Thank You – An announcement on the Early Access to Medicines Scheme for Raxone

We are delighted to announce that the MHRA have decided to maintain a positive opinion on the Early Access to Medicines Scheme for Raxone to treat people living with Duchenne muscular dystrophy. This means that Raxone remains on course to have its EAMS award renewed, so that patients over 10 years of age, showing a decline in respiratory function and who are currently not taking glucocorticoids, can continue to access Raxone until June 2019

This was a joint effort from the entire Duchenne community and Action Duchenne, DMD Pathfinders, Duchenne Family Support Group, Duchenne UK and Muscular Dystrophy UK cannot thank enough, all of you who provided vital feedback to this process.

Each and every organisation appreciates how much effort and courage goes into providing this testimony, whether it is delivered in writing or in person, and we have no doubt this had a positive impact on the committee’s decision.

The UK Commission on Human Medicines (CHM) determined from the evidence and testimony received, that the risk/benefit profile was acceptable and there is a clear high unmet medical need for patients with Duchenne. Receiving feedback from such a wide spectrum of the community was integral to this.

“Action Duchenne is delighted with the positive news that the EAMS scheme for Raxone is being renewed based on scientific opinion of the risk/benefit ratio. The MHRA involved Action Duchenne and all the UK patient organisations in this process. As a collective powerful UK community, we supported young people, adults living with Duchenne and caregivers both in collecting vital lived experience testimonies on respiratory health and in person. Raxone is available for those who are eligible and after appropriate medical consultation, but please do feel free to reach out to myself and the team with any other questions or queries. As outlined in our recently updated research strategy, Action Duchenne is committed to supporting treatment advances for the entire spectrum of the Duchenne community.” – Action Duchenne.

The Early Access to Medicines Scheme (EAMS) for Raxone

• The aim of the EAMS is to provide earlier availability of promising medicines to patients in the UK where there is high unmet clinical need.

• It is to slow the decline of respiratory function in patients with Duchenne from the age of 10 years who are currently not taking glucocorticoids.

• At the same time more data is able to be captured, to evaluate the effectiveness of the medicine.

• Treatment will only be provided in specialist centres that are experienced in managing Duchenne and there is the requirement for careful monitoring to ensure the safety of patients during the EAMS.

• People with Duchenne may discuss together with their doctor whether they are suitable for this treatment.

This Frequently Asked Questions document provides more detail, but if you have any questions please do not hesitate to contact Director of Research, Neil Bennett neil@actionduchenne.org