Santhera Pharmaceuticals has announced that the UK’s medicines regulator (the MHRA) has renewed the Early Access to Medicines Scheme (EAMS) for Idebenone in Duchenne. Idebenone will continue to be available to people living with Duchenne with respiratory decline, and who are not taking glucocorticoids.
Idebenone has been available in the UK through EAMS since June 2017. At present, 67 patients with DMD are taking part in the scheme. If you are interested in the scheme, you can find out more about eligibility and how to take part here.
This renewal means the EAMS will continue while a corresponding European marketing authorization application (MAA) is examined. Santhera recently submitted an application for conditional marketing authorization (CMA) for idebenone (under the trademark Puldysa®) to treat respiratory issues in Duchenne.
We welcome the EAMS renewal for idebenone as it provides a therapeutic option for DMD-patients with deteriorating respiratory function in the UK who really have no treatment alternative
Kristina Sjöblom Nygren of Santhera
A note on drug names
In the context of Duchenne, Idebenone, Raxone and Puldysa are essentially different names for the same potential treatment. For various reasons, pharmaceutical companies often choose different names if a drug is being developed for different conditions.
CPG meeting in Scottish Parliament
