EMA give negative opinion of Duchenne drug Raxone

Published Date
Muscular Dystrophy UK
Breaking news

Muscular Dystrophy UK is disappointed by the news that the EMA have given a negative opinion of Duchenne drug Raxone. However, having spoken to Santhera, the company behind the drug, we would like to reassure UK patients that families enrolled or planning to enrol in the Early Access to Medicines Scheme should not be affected. We welcome the news that Santhera are planning to appeal the decision.

Muscular Dystrophy UK’s Director of Campaigns, Care and Information, Nic Bungay, said:

This is disappointing but not unprecedented in the regulation process. Another Duchenne drug, Translarna, was initially rejected at an early stage but is now available to eligible patients in the UK. However, we are frustrated that this will likely result in a delay to NICE’s evaluation process. We will continue to keep in close contact with Santhera and share further news as we receive it.

Thomas Meier, CEO of Santhera, said:

We are surprised and disappointed by the opinion of the CHMP. Data from the phase III DELOS trial demonstrated statistically significant and clinically relevant evidence that Raxone slows the decline of respiratory function, and reduces the risk of bronchopulmonary complications and hospitalization in patients with DMD not using glucocorticoids.

These patients in the respiratory decline stage currently have no treatment options, and because we are confident that they could benefit from treatment with Raxone, we plan to appeal this opinion and seek re-examination.

The EMA has published a summary of its main concerns in this Q&A document.

Read the full press release from Santhera here.

For further information on Raxone or if you have any concerns about this news please email research@musculardystrophyuk.org or call the national helpline on 0800 652 6352.

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