Ad hoc announcement pursuant to Art. 53 LR
- Committee for Medicinal Products for Human Use (CHMP) issues positive opinion for AGAMREE® (vamorolone) for the treatment Duchenne muscular dystrophy (DMD) in children and adults aged 4 years and older
European Commission (EC) decision on marketing authorization is expected in late 2023- AGAMREE could become the first drug fully approved by the
European Medicines Agency (EMA) for the treatment of patients with DMD - Anticipation of vamorolone approval in the
U.S. on the PDUFA date ofOctober 26, 2023
Pratteln,
“We are thrilled about the CHMP’s positive opinion, which recognizes the urgent medical need for an effective and well tolerated treatment for this devastating disease. We can now execute on our plans to ensure AGAMREE is made available to patients in the EU as soon as the
“We are very pleased about the positive vote of the
“We are very excited about the positive opinion from the
The clinical evidence for the efficacy and safety of AGAMREE (vamorolone) in the regulatory submission was derived from the positive pivotal VISION-DMD study and three open-label studies (including extensions), in which vamorolone was administered at doses between 2 and 6 mg/kg/day for a total treatment period of up to 30 months, as well as an external comparator study (FOR-DMD [6]) and several clinical pharmacology studies. The studies were carried out by Santhera’s partner ReveraGen and 32 academic clinical trial centers in 11 countries.
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 safety differentiation of vamorolone.
A marketing authorization decision from the EC is expected within approximately two months of the positive CHMP opinion. Subject to approval, AGAMREE will be the first and only medicinal product fully approved by the EMA for the treatment of DMD and the marketing authorization will be valid in all 27 member states of the
In the
About AGAMREE® (vamorolone)
Vamorolone is a novel drug candidate 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, and as such is considered a dissociative anti-inflammatory drug [1-3]. 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 [1-3].
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 [1]. 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 [4] and no negative effects on bone metabolism as demonstrated by normal bone formation and bone resorption serum markers [5].
Vamorolone has Orphan Drug status for DMD in the
References:
[1] Guglieri M et al (2022). JAMA Neurol. 2022;79(10):1005-1014. doi:10.1001/jamaneurol.2022.2480. Link.
[2] Liu X et al (2020).
[3] Heier CR et al (2019). Life Science Alliance DOI: 10.26508
[4] Ward et al., WMS 2022, FP.27 - Poster 71. Link.
[5] Hasham et al., MDA 2022 Poster presentation. Link.
[6] FOR-DMD. 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
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Attachment
- 2023 10 13_CHMP opinion_e_FINAL
Source:
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