You have an important voice – make it heard in the discussion about Raxone and the EAMS

What is the EAMS?
The Early Access to Medicines Scheme (EAMS) aims to give patients with life threatening or seriously debilitating conditions, access to medicines that do not yet have a marketing authorisation where treatment options are severely limited or absent (as is the case with Duchenne).

What is Raxone?

Raxone is a medicine that is believed to improve the functioning of mitochondria (important for the generation of cellular energy) inside cells. Under EAMS it is being made available as an unlicensed medicine to slow the decline in respiratory (lung) function in patients with Duchenne and, with this, potential slow the emergence of serious breathing difficulties in later stages of the disease such as infection (pneumonia) and low oxygen levels (hypoxia).

It is only given to patients not taking glucocorticoids (steroids) who in addition have clear evidence of deteriorating lung function in tests that are routinely conducted by specialists involved in the care of Duchenne patients.

Who can provide feedback?
1. People living with Duchenne who have received Raxone via the EAMS, or their carer (parent/legal guardian etc.)
2. Adults living with Duchenne
3. Parents/carers/legal guardians of a person living with Duchenne who is over 10 years of age

Why feedback is important?
A positive EAMS scientific opinion was awarded by the MHRA in June 2017 for Raxone in the treatment of respiratory decline in Duchenne, however the EMA recommended against the approval in September 2017 and again in January 2018, following a re-examination of data.

The MHRA and Commission of Human Medicines are currently reconsidering whether to make Raxone available through the EAMS and are inviting patients and carers to contribute to this discussion.

The DEADLINE to submit testimony is midnight 20 March 2018, so we don’t have long!

If you are interested in participating please contact Iain Clarke on 020 8556 9955 or iain@actionduchenne.org.

We are working with DMD Pathfinders, Duchenne Family Support Group, Duchenne UK and Muscular Dystrophy UK to collate this evidence. If you have already provided testimony to one of those organisations, you do not need to provide it to us as well.

We will treat your testimony with the utmost privacy and it will only be shared with those organisations and the MHRA and only for the purpose set out above.