SANTHERA PHARMACEUTICALS HOLDING AG
Ad hoc announcement pursuant to Art. 53 LR
Food and Drug Administration (FDA) approved AGAMREE® (vamorolone) for the treatment of Duchenne muscular dystrophy (DMD) in children and adults aged 2 years and olderU.S. license holder Catalyst Pharmaceuticals plans for commercial launch in Q1-2024- FDA approval triggers USD 36 million payment obligation from Catalyst to Santhera, of which USD 26 million will be used to cover Santhera’s third-party milestone obligations
- Following the recent positive CHMP opinion on AGAMREE in DMD, approval in the
European Union by theEuropean Commission (EC) is expected in late 2023 - AGAMREE would then become the first drug fully approved in both the
U.S. andEurope for the treatment of patients with DMD
Pratteln,
“We are delighted to secure FDA approval which comes just weeks after the positive opinion from the CHMP of the
“This new drug approval gives Duchenne patients and their families one more reason for hope,” said
“I am excited for the Duchenne community who have been waiting a long time for an alternative to the current standard of care. Today’s news represents the culmination of many years of research to bring vamorolone to patients,” said
The FDA approval of AGAMREE® was based on the data from the pivotal Phase 2b VISION-DMD study as supplemented with safety information collected from three open-label studies, including extension studies. In these trials, AGAMREE was administered at doses ranging from 2 to 6 mg/kg/day, extending for a period of up to 48 months. Compared with current standard of care corticosteroids, this novel corticosteroid treatment exhibited comparable efficacy, with data suggesting a reduction in adverse events, notably related to bone health, growth trajectory and behavior. The studies in the development program were carried out by Santhera’s partner ReveraGen and 32 academic clinical trial centers in 11 countries.
With the FDA approval of AGAMREE for DMD, Catalyst will pay USD 36 million to Santhera, consisting of a USD 10 million approval milestone to the Company and an additional USD 26 million to cover contracted third-party milestone obligations. Furthermore, under the terms of the agreement, Catalyst will pay Santhera sales-based milestones of up to USD 105 million as well as up to low-teen percentage royalties, and will assume Santhera's corresponding third-party royalty obligations on vamorolone sales in all indications in
Santhera will now transfer the
In
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 [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].
AGAMREE (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.
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 27_FDA approval_e_final
2023 GlobeNewswire, Inc., source
