Access extended for investigational DMD drug in UK

22 June 2018

The UK's Medicines and Healthcare products Regulatory Agency (MHRA) has renewed the Early Access to Medicines Scheme (EAMS) scientific opinion for Raxone (idebenone) for patients with Duchenne muscular dystrophy (DMD) in respiratory function decline who are not taking glucocorticoids.

Raxone, from Swiss specialty pharma Santhera (SIX: SANN), has been available in this indication in the UK through the EAMS since June 2017. There are currently more than 40 DMD patients enrolled in the EAMS at several specialized DMD centers in the UK.

The aim of the scheme is to provide patients with life threatening or seriously debilitating conditions access to medicines that do not yet have a marketing authorization when there is a clear unmet medical need.

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