CHMP Publishes Positive Opinion on Risdiplam
26 February 2021
The European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) announced today that it has recommended the approval of Evrysdi™ (risdiplam) for the treatment of 5q 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 - a final decision regarding approval is expected from the European Commission in the next two months, but could be as early as the end of March.
What does this mean for the UK?
The EMA marketing authorisation (licensing) date will apply to Northern Ireland only.
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 (Medicines and Healthcare products Regulatory Agency). 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 Roche anticipate marketing authorisation for England, Scotland and Wales at some point in the next few months.
What does this mean for the Early Access to Medicines Scheme (EAMS)?
As Northern Ireland marketing authorisation timelines are the same as the European Union, the EAMS in Northern Ireland will close to new patients once the EMA licence has been granted.
- Patients enrolled in EAMS before the EMA marketing authorisation will continue to receive supply of treatment.
- Clinicians may have a short period (previously three weeks) from the time the EMA licence is granted (which could be as early as the end of March) to complete enrolment of any patients identified for the EAMS before the licence was granted.
England, Scotland, Wales
The Early Access to Medicines Scheme (EAMS) will close for new patients in England, Scotland and Wales once the MHRA licence has been granted.
- Patients enrolled in EAMS before the MHRA marketing authorisation will continue to receive supply of treatment.
- Clinicians may have a short period (previously three weeks) from the time the MHRA licence is granted (at some point in the next few months) to complete
enrolment of any patients identified for the EAMS before the licence was granted.
If you're not sure what stage your clinician has reached with enrolling you in the EAMS, or have any questions, please speak to them directly.
The Clinical Trials that Supported the Decision
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 SMA Types 2 and 3. Read more about clinical trial updates, here.
Roche has told us:
"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."