Santhera receives UK approval for Duchenne muscular dystrophy treatment

• Approval by the Medicines and Healthcare products Regulatory Agency (MHRA) of AGAMREE in the United Kingdom follows marketing authorization for this medicine in the EU and U.S.
• MHRA acknowledges safety benefits of AGAMREE with regards to preserving bone health and maintaining growth compared to standard of care corticosteroids
• Launches of AGAMREE in Europe, with Germany taking the lead, are planned to start in Q1-2024

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 delighted that the first drug designed specifically for everyone with Duchenne has been approved in the UK,” said Emily Reuben OBE, Chief Executive of Duchenne UK, and Alex Johnson OBE, Chief Executive of Joining Jack, who are the co-founders of Duchenne UK. “When our sons were diagnosed with Duchenne, we were told that steroids, the standard medication for children with it, would keep them independently mobile for longer. But with harmful side effects. We didn’t think this was good enough, and invested in finding better treatments. Duchenne UK and our partner charities, Joining Jack and the Duchenne Research Fund, funded the early-stage clinical research to test vamorolone in patients, when no-one else would. For it now to be available in the UK to treat DMD is proof that we can find better treatments for Duchenne and change things 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 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. In addition, patients who switched from a standard of care corticosteroid to AGAMREE maintained the efficacy benefit while recovering their growth and bone health.

Santhera will continue to collect data to further characterize the long-term effectiveness and the broader safety differentiation of vamorolone.

This approval follows the approval of AGAMREE by the U.S. Food and Drug Administration (FDA) for the treatment of DMD in patients aged 2 years and older in the U.S. and approval by the European Commission for the treatment of DMD in the EU in patients aged 4 years and older. This makes AGAMREE the first and only medicinal product fully approved in the EU and UK for DMD, and the first treatment approved for the treatment of DMD all three geographies.

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. doi: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.