Risdiplam / Evrysdi / RG7916 

Pages in this website section last updated 30th July 2021

Recent Headlines

30th July 2021
Data for Evrysdi Published in NEJM shows Significant Improvement in Survival and Motor Milestones in Babies with SMA Type 1

23rd June 2021
SMA UK and Many Others Respond to NICE’s Consultation on Access to Risdiplam

14th June 2021
Evrysdi™ improves motor function in pre-symptomatic babies

You can find our Scientific Correspondent’s September 2020 summary of information about Risdiplam, how it works and the clinical trial results, here, and a summary table of the outcomes of the four trials: FIREFISH, SUNFISH, JEWELFISH and RAINBOWFISH, here.

Scroll down further for:

  • Progress Towards Licensing and Access
  • Early Access to Medicines Scheme
  • Global Pre-Approval Access Programme

Progress Towards Licensing and Access

Roche is seeking a broad label for the treatment of SMA in paediatric and adult patients – that’s access for those who have SMA Type 1, Type 2 and Type 3.

Europe

On 30th March 2021, the European Medicines Agency (EMA) approved 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 1-4 SMN2 copies. 

UK

On 20th May 2021, the Medicines and Healthcare products Regulatory Agency (MHRA) approved 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 1-4 SMN2 copies. 

The National Institute for Health and Care Excellence (NICE) and the Scottish Medicines Consortium (SMC) are the two regulatory authorities that manage access to licensed medicines in the UK. They may not recommend access wider than a treatment’s licence / marketing authorisation and they may limit access based on their own assessment of the evidence for the clinical and cost effectiveness of a treatment.

England

NICE started its risdiplam appraisal and held its first committee meeting on 11th May 2021. We joined two other patient experts to advocate for access. We delivered a powerful statement from a parent of a child who has SMA Type 3 and to date had had no access to treatment. The others (a parent of a child with SMA Type 2 and an adult with SMA Type 2 both receiving risdiplam) gave equally powerful statements about the impact of SMA on their lives and the hugely positive changes they have seen with the treatment.

On 2nd June 2021, NICE released its Appraisal Consultation Document (ACD) with an initial ‘no’ to approving risdiplam for NHS funding. Though disappointing this is not unexpected at this stage. NICE was open to comments from anyone by 23rd June, with a second committee meeting scheduled for 13th July. Our thanks to the many people we talked with who planned to make their own personal responses that will echo the voices of those who responded to our surveys and spoke up at the committee meeting. You can read our consultation response (jointly with MDUK), here. The NICE website no longer says when they hope to publish a decision about access. 

Thank you to those of you who completed those surveys we ran jointly with MDUK about your views on whether NICE should recommend that NHS England funds risdiplam. Your responses were crucial for the submission we made together to NICE on 30th October 2020. You can view what we said and survey results, hereYou can read our earlier February 2020 comments in response to NICE’s pre-referral ‘draft scoping’ response request here

Scotland

Roche submitted its application to the Scottish Medicines Consortium (SMC) in June 2021. We have no further news yet as to when the appraisal will start.

READ MORE ABOUT ACCESS ROUTES IN THE UK

PREVIOUS STEPS TO ACCESS in the UK

USA

In November 2019, the Food and Drug Administration (FDA) granted risdiplam priority review as a New Drug Application (NDA). On 7th August 2020, the FDA approved risdiplam for the treatment of 5q SMA in adults and children 2 months of age and older.

Early Access to Medicines Scheme

On 17th September 2020, the Medicines and Healthcare Products Regulatory Agency (MRHA) announced an EAMS scheme that gave access to Risdiplam for those who have SMA Type 1 or Type 2, aged 2 months and older, who are not suitable for authorised treatments.

Marketing authorisation by the European Medicines Authority (EMA) on 30th March 2021, led to applications for eligible people newly identified in Northern Ireland closing in May.

Marketing authorisation by the Medicines and Healthcare products Regulatory Agency (MHRA) on 20th May 2021, led to applications for eligible people in England, Scotland and Wales closing at the beginning of July. 

We worked with local neuromuscular teams, Trusts and Roche, wherever there were access challenges - with some considerable success. However, in a few areas of the country there was no easily accessible scheme for adults, some of whom were unable to travel for an assessment for treatment. For them and anyone who has SMA Type 3, a positive recommendation from NICE at the earliest possible time is vital. In the meantime, we thank Roche for funding the 241 people in the UK who do now have access to risdiplam through this scheme.

For more information about the scheme:

ROCHE FAQS

CLINICIANS FAQS

MHRA PATIENT INFORMATION LEAFLET

PAEDIATRIC AND ADULT CENTRES

Global Pre-Approval Access Programme

Roche’s Pre-Approval Access (PAA) / Compassionate Use Programme (CUP) for risdiplam was available in the UK from 17th August – 17th September 2020. A CUP enables patients who are facing the most urgent medical need and have no other treatment options, to access investigational therapies before these receive regulatory approval.

Access is strictly regulated and varies between countries. FAQs about how it worked are here.

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