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The National Institute for Health and Care Excellence (NICE) has announced that more patients with spinal muscular atrophy (SMA) will benefit from using nusinersen as a treatment option.

Nusinersen is sold with the brand name Spinraza and is manufactured by Biogen.

The NICE’s latest recommendation follows a review of data collected as part of the Managed Access Agreement (MAA).

The review, involving Biogen, patient groups, clinicians and other stakeholders, assessed whether new evidence that had become available would support a change in the MAA treatment eligibility criteria.

Specifically, it looked at whether people with type III SMA who are unable to walk can benefit from nusinersen and therefore should be included in the MAA.

The MAA is a special arrangement between the NHS and Biogen. It allows patients to access treatment with nusinersen while at the same time collecting data on its impact in groups where additional evidence is required to address the uncertainties identified by the independent NICE appraisal committee in its original assessment of nusinersen.

The MAA is also designed to address the financial risk and challenges for implementation in the NHS. It is not routine commissioning of nusinersen in line with its marketing authorisation.

Meindert Boysen, a director at NICE, said: “There are people with SMA who are not able to access treatment with nusinersen under the terms of the MAA which began in July 2019.

“At the time we made a commitment that we would review new evidence on the potential benefits of nusinersen for type III SMA patients who are not currently receiving it. We are therefore pleased that the review has concluded that it is appropriate to extend the clinical eligibility criteria to allow access to nusinersen for type III SMA patients who aren’t able to walk.

“It will also allow the removal of the rule which meant that patients who had lost the ability to walk needed to regain that ability within 12 months of treatment in order to be eligible for further treatment.”

Samuel Cooper, from ‎Biogen UK and Ireland, said: “Biogen is delighted by this decision for non-ambulant type III SMA patients who previously had no therapeutic options available. Biogen remains fully committed to supporting NHS England to ensure this positive decision translates into services for all eligible patients, enabling access to nusinersen, without delay.”

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