Vamorolone (Agamree)

Key Messages

• Canada’s Drug Agency (CDA-AMC) recommends that Agamree be reimbursed by public drug plans for the treatment of Duchenne muscular dystrophy (DMD) in patients aged 4 years or older, if certain conditions are met.
• The Canadian Drug Expert Committee (CDEC) determined that, in patients with DMD, Agamree demonstrates acceptable clinical value versus prednisone; however, the clinical value compared to deflazacort is uncertain. This determination was sufficient for CDEC to recommend that Agamree be reimbursed. Given that Agamree is expected to be an alternative to prednisone or deflazacort, acceptable clinical value refers to at least comparable value versus corticosteroids.
• Evidence from a clinical trial (the VBP15-004 study [N = 121]) in children with DMD aged 4 to 7 years showed that Agamree given for 24 weeks improved physical function outcomes (North Star Ambulatory Assessment [NSAA] total score, time-to-stand [TTSTAND] velocity, and 6-minute walk test [6MWT] distance) versus placebo, and that it likely has a similar effect on physical function compared to prednisone. Evidence from 1 external comparison of Agamree, prednisone, and deflazacort suggested that the 3 treatments likely provide comparable clinical benefits in pediatric patients with DMD after 48 weeks of treatment. However, there was little to no difference between vamorolone and prednisone in side effects commonly linked to steroids, including behaviour problems, weight gain, and broken bones.
• Patients and clinicians identified a need for accessible treatments that preserve physical function, prevent cardiac and respiratory decline, improve survival and health-related quality of life (HRQoL), and reduce caregiver burden without the adverse effects associated with long-term corticosteroid use. Compared with placebo, Agamree may help preserve short-term physical function. However, it is uncertain whether Agamree addresses these needs better than currently available treatments. CDEC also recognized that there remains a significant unmet need in DMD because the disease is severe, existing treatments have long-term safety considerations and access limitations, and generating high-quality evidence is challenging.
• Based on all of the preceding considerations, CDEC recommended that Agamree be reimbursed.