- AGAMREE® is the only approved medication in the
European Union (EU) for treating all patients from age 4 years with DMD, and the first DMD treatment approved across theU.S. , EU andUK - This marks the first commercial launch of AGAMREE® globally
Pratteln,
AGAMREE® is the first and only medicinal product for DMD to have received full approval in the EU and, following approval in the
“We are very happy that AGAMREE is now commercially available in
Silvia Hornkamp, Managing Director of the German Duchenne Foundation, commented: “We are delighted that Santhera has been investing in Duchenne research for so many years. Now the time has come—with the new drug AGAMREE, patients suffering from Duchenne muscular dystrophy have access to a better tolerated alternative to steroids. It is a benefit for the sick children, who until now have been burdened with many side effects due to the long-term use of steroids.”
This launch follows the European Commission’s approval of AGAMREE® on
For more information about AGAMREE in the
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 [2-4]. 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 [2-4].
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 [3]. The most commonly reported adverse events versus placebo from the VISION-DMD study were cushingoid features, vomiting and vitamin D deficiency. Adverse events were generally of mild to moderate severity.
Currently available data show that vamorolone, unlike corticosteroids, has no restriction of growth [5] and no negative effects on bone metabolism as demonstrated by normal bone formation and bone resorption serum markers [6].
AGAMREE® (vamorolone) has Orphan Drug status for DMD in the
References:
[1] Summary of Product Characteristics (SmPC). English. German.
[2] Guglieri M et al (2022). JAMA Neurol. 2022;79(10):1005-1014. doi:10.1001/jamaneurol.2022.2480. Link.
[3] Liu X et al (2020).
[4] Heier CR et al (2019). Life Science Alliance DOI: 10.26508
[5] Ward et al., WMS 2022, FP.27 - Poster 71. Link.
[6] Hasham et al., MDA 2022 Poster presentation. Link.
About Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a rare inherited X-chromosome-linked disease, which almost exclusively affects males. DMD is characterized by inflammation which is present at birth or shortly thereafter. Inflammation leads to fibrosis of muscle and is clinically manifested by progressive muscle degeneration and weakness. Major milestones in the disease are the loss of ambulation, the loss of self-feeding, the start of assisted ventilation, and the development of cardiomyopathy. DMD reduces life expectancy to before the fourth decade due to respiratory and/or cardiac failure. Corticosteroids are the current standard of care for the treatment of DMD.
About Santhera
AGAMREE® is a trademark of
For further information please contact:
public-relations@santhera.com or
Phone: +41 79 875 27 80
eva.kalias@santhera.com
Disclaimer / Forward-looking statements
This communication does not constitute an offer or invitation to subscribe for or purchase any securities of
# # #
Attachment
- 2024 01 15_AgamreeLaunch_e_final
Source:
2024 GlobeNewswire, Inc., source