Risdiplam / RG7916 

Pages in this website section last updated 7th May 2020

  • What is it?
  • How it works?
  • Clinical Trials
  • Global Access Programme
  • Progress towards licensing and access in the USA, Europe and the UK

Roche Updates SMA UK about their COVID-19 Response Measures and Pre-Approval Access to Risdiplam (17th April)

FAQs re Access to Roche’s risdiplam Compassionate Use Programme (CUP) in the UK 

What is it?

Pharmaceutical company Roche has been leading the development of this small molecule that targets and encourages the SMN2 ‘back-up’ gene to produce a greater amount of functional SMN protein, which is lacking in people with SMA.

Risdiplam is being studied in a broad range of patients who have SMA from birth to 60 years of age. It is an orally-administered liquid designed to provide a sustained increase in SMN protein centrally and peripherally, through daily dosing.

How it works

Risdiplam works by interacting with the SMN2 gene.

Risdiplam is a small molecule drug that specifically modulates how effectively the SMN2 gene is used to make SMN protein. It does this by interacting with SMN2 RNA (which serves as a template from which SMN protein is made) so that more SMN protein is made. 

To make SMN protein from SMN2, the gene must first be copied into RNA (the template). The SMN2 gene is not as good at this process as the SMN1 gene, and therefore produces a lot less SMN protein than SMN1.

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Clinical Trials

Risdiplam is being studied in clinical trials:

FIREFISH: SMA Type 1, 21 children aged 1-7 months
SUNFISH: SMA Types 2 and 3, 51 people aged between 2-25 years
JEWELFISH:trial for people who have previously participated in a trial with SMN2-targeting therapies, or olesoxime, or who received previous treatment with nusinersen; people aged 6 months-60 years 

RAINBOWFISH:trial of risdiplam in infants with genetically diagnosed SMA who are not yet presenting symptoms (pre-symptomatic); 25 children

Trials have recruited from around the world, including for JEWELFISH in the UK. For the latest updates on trial recruitment, please go here.

To date, there have been no drug-related safety findings leading to withdrawal from any study. Roche presented data from the trials at the October 2019 Annual Congress of the World Muscle Society, demonstrating risdiplam has been well tolerated at all dose levels and in all clinical studies for all Types of SMA. In November, it presented further data from its SUNFISH trials.

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Global Access Programme

Roche has initiated a global Pre approval Access (PAA) / Compassionate Use (CU) programme for risdiplam. A PAA / CU programme 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 to these programmes is strictly regulated and varies from country to country. It is already available for patients in participating countries within the European Union with SMA Type 1 and will expand to patients with SMA Type 2 when Roche files for a Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA). This is currently planned for mid 2020.

In the UK, access to this programme is via the Early Access to Medicines Scheme (EAMS). This is regulated by the Medicines and Healthcare products Regulatory Agency (MHRA). Roche UK submitted their application for an EAMS on 4th May 2020. An EAMS application usually takes approximately 90 days to process, although in some circumstances it can be a little less.

In the meantime, Roche UK will consider individual compassionate use applications made by healthcare professionals on behalf of their patients who have SMA Type 1 and meet the programme’s criteria.

Read more about the programme

The 17th April communication from Roche also gives important information, and they've answered some FAQs here.

Progress towards licensing and access in the USA and Europe

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.

In the USA in November 2019, the Food and Drug Administration (FDA) granted risdiplam priority review as a New Drug Application (NDA). A Priority Review status is given for drugs that, if approved, would represent significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions when compared to standard applications. Due to the volume of additional data submitted by Roche, a decision from the FDA is expected by 24th August 2020.

In Europe, Roche anticipate filing a Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) by the middle of 2020.

In the UK, licensing procedures for medicines are managed by the National Institute for Health and Care Excellence (NICE) and the Scottish Medicines Consortium (SMC). Currently, these regulatory authorities may not recommend access wider than an EMA licence and they may limit access based on their assessment of the evidence for the clinical and cost effectiveness of a treatment. Roche is working with both authorities. On 24th February 2020 we submitted our comments in response to NICE’s pre-referral ‘draft remit and draft scoping’ response request. This process gathers evidence so that NICE can decide if this is a suitable treatment for them to appraise to see if they would recommend it is funded by the NHS. You can read what we have said, here. On 7th April 2020, NICE announced its decision to suspend the scoping due to COVID-19. On 6th May 2020 we wrote to NICE urging them to continue with the appraisal.

Read More about Access Routes in the UK

Previous steps to access