Outlook
Future of DMD Therapy
With rising interest in gene therapies, exon skipping, and combination therapies, AGAMREE may serve as a long-term foundational therapy for early diagnosed DMD patients.
Potential Expansion
Santhera may explore:
- Global accessibility and reimbursement programs
- Combination studies with gene and cell therapies
- New data supporting early-stage diagnosis and treatment protocols
What’s Next?
The continuation of the Phase 4 GUARDIAN study will:
- Validate real-world long-term outcomes
- Strengthen regulatory and clinician confidence
- Potentially improve the standard-of-care guidelines
