Breaking News – First Treatment for ALL patients gets approved in the UK

January 16, 2024 8:34 am

We are delighted to welcome the news that the Medicines and Healthcare products Regulatory Agency (MHRA) has approved vamorolone (sold under the brand name Agamree) for the treatment of Duchenne muscular dystrophy (DMD) in patients aged 4 years and older.

This is the first treatment approved for all people with DMD, irrespective of their underlying mutation and ambulatory (walking) status.

This is a key milestone towards access to vamorolone for patients and families in the UK. However, it is not yet available on the NHS.

The National Institute for Health and Care Excellence (NICE), the regulator responsible for assessing the clinical and cost effectiveness of drugs in England, is currently undertaking a health technology assessment (HTA) for Vamorolone. This will decide if it is clinically and cost effective for NHS patients. A decision is expected in mid 2024.

Our very own Alex, has been on steriods since the age of 4, and he’s had his fair share of issues taking steriods. From bone health issues (he broke his hip when he was just 9 years old), to unwanted weight gain, mood swings and delayed puberty. Alex is very excited about this amazing news, as this will have real impact on his quality of life.

We would like to thank our supporters for their contribution towards making this a reality.

About Vamorolone

Vamorolone was developed as an alternative to corticosteroids, also called glucocorticoids, which are routinely used in the treatment of DMD as they can reduce muscle inflammation and maintain muscle strength.

Steroid treatment has several negative side effects, and Vamorolone was developed to keep or improve the positives (efficacy profile) in comparison to steroid, but with the aim of showing fewer side-effects.

Alex’s Wish support with this project

We gave £47,000 towards this project back in 2015, working with Dr Eric Hoffman (ReverGen).

This work included a 48-week Phase 2b VISION-DMD study is designed as a pivotal trial to demonstrate efficacy and safety of Vamorolone administered orally at doses of 2.0 mg/kg/day and 6.0 mg/kg/day versus prednisone 0.75 mg/kg/day and placebo in ambulant boys.

Dr Hoffman said at the time “We are delighted about having achieved this important milestone and are looking forward to announcing the topline 6-month results of this pivotal study together with Santhera,” said Eric Hoffman, PhD, President and CEO at ReveraGen BioPharma. “The use of glucocorticoids, despite having proven benefits in the treatment of DMD, is severely limited due to side effects and poor tolerability. Our expectation is that vamorolone will have the benefits but avoids many of the tolerability issues that limit the use of this standard of care. Our thanks go out to the study participants, their families and healthcare professionals who, in the midst of the COVID-19 pandemic, are enabling us to advance this pivotal study as intended.”

“Based on previously established data, we believe that vamorolone has the potential to become a foundational therapy in DMD for patients irrespective of the underlying gene mutation and a promising alternative to existing corticosteroids,” noted Dario Eklund, CEO of Santhera. “Our organization is whole heartedly dedicated to bringing this novel therapy to patients who are hoping for a DMD therapy with fewer treatment limiting side effects, making it suitable for longer term administration and also improving quality of life.”

In the currently completed studies, a total of 48 patients have received various doses of vamorolone; of which 41 patients have been treated and evaluated for a period of 2.5 years. Aggregate clinical data from these open label studies in DMD published to date showed sustained efficacy and clinical improvement with vamorolone across multiple endpoints [2]. Additionally, vamorolone did not show stunting of growth seen with deflazacort and prednisone, and also showed fewer physician-reported adverse events such as mood disturbance, excessive hair growth, and Cushingoid appearance [2].

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