European Commission (EC) grants marketing authorization for AGAMREE® for the treatment of Duchenne muscular dystrophy (DMD) in patients aged 4 years and older- AGAMREE® is the only approved medication in the
European Union (EU) for treating DMD, and the first DMD treatment approved in both theU.S. and EU - First commercial launch of AGAMREE®, in
Germany , expected in Q1-2024 - EMA acknowledges safety benefits of AGAMREE® with regards to preserving bone health and maintaining growth compared to standard of care corticosteroids
Pratteln,
This makes AGAMREE® the first and only medicinal product to have received full approval in the EU and, following
“We are thrilled to receive approval from the EC to bring AGAMREE to patients in the EU, which highlights the favorable safety and tolerability profile over conventional corticosteroids, including benefits for bone health and growth,” said
“World Duchenne Organization is happy to see the development of AGAMREE, during which the involvement of many Duchenne patient organizations has been instrumental by providing early funding and contributing to clinical trial design, outcomes and recruitment, led to the first full market authorization in the
The approval by the EC was based on data from the positive pivotal VISION-DMD study and three open-label studies in which vamorolone was administered at doses between 2 and 6 mg/kg/day for a total treatment period of up to 30 months. In the pivotal VISION-DMD study, boys treated with vamorolone on average maintained growth similar to those treated with placebo, whilst those treated with prednisone on average experienced growth stunting. Patients who switched from prednisone to vamorolone after 24-weeks were, on average, able to resume growing in height over the remainder of the study.
Unlike corticosteroids, vamorolone did not result in a reduction of bone metabolism as measured by bone biomarkers, nor in a significant reduction of bone mineralization in the spine as measured by Dual Energy X-Ray Absorptiometry (DXA) after 48 weeks in the clinical studies.
Santhera will continue to collect data to further characterize the long-term effectiveness and the broader safety differentiation of vamorolone.
The EC’s decision follows the positive opinion for AGAMREE from the Committee for Medicinal Products for Human Use (CHMP) which was announced on
Santhera announced on
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] European public assessment report (EPAR), summary of product characteristics (SmPC)
[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
- 2023 12 18_EC-approval_e_finalx
Source:
2023 GlobeNewswire, Inc., source