Muscular Dystrophy drug set to be made available on NHS

The Muscular Dystrophy drug ‘Spinraza’, which treats spinal muscular atrophy (SMA) is set to be made available on the NHS.

The drug will be available for children and adults with SMA Types 1, 2 and 3.

NHS England has said that the treatment will be made available to the youngest and most-severely affected patients (SMA Type 1) immediately.

Biogen, NHS England and the National Institute for Health and Care Excellence (NICE) held successful negotiations that will see NHS England offering funding on NICE’s guidance, with the treatment being made available by Biogen.

NICE’s guidance is expected to be published in full in the next few weeks, which will allow babies, children and young adults with SMA Types 2 and 3 to be treated with Spinraza.

Patients are able to receive the treatment through the Managed Access Agreement (MAA), which means they can be given Spinraza while long-term data on the treatment is collected.

Spinraza has been available to SMA Type 1 patients in Scotland since May 2018 and is set to be rolled out for people with Types 2 and 3 soon.

However, Muscular Dystrophy UK and Spinal Muscular Atrophy UK are encouraging NICE to make formal recommendations for the treatment.

They are urging Biogen and NHS England to implement the guidance so that patients can access treatment quickly, as well as urging Northern Ireland and Wales to offer the treatment.