AGAMREE Sustains DMD Function With Fewer Side Effects
Santhera Pharmaceuticals has reported positive topline results from a long-term analysis of AGAMREE (vamorolone) in boys with Duchenne muscular dystrophy (DMD), including the first read-out from the ongoing phase 4 GUARDIAN study.
The dataset covers up to 110 corticosteroid-naïve patients who started treatment between ages four and seven and then continued therapy through various access programmes.
Patients received AGAMREE for as long as eight years, with a median follow-up of five years, offering one of the most extended real-world looks at the medicine to date.
Durable motor outcomes at real-world doses
In practice, most patients remained on higher dosing (4–6 mg/kg/day). Across this prolonged window, children maintained motor function, with efficacy comparable to standard corticosteroids.
Subgroup analyses showed no differences versus deflazacort or prednisone, underscoring that vamorolone can deliver the expected functional benefits while aiming to mitigate well-known steroid burdens.
Differentiated safety: fewer fractures, healthier growth
The safety profile stood out. Investigators observed significantly fewer vertebral (spine) fractures, normal growth patterns and a reduced incidence of cataracts in boys receiving AGAMREE.
Notably, there were no cases of glaucoma and no new safety signals emerged over the extended follow-up.
According to a Professor of Paediatrics and Child Neuropsychiatry at Università Cattolica del Sacro Cuore, the data provides important evidence that long-term vamorolone offers durable efficacy together with a substantially lower risk of spine fractures and improved height trajectories compared with conventional steroids.
Expert reaction: efficacy without stunting growth
A Professor of Neurology at University College London described the preliminary findings as encouraging – particularly the reassurance that children continue to grow in height without an apparent trade-off in treatment efficacy.
What’s more, a Professor of Physical Medicine & Rehabilitation and Paediatrics at UC Davis added that the results now clearly support starting early and maintaining effective doses over time to secure clinical benefit.
Company outlook
Santhera’s Chief Medical Officer said the company is delighted to provide the DMD community with a corticosteroid option that can deliver long-term benefit while significantly reducing some of the most debilitating side effects associated with standard steroid therapy.
With GUARDIAN continuing and longer follow-up accruing, the company believes these data strengthen the case for AGAMREE as a foundational treatment option in young, steroid-naïve patients.
Conclusion
Taken together, the long-term analysis – including the first GUARDIAN findings – suggests that AGAMREE (vamorolone) can match the functional efficacy of conventional corticosteroids in DMD while offering a more favourable safety profile: fewer vertebral fractures, healthier growth and lower cataract burden, with no glaucoma observed and no new safety signals.
For families and clinicians navigating the delicate balance between preserving motor function and limiting steroid toxicity, these results point to a treatment strategy that supports both sustained efficacy and better long-term tolerability.
News Credits: Santhera reports promising long-term data for AGAMREE in Duchenne muscular dystrophy
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