Over a number of years we have worked with Santhera Pharmaceuticals and other Duchenne charities in relation to Santhera’s Phase III Clinical Trial study. We recognised that it was important through the trial to investigate the potential for Raxone (idebenone) to help delay the onset of severe breathing difficulties for those with Duchenne. The evidence from the Phase III trial found that in a 12 month period there were indications that it slowed the rate of respiratory function loss for those living with Duchenne who took part in this trial. This data has been made publicly available as you can see here. This evidence along with other evidence was examined by the Medicines and Healthcare Products Regulatory Agency (MHRA) as part of its decision-making process that led to the MHRA giving Raxone a positive scientific opinion through the Early Access to Medicines Scheme (EAMS).
Further details about the announcement can be found via a press release here from the manufacturer of the drugs Santhera Pharmaceuticals, including a statement from Action Duchenne’s Chair of the Board of Trustees, Janet Bloor who highlighted how Action Duchenne provided key advice in the development of the EAMS programme, which we believe as shown in the approval of Raxone can help provide faster access to new, innovative and potentially life-transforming drugs for those living with Duchenne.
In an accompanying article on our website which we link to here, we explain in further detail what the EAMS Programme is.
Action Duchenne’s Director of Research added;
“We are delighted to hear that Raxone (idebenone) has become the first ever Duchenne muscular dystrophy treatment to be given a positive scientific opinion by the EAMs. This means earlier access for the young people living with Duchenne who are eligible and a further potential treatment advance to help slow the progression of Duchenne.”
We realise that those with Duchenne and their families will want to know what does this all mean for them and with that in mind we have outlined below what Raxone is, who will be able to take Raxone and how it is administered.
What is Raxone?
Raxone is a 150mg tablet that contains a substance called idebenone and is to be taken with food and swallowed with a glass of water.
What does the treatment aim to do?
The treatment is designed to slow the progressive rate of decline in breathing ability for those with Duchnene. The drug works by aiming to help slow down the weakening of muscles in respiration and through this slow down the emergence of serious breathing difficulties for those living with Duchenne.
Who is eligible to take Raxone?
Those living with Duchenne who are 10 years old and over who are not taking glucocorticoids (steroids) and through a series of tests are shown to be suffering from a decline of respiratory function. This includes those who have previously been treated with steroids and those who cannot tolerate steroids.
It is important to note that Raxone is not a replacement for steroids and we would advise against those living with Duchenne stopping taking steroids for the sole reasons of being able to take Raxone.
All the details for patients who are eligible to take Raxone can be found in the treatment protocol document provided by the MHRA on Raxone which can be found here.
In a series of online documents the MHRA has outlined in further details why Raxone was given a positive scientific opinion under the EAMS Programme and provides further detail on how Raxone should be taken by patients and administered by clinicians. All these documents can be found here.
Contact Neil Bennett, Director of Research with any questions neil@actionduchenne.org .
FHA Wales staff present Three Peaks cheque to Action Duchenne
