Introduction: AGAMREE® (vamorolone), approved by the US FDA in October 2023 for Duchenne muscular dystrophy (DMD) in patients ≥2 years and granted Orphan Drug and Rare Pediatric Disease status, is an important advancement in treating this severe, X-linked recessive disorder caused by mutations in the dystrophin gene and loss of dystrophin protein expression. No cure for DMD exists. While therapies such as exon-skipping and gene therapy have been approved in subsets of patients, corticosteroids remain the fundamental treatment for DMD. AGAMREE® is an alternative to other steroid-based treatments appropriate for all boys with DMD. It can be prescribed irrespective of mutational status and/or in conjunction with other treatments. AGAMREE® is a novel corticosteroid with a unique structure that binds to the same target receptors (glucocorticoid and mineralocorticoid receptors) but shows differences in downstream effects. Specifically, AGAMREE® lacks an 11β-hydroxyl/carbonyl moiety, resulting in it being a potent antagonist of the mineralocorticoid receptor whereas most corticosteroids are agonists. This unique structure is expected to result in improved safety. The short-term safety and efficacy of vamorolone was demonstrated in randomized, double-blind, placebo- and active-controlled studies, and its longer-term safety in a pivotal open-label study (NCT0308399). Expected to start in March 2024, this registry will collect additional long-term safety and quality-of-life (QoL) data in male patients with DMD treated with AGAMREE®.
Methods: The multicenter, observational, prospective, longitudinal registry includes approximately 25 US sites and collects safety and QoL data in 250 male patients ≥2 years of age with a confirmed diagnosis of DMD and receiving AGAMREE®. Enrollees will be monitored up to 5 years and more closely than those receiving standard of care (SoC). As such, any safety concerns will be identified and addressed sooner than while on SoC. Following enrollment, participants will be assessed at week 12 and then every 12 months.
Results: Up to 4 interim analyses with the first estimated in May 2025.
Conclusions: Results from clinical trials need to be complemented with real-world data and this planned registry will provide important information regarding long-term use of AGAMREE®. We wish to raise awareness among physicians treating patients with DMD and encourage them and caregivers alike to consider enrolling eligible patients in this registry.