On Monday Santhera Pharmaceuticals announced that the Medicines and Healthcare Products Regulatory Agency (MHRA) has renewed the Early Access to Medicines Scheme (EAMS) for idebenone (also known as Raxone). The treatment is used to slow the decline of respiratory function in people living with Duchenne muscular dystrophy from the age of 10 years who are currently not taking glucocorticoids.
Idebenone has been available in the UK through EAMS since June 2017, with over 60 patients benefiting from the scheme to date.
For more information about the treatment and the access scheme you can read this factsheet which we produced in collaboration with the other Duchenne charities.
Recently, Santhera Pharmaceuticals also submitted an application for conditional marketing authorization for idebenone (under the trademark name of Puldysa) to treat respiratory issues experienced by people living with Duchenne. We will share any updates on this as soon as we have them.