Definitive Data Hopes Keep Santhera's Duchenne Drug Alive in UK Despite EMA No

The European Medicines Agency rejected Santhera’s Raxone as a treatment for Duchenne muscular dystrophy but the prospect of definitive data supporting the new indication means idebenone will still be available to patients in the UK under the early access scheme for promising new medicines. 

Duchenne
Santhera's DMD treatment remains on the UK early access scheme • Source: Shutterstock

The UK drug regulator has decided not to drop Santhera Pharmaceuticals’s drug Raxone (idebenone) from its early access to medicines scheme (EAMS) as a treatment for Duchenne muscular dystrophy, despite the European Medicines Agency saying the product should not be approved.

The EAMS designation for Raxone was granted last June and allows Santhera to make the drug available to patients in...

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