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MHRA renews early access medicines scheme support for Duchenne muscular dystrophy treatment idebenone

Raxone▼, for Duchenne muscular dystrophy from Santhera (UK) Ltd

The Medicines and Healthcare products Regulatory Agency (MHRA) has renewed for a further year the Early Access to Medicines Scheme (EAMS) scientific opinion for idebenone (Raxone▼) for patients with Duchenne muscular dystrophy (DMD) in respiratory function decline who are not taking glucocorticoids. With this renewal, the MHRA again confirmed its positive scientific opinion for idebenone under the EAMS.

The aim of EAMS is to provide earlier availability of promising new unlicensed medicines to UK patients that have a high unmet clinical need. The MHRA scientific opinion provides benefit and risk information to doctors who may wish to prescribe the unlicensed medicine under their own responsibility.

When applying for the EAMS renewal, Santhera submitted new efficacy data (including results from the long-term SYROS study) supporting the potential for a clinically relevant preservation of respiratory function during idebenone treatment for up to six years in a real-world setting. By renewing the EAMS, the MHRA has enabled access to idebenone for DMD patients with the highest need.

Recently the manufacturers, Santhera, has submitted a conditional marketing authorization application to the European Medicines
Agency for idebenone (under the trademark Puldysa®) to treat respiratory function decline in DMD.

Raxone is licensed in the EU to treat a rare condition called Leber's hereditary optic neuropathy. It is used as an unlicensed medicine in the treatment of patients with DMD. The manufacturer has applied for approval for idebenone, under the brand name Puldysa, to treat DMD.

About idebenone - Raxone (source MHRA)
Raxone is already licensed as a medicine to treat a rare eye condition called Leber’s hereditary optic neuropathy (LHON). Under EAMS it is being made available as an unlicensed medicine to slow the decline in respiratory (lung) function in patients with Duchenne’s muscular dystrophy (DMD), a degenerative disease of the muscles. It is only given to patients not taking glucocorticoids (steroids) who in addition have clear evidence of deteriorating lung function in tests that are routinely conducted by specialists involved in the care of DMD patients. Patients may not notice breathing difficulties at this stage but this becomes inevitable with time, if left untreated.

The aim of Raxone treatment is to slow down the weakening of muscles involved in respiration and, with this, potential slowing of the emergence of serious breathing difficulties in later stages of the disease. It may take some considerable time for these longer term benefits to become evident given the slow rate of progression of the disease. Early treatment may be warranted to prevent complications
related to respiratory muscle weakening such as infection (pneumonia) and low oxygen levels (hypoxia).

Raxone is only given to patients 10 years and older, the age range when decline in lung function becomes apparent. Lung function decline must be confirmed by spirometry (which measures the maximum flow and amount of air that can be exhaled in one breath) before Raxone is given.

Raxone is given without glucocorticoids and is only given to patients previously treated with glucocorticoids such as prednisolone or deflazacort, or in patients who are not candidates for continuation or commencement of glucocorticoid treatment. This group of patients represents those with the highest unmet need for whom no other medicinal treatment options are currently available.

Raxone was first made available in the EU in 2015. It is manufactured by Santhera.


European Medicines Agency website page for Raxone
Accessed 24/06/19
Links available in full article

MHRA information on idebenone in treating DMD

New medicines and vaccines that are under additional monitoring have an inverted black triangle symbol (▼) displayed in their package leaflet and summary of product characteristics, together with a short sentence explaining what the triangle means – it does not mean the medicine is unsafe. You should report all suspected adverse drug reactions (ADRs) for these products. ADRs can be reported by your doctor, pharmacist or online via the Yellow Card system.

Reporting of suspected adverse reactions

Reporting suspected adverse reactions (side effects) after authorisation of the medicinal product is important. It allows continued monitoring of the benefit/risk balance of the medicinal product. Healthcare professionals or patients are asked to report any suspected adverse reactions via the Yellow Card Scheme at or search for MHRA Yellow Card in the Google Play or Apple App Store.

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