Santhera Announces Publication of Efficacy, Safety and Tolerability Data with Vamorolone (AGAMREE) in Patients with Duchenne Muscular Dystrophy in Neurology - Seite 2

Improvements of motor outcomes seen with 6 mg/kg/d of vamorolone at 24 weeks of treatment were maintained to 48 weeks of treatment. Vamorolone at a dose of 6 mg/kg/d showed better maintenance of effect compared with vamorolone at a dose of 2 mg/kg/d for most (3/5) motor outcomes. Bone morbidities of prednisone (stunting of growth and declines in serum bone biomarkers) were reversed when treatment transitioned to vamorolone.”

AGAMREE is the first and only medicinal product for DMD to have received full approval in the EU and, following approval in the U.S. last October and in the UK in January, it is the first authorized treatment for patients with the disease in all three territories.

About AGAMREE (vamorolone)
Vamorolone is a novel drug with a mode of action based on binding to the same receptor as glucocorticoids but modifying its downstream activity and is not a substrate for the 11-β-hydroxysteroid dehydrogenase (11β-HSD) enzymes that may be responsible for local tissue amplification and corticosteroid-associated toxicity in local tissues [3-5]. This mechanism has shown the potential to ‘dissociate’ efficacy from steroid safety concerns and therefore vamorolone is positioned as an alternative to existing corticosteroids, the current standard of care in children and adolescent patients with DMD [3-5].

In the pivotal VISION-DMD study, vamorolone met the primary endpoint Time to Stand (TTSTAND) velocity versus placebo (p=0.002) at 24 weeks of treatment and showed a good safety and tolerability profile [4]. The most commonly reported side effects were cushingoid features, vomiting, weight increase and irritability. Side effects were generally of mild to moderate severity.

Currently available data show that vamorolone, unlike corticosteroids, has no restriction of growth [6] and no negative effects on bone metabolism as demonstrated by normal bone formation and bone resorption serum markers [7].

AGAMREE (vamorolone), an orphan medicinal product, is approved for use in the United States (Prescribing Information), the European Union (Summary of Product Characteristics) and the United Kingdom.

References:
[1]        Applicable drug labeling: Summary of Product Characteristics (SmPC). English. German.
[2]        Dang UJ et al. (2024) Neurology 2024;102:e208112. doi.org/10.1212/WNL.0000000000208112. Link.
[3]         Guglieri M et al (2022). JAMA Neurol. 2022;79(10):1005-1014. doi:10.1001/jamaneurol.2022.2480. Link.