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Further Updates Regarding EMA Approval And How It Affects EAMS

Further Updates Regarding EMA Approval and how it Affects EAMS

With the news regarding EMA Approval we are aware there may be some questions/concerns regarding EAMS. Please see below the following statement we have received from Roche.

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

As a result, risdiplam is now licensed in Northern Ireland for the treatment of 5q spinal muscular atrophy (SMA) in patients 2 months of age and older, with a clinical diagnosis of SMA Type 1, Type 2 or Type 3 or with one to four SMN2 copies.

What does this mean for EAMS in Northern Ireland?

Following marketing authorisation, the Early Access to Medicines Scheme (EAMS) is now closed in Northern Ireland for new patients (i.e. patients identified after EU marketing authorisation). However we 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 we 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.

Roche is in regular contact with the MHRA and we commit to providing you with timely updates if this marketing authorisation application process post-EU exit has a significant impact on the timelines for this medicine.

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.

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