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Duchenne drug made available to UK patients early

Published on 27/06/17 at 09:00am

UK patients will be given access to Santhera Pharmaceutical’s Duchene Muscular Dystrophy (DMD) drug, Raxone (idebenone), through the Early Access to Medicines Scheme (EAMS). The drug will be made available for those aged 10 years and over with respiratory function decline who are not taking glucocorticoids.

The decision was reached by the MHRA, after it deemed that the drug had safety criteria and had the potential to help patients with the condition, despite not yet having marketing authorisation. Raxone is currently under review by the EMA CHMP to determine whether to give a positive opinion of the drug for marketing authorisation. The MHRA’s EAMS decision is an early indication that experts believe the drug to be both safe and effective.

The action of the drug is to slow respiratory decline; in particular, idebenone is a synthetic short-chain benzoquinone and a cofactor for the enzyme NAD(P)H:quinone oxidoreductase capable of stimulating mitochondrial electron transport, reducing and scavenging reactive oxygen species and supplementing cellular energy levels.

“We're proud to receive the positive EAMS scientific opinion for Raxone in the UK and to have our lead compound designated as a promising innovative medicine, the first for a drug intended for the treatment of DMD," said Thomas Meier, PhD, CEO of Santhera. "This decision allows patients with DMD to receive treatment for respiratory function decline who otherwise would not have access to such treatment options.”

The decision to provide access for the drug through the EAMS is indicative of the limited options for those suffering from DMD. At present, there is no cure and there are few treatments designed specifically to improve DMD symptoms, with steroids, to improve muscle strength, and beta-blockers, to treat heart problems, being the common treatments.

“This is excellent news for patients with respiratory decline in DMD," said Janet Bloor, Chair of the Board of Trustees at Action Duchenne. "The need for new treatments in DMD is very great and the EAMS can help to accelerate access for patients. Action Duchenne was pleased to provide advice during the development of the EAMS program and we are delighted to see this first positive opinion in DMD.”

Ben Hargreaves

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