We are delighted to announce that the Medicines & Healthcare Products Regulatory Agency (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 Early Access to Medicines Scheme (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
You made it happen
This was a joint effort from the entire Duchenne community and Muscular Dystrophy UK, Action Duchenne, DMD Pathfinders, Duchenne Family Support Group and Duchenne UK cannot thank all of you who provided vital feedback to this process enough.
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. We were told that receiving feedback from such a wide spectrum of the community was integral to this decision.
Nic Bungay, Director of Campaigns, Care and Information at Muscular Dystrophy UK commented:
We welcome the decision of the MHRA to maintain its positive opinion of the Raxone EAMS. The testimonies provided by people living with Duchenne highlights the hope that Raxone represents for people. We are so grateful to everyone who fed into the process and shared their story.
Duchenne still has few treatment options and no cure. We must continue to look at innovative ways of bringing promising Duchenne treatments at the earliest possible stage to those who could benefit from them.
Access to Raxone
Raxone is used to slow the decline of respiratory function in patients with Duchenne from the age of 10 years who are currently not taking glucocorticoids. It is currently accessed via the EAMS which provides earlier availability of promising medicines to patients in the UK where there is high unmet clinical need. 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 a requirement for careful monitoring to ensure the safety of patients during the EAMS. People with Duchenne should speak with their doctor to find out whether the treatment is suitable for them.
You can find out more by reading this Frequently Asked Questions document produced by Santhera, the pharmaceutical company providing Raxone.