Basel, 26 February 2021 – Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended the approval of Evrysdi™ (risdiplam) 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.
The CHMP recommendation was completed under the accelerated assessment pathway, which is offered to medicines deemed to be of major interest for public health and therapeutic innovation. A final decision regarding approval is expected from the European Commission in the next two months and will be applicable to all 27 European Union member states, as well as Iceland, Norway, and Liechtenstein. Evrysdi was approved by the U.S. Food and Drug Administration (FDA) in August 2020.
The CHMP recommendation is based on data from two clinical studies designed to represent a broad spectrum of people living with SMA: FIREFISH in symptomatic Type 1 infants aged 2 to 7 months and SUNFISH in symptomatic Type 2 and 3 children and adults aged 2 to 25 years. SUNFISH is the first and only placebo-controlled trial to include adults with Types 2 and 3 SMA.
We have also received the following information from Roche directly:
CHMP positive opinion
Today, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion recommending the approval of risdiplam 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.
The CHMP positive opinion is now referred to the European Commission (EC), which grants marketing authorisations for centrally authorised medicines in the European Union (EU). The EC will review the CHMP recommendation and give its final decision within approximately two months. As risdiplam underwent an accelerated assessment process this final decision could be received as soon as the end of March.
What does this mean for the UK?
As the UK left the European Union during the ongoing EMA assessment of risdiplam, Roche will submit risdiplam for marketing authorisation consideration by the MHRA under the European Commission Decision reliance procedure in the coming weeks. EMA marketing authorisation date will apply to Northern Ireland only. Risdiplam is one of the first medicines to undergo this transition 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 at some point in the next few months. This is a separate process to that for EU member states.
What does this mean for the Early Access to Medicines Scheme (EAMS)?
Once Great Britain marketing authorisation is received, the Early Access to Medicines Scheme (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 Great Britain marketing authorisation will continue to receive supply of treatment.
Northern Ireland process
Northern Ireland marketing authorisation timelines are the same as the European Union. The EAMS in Northern Ireland will therefore close to new patients once the EMA licence has been granted. Patients enrolled in EAMS in Northern Ireland before 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 the new marketing authorisation application process post-EU exit has a significant impact on the timelines for this medicine.
As you may know, 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.
We remain committed to working with you and the SMA community across the UK – thank you for your continued support.
You can read the full press release here.