SMA drug Evrysdi (risdiplam) approved in the EU

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Research Team
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The European Commission (EC) has granted marketing authorisation to Evrysdi™ (risdiplam) for the treatment of spinal muscular atrophy (SMA). Evrysdi has been authorised for people aged two months and older with 5q SMA Type 1, Type 2 or Type 3 with one to four SMN2 copies.

Risdiplam is an oral drug that increases SMN protein levels by targeting the SMN2 gene. The EC approval is based on data from the FIREFISH and SUNFISH clinical trials.

Under the terms of the UK’s departure from the European Union (EU), EU marketing authorisation will apply to Northern Ireland. Risdiplam remains an unlicensed medicine in the rest of the UK.

Roche has shared the following information on what this announcement means for the UK and the Early Access to Medicines Schemes (EAMS):

What does this mean for EAMS in Northern Ireland?

Following marketing authorisation, EAMS is now closed in Northern Ireland for new patients (i.e. patients identified after EU marketing authorisation). However, Roche have agreed with the MHRA that, for patients already identified at the time of EU marketing authorisation, new applications can be accepted up to six weeks after EU marketing authorisation. These previously identified patients – should they fulfil the EAMS eligibility criteria – are therefore still able to receive risdiplam through the EAMS. Existing patients participating in the EAMS will continue to be treated under the terms of the EAMS.

What does this mean for the rest of the UK?

As the UK has left the European Union, EU marketing authorisation date applies to Northern Ireland only. Roche has submitted a marketing authorisation application for risdiplam in Great Britain for consideration by the Medicines and Healthcare products Regulatory Agency (MHRA) via the European Commission Decision reliance procedure.

Risdiplam is one of the first Roche medicines to undergo this approval pathway. As this is a new process it comes with some uncertainty as to the precise timelines, however Roche anticipate Great Britain marketing authorisation in May 2021.

What does this mean for the Early Access to Medicines Scheme (EAMS)?

Once Great Britain marketing authorisation is granted, the EAMS will close for new patients (i.e. patients identified as eligible for risdiplam after Great Britain marketing authorisation) in line with the MHRA process. Patients enrolled in EAMS before the Great Britain marketing authorisation will continue to receive supply of treatment.

A National Institute for Health and Care Excellence (NICE) appraisal for risdiplam is currently underway. Risdiplam will be submitted to the Scottish Medicines Consortium (SMC) in the first half of 2021.

Roche leads the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics. For more information about the marketing authorisation read PTC Therapeutics press release.


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