NHS England agrees to allow access to Sapphire Trial to enhance treatments for SMA

On Monday 23 January, we let you know that NHS England had made a decision that made it impossible for anyone whose nusinersen or risdiplam treatment is funded via a Managed Access Agreement to apply to join a commercial trial for another potentially disease-modifying drug.

We are therefore delighted to have had the following communication from NHS England:

"In response to communications from stakeholders, NHS England has carefully considered all of the information about this complex situation. We have an ongoing concern that this may impact the ongoing data collection within the managed access agreement if patients already accessing nusinersen or risdiplam through one of the managed access agreements (MAAs) join a commercial trial for another potentially disease-modifying drug. We do however acknowledge that, in this specific case, there has been a relatively long period of data collection (especially for nusinersen), including for some of those individuals who may now be eligible to join the Sapphire trial. In this specific case, therefore, NHS England will exceptionally continue to fund nusinersen or risdiplam through the MAAs for those patients accessing the Sapphire trial. NICE will consider how this will impact the data collection agreement going forward and work with the key stakeholders to ensure the data collection can address the key uncertainties identified by the NICE committee.

This decision has been made on an exceptional case basis. Depending on the nature of the trial, NHS England may not always be able to commit to continuing the funding nusinersen or risdiplam through an MAA where a patient then joins a commercial trial for another potentially disease-modifying drug. Cases will be considered on a case-by-case basis on their individual merits.

Clinicians should always contact NHS England before committing to such commercial trials or to individual patients / families."

Sapphire Trial of Apitegromab funded by Scholar Rock

Applicant must be:

  • receiving risdiplam or nusinersen
  • non-ambulant
  • age 2 – 21 years.

More detail about the trial

Trial Centre Current status / Contact  Cut-off date



March – April 2023.

Internationally competitive recruitment.
Oxford Approved – at set up stage
Great Ormond Street Approved – at set up stage
Liverpool Approved – at set up stage

NHS England has advised that they need additional detail about the RESILIENT trial as decisions on trials are being made on a case-by-case basis.

We will continue to work with clinicians and NHS England and update you when we have further news.