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Breaking news: Risdiplam to be made available on NHS in England

18 November 2021

A third treatment for Spinal Muscular Atrophy is set to be made available on the NHS in England, following draft guidance published by NICE today.

The guidance will be adopted by the NHS if there are no objections by 2 December.

Risdiplam – also known as Evrysdi – has been recommended for use under a Managed Access Agreement (MAA) to treat the condition. 

This means Risdiplam will be available on the NHS in England while long-term data is gathered and before final funding decisions are taken. 

MDUK is delighted at today’s announcement, especially as initial draft guidance published earlier this summer did not recommend the treatment for use on the NHS in England. 

What is Risdiplam and who can access it?

Risdiplam is an oral drug manufactured by Roche. It is taken daily and increases SMN protein levels by targeting the SMN2 gene.

Risdiplam will now be available for everyone with SMA Types 1 to 3 or with 1 to 4 SMN2 copies under the MAA. It is estimated that around 1,500 people will be able to access the treatment. 

Further details on access to Risidiplam can be read in NICE’s final appraisal document here.

Those in England seeking guidance on access should speak with their specialist healthcare team for advice. 

Why is this news important?

SMA is a devastating condition which, in the most severe cases, rarely sees babies survive beyond two years of age. 

In June 2021 NICE published draft guidance which did not recommend the treatment for use on the NHS in England. 

As part of the consultation process following that news, we submitted a joint response with Spinal Muscular Atrophy UK (SMA UK) that reiterated the evidence we had previously submitted. 

We shared details on the benefit of the treatment and urged NICE to reverse its decision. 

Risdiplam can be taken at home, making it an accessible and flexible treatment option. 

Clinical evidence shows that Risdiplam improves the ability to sit up, stand or walk for people with SMA Type 1, 2 and 3 and that it may be effective for people before they start showing symptoms of SMA. 

There is also some evidence suggesting that people with type 1 SMA live for longer on Risdiplam and that it may be an effective treatment for those with pre-symptomatic SMA. 

Ross Hovey, from Cambridgeshire, has been treated with Risdiplam since April 2021.

He said: “Today’s announcement is fantastic news for the SMA community as a whole and another breakthrough in the progression of treatments. This news removes some anxiety around continued access to the drug. I’ve seen immediate positive impacts in terms of strength and some minor motorneuron functional gains and energy. In my first check up I performed marginally stronger on the assessment and my breathing has also not deteriorated for the first time in many years between check ups. I’m hoping that I continue to see no declines and gains when I can balance exercise and work better. In terms of benefits I do notice my driving has improved and my posture, head control and stamina are all stronger.” 

What do we think, and what happens next?

We’ve worked hard with SMA UK through our roles as official consultees in the appraisal process to represent the voices of those in our community, and we’ll continue to advocate for the full provision of Risdiplam across the UK. 
Dr Kate Adcock, Director of Research and Innovation at MDUK, said: “We’re really pleased that NICE has reversed its earlier decision and that Ridsiplam will be made available on the NHS in England under a Managed Access Agreement. We’ve worked closely with SMA UK to represent the SMA community throughout this process so today’s announcement is excellent news. However, we must not be complacent. We will continue to call for new born SMA screening because we know that the earlier treatments are delivered the better chance children with SMA have of living well for longer.” 

Liz Ryburn, Support Team Manager at SMA UK said, “We’re absolutely delighted that this third treatment for spinal muscular atrophy will now be available in England. It offers children, young people and adults who live with this progressive and complex neuromuscular condition, an oral medication that can be taken daily at home to combat the deterioration they otherwise face. Many are powerchair users who have shielded throughout the pandemic and face huge challenges with travel to hospital for treatment – Risdiplam offers so much more flexibility in people’s lives. We will continue to advocate with MDUK for its provision across all the UK.”

What other SMA treatments are available in England?

Spinraza (also known as nusinersen) is currently available for people with SMA through an MAA. More information can be read here.

Zolgensma (also known as onasemnogene abeparvovec) is a gene therapy for babies with SMA Type 1 if aged six months or younger, or if aged between seven and 12 months and agreed by the national multidisciplinary team. 
More information on Zolgensma can be read here.

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