Raxone (also known as idebenone), developed by pharmaceutical company Santhera, is used to slow the decline of respiratory function in patients with Duchenne muscular dystrophy from the age of 10 years who are currently not taking glucocorticoids.
Decisions on approval for Raxone
In June 2017, Raxone was approved in the UK by the Medicines and Healthcare Products Regulatory Agency’s via the Early Access to Medicines Scheme (EAMS).
However, in September 2017, the European Medicines Agency (EMA) provided a negative opinion for the use of Raxone in patients with Duchenne. Santhera resubmitted the clinical trial data again but the EMA delivered a negative opinion again in January 2018 and has invited Santhera to provide further evidence.
Muscular Dystrophy UK and other Duchenne charities were invited by the Medicines and Health products Regulatory Agency (MHRA) to provide patient testimonies on the impact of the condition to assist the process, and following this the MHRA decided in June 2019 that it is maintaining its positive scientific opinion through the EAMS.
Access to Raxone
Raxone 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.
In collaboration with the other Duchenne charities we have produced two guides on access to Raxone:
- For individuals and families interested in accessing the treatment
- For clinicians and healthcare professionals
For more information, please contact Clare Lucas on firstname.lastname@example.org or call 020 7803 4838.