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The approval through the Medicines and Healthcare Regulatory Agency (MHRA) of AGAMREE® in the UK follows the marketing authorisation of this medicine in the EU and US. U. S.
The MHRA recognises the protective benefits of AGAMREE in terms of preserving bone health and maintaining the expansion of popular care corticosteroids.
AGAMREE’s launches in Europe, led by Germany, are expected to begin in the first quarter of 2024.
Pratteln, Switzerland, January 12, 2024 – Santhera Pharmaceuticals (SIX: SANN) announces that AGAMREE® (vamorolone) has been approved in the United Kingdom (UK) for the treatment of Duchenne muscular dystrophy (DMD) in patients 4 years of age and older, independent of the underlying mutation and ambulatory status. The UK’s MHRA, adopting the view of the European Medicines Agency (EMA), acknowledged clinically important safety benefits of AGAMREE with regards to maintaining normal bone metabolism, density and growth compared to standard of care corticosteroids, alongside similar efficacy [1].
“We are delighted to have secured a third approval for AGAMREE to treat Duchenne from a major regulatory agency, after the U.S. FDA and the EU EMA, within a couple months,” said Shabir Hasham, MD, Chief Medical Officer of Santhera. “In addition to its anti-inflammatory efficacy, both the EMA and the MHRA recognize the benefits of treatment with AGAMREE for bone health and growth, underlining the favorable safety and tolerability profile of this novel medicine compared to conventional corticosteroids. We are working towards making AGAMREE available to patients in the UK in the second half-year 2024, after NICE completes its pricing review. Initial European launch will be in Germany in Q1.”
“We are very pleased that the first medicine designed in particular for all other people with Duchenne has been approved in the UK,” said Emily Reuben OBE, CEO of Duchenne UK, and Alex Johnson OBE, CEO of Joining Jack, who are the co-founders of Duchenne, UK. When our children were diagnosed with Duchenne, we were told that steroids, the popular drug for children with this disease, would keep them cellularized autonomously for longer, but with negative side effects. We didn’t think about this enough and invested in finding better treatments. Duchenne UK and our partner charities, Joining Jack and the Duchenne Research Fund, funded early-stage clinical studies to test vamorolone in patients, when no one else would. that is now available in the UK to treat DMD is evidence that we can find better remedies for Duchenne and make a difference for Duchenne.
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 produce relief in bone metabolism as measured by bone biomarkers, nor significant relief in bone mineralization in the spine as measured by dual-energy X-ray absorptiometry (DXA) after 48 weeks in clinical studies. In addition, patients who switched from a corticosteroid to AGAMREE maintained efficacy and gained advantages in restoring their bone expansion and health.
Santhera will continue to gather knowledge to better characterize the long-term efficacy and broader protection differentiation of vamorolone.
This approval follows the approval of AGAMREE through the U. S. Food and Drug Administration (FDA) for the treatment of DMD in patients over 2 years of age in the United States and the approval through the European Commission for the treatment of DMD in the EU in patients. Four-year-old and older. This makes AGAMREE the first and only medicine fully approved in the EU and UK for DMD, and the first cure approved for the treatment of DMD in all three geographies.
About AGAMREE® (vamorolone) Vamorolone is a novel drug whose mode of action is based on binding to the same receptor as glucocorticoids, but alters its subsequent activity and is not a substrate for the enzymes 11-β-hydroxysteroid dehydrogenase (11β- HSD) which is possibly culprit for local tissue amplification and corticosteroid-associated toxicity in local tissues [2-4]. This mechanism has demonstrated the potential to “decouple” efficacy from protective considerations of steroids and, as a result, vamorolone is being positioned as an option to existing corticosteroids. , the popular existing care in young people and adolescents 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.
Current knowledge shows that vamorolone, unlike corticosteroids, has no expansion restrictions [5] or negative effects on bone metabolism, as evidenced by general serum markers of bone formation and resorption [6].
AGAMREE (vamorolone) has an orphan drug for DMD in the U. S. U. S. and European Fast-Track designations and Pediatric Rare Diseases through the U. S. FDA have been approved by the U. S. Food and Drug Administration. The U. S. Department of Homeland Security has been granted the U. S. Advanced Promising Medicine (PIM) designation through the UK MHRA for DMD. AGAMREE is approved for use in the United States. The United States, the European Union, and the United Kingdom.
References:[1] Summary of Product Characteristics (SmPC) / European Public Assessment Report (EPAR)[2] Guglieri M et al (2022). JAMA Neurol. 2022; 79(10):1005-1014. com what I:10. 1001/jamaneurol. 2022. 2480. Link. [3] Liu X et al. (2020). Proc Natl Acad Sci USA 117:24285-24293[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 SantheraSanthera Pharmaceuticals (SIX: SANN) is a Swiss pharmaceutical company focused on the development and commercialization of cutting-edge medicines for the treatment of rare neuromuscular and lung diseases with significant unmet medical needs. The company holds an exclusive license from ReveraGen for all indications internationally. for AGAMREE® (vamorolone), a dissociative steroid with a novel mode of action, which has been studied in a pivotal study in patients with Duchenne muscular dystrophy (DMD) as an alternative to popular corticosteroids. AGAMREE is approved for the treatment of DMD in the United States through the Food and Drug Administration (FDA), in the EU through the European Medicines Agency (EMA), and in the United Kingdom through the United Kingdom Medicines and Healthcare products Regulatory Agency (MHRA). Santhera has licensed the North American rights to vamorolone to Catalyst Pharmaceuticals and Sperogenix Therapeutics in China. The clinical-stage product portfolio also includes lonodelestat to treat cystic fibrosis (CF) and other neutrophil lung diseases. For more information, visit www. santhera. com.
AGAMREE® is a trademark of Santhera Pharmaceuticals.
For further information please contact: [email protected] orEva Kalias, Head Investor Relations & CommunicationsPhone: +41 79 875 27 [email protected]
This communication does not constitute an offer or invitation to subscribe for or acquire securities of Santhera Pharmaceuticals Holding AG. This publication may include certain forward-looking statements about the Company and its business. Such statements involve certain risks, uncertainties and other points. that may also cause the Company’s actual results, monetary condition, functionality or achievements to differ materially from those expressed or implied by such statements. Therefore, readers do not place undue reliance on such statements, i. e. , in connection with a contract or investment decision. The Company disclaims any legal responsibility to update such forward-looking statements.
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