Spinraza

Pharmaceutical company: Biogen

Spinraza is a small piece of man-made genetic material (called an antisense oligonucleotide). It targets the ‘back up’ SMN2 (survival motor neuron) gene so that it produces full-length, functional SMN protein. This protein is essential for the health of nerve cells which help to control muscles.

Spinraza comes as a liquid and is administered directly into the fluid around the spine and brain (cerebral spinal fluid) through an injection into the spine (lumbar puncture).

Eligible people with SMA can access Spinraza through a Managed Access Agreement (MAA) in England, Wales and Northern Ireland. This agreement means Spinraza is currently available on the NHS at a discounted price.

Children with SMA in Scotland can access Spinraza through the NHS.

In 2019, the National Institute for Health and Care Excellence (NICE) recommended Spinraza for use on the NHS in England, Wales and Northern Ireland. However, NICE requested the collection of additional data to confirm how well Spinraza works. NICE therefore made Spinraza available through a Managed Access Agreement (MAA), so that eligible people with SMA could access the treatment while the additional data was collected. Once additional data is collected, NICE will reassess Spinraza and decide whether it should be permanently made available on the NHS.

Spinraza is being assessed alongside a second SMA treatment, Evrysdi (also known as risdiplam), in a process called a Multi-Technology Appraisal (MTA). The treatments are being assessed together as they are both designed to increase SMN protein levels, and the assessments will cover similar topics. Read about the NICE process of Spinraza and Evrysdi.

In 2018, Spinraza was recommended for use on the NHS in Scotland under the ultra-orphan process, and its availability was widened in 2019. Read about the Scottish Medicines Consortium (SMC)’s assessment of Spinraza.

NICE are currently reassessing Spinraza, with a decision expected in 2026. As there have been some unexpected delays with the assessment, NICE are planning to extend the MAA. This means current and new patients can still access Spinraza while the assessment continues.

We were heavily involved in the first assessment of Spinraza by NICE, which led to access of Spinraza through the MAA. We are also a member of the Managed Access Oversight Committee (MAOC) which oversees the operation of the agreement, including how the additional data is collected and the discussion of any potential changes to the eligibility criteria.

We are currently working in partnership with the charities SMA UK and Treat SMA throughout the NICE reassessment to make sure the experience and views of the SMA community continue to be heard.

Spinraza has been tested in several clinical trials, including in babies and young children. In these trials Spinraza was found to be safe.

In the phase 3 ENDEAR trial, 122 infants with type 1 SMA aged under eight months were randomly assigned to receive Spinraza (81 participants) or placebo (dummy drug, 41 participants). The trial found that infants who received Spinraza had improved survival and motor function (measures of movement), compared to infants who received placebo. Over half (51%) of infants who received Spinraza reached a motor development milestone (such as rolling over, crawling), measured by the Hammersmith Infant Neurological Examination. None of the infants in the placebo group reached a motor milestone.

In the phase 3 CHERISH trial, 126 children aged between two and nine years, whose SMA symptoms started after six months of age took part in the trial. They were randomly assigned to receive Spinraza (84 participants) or placebo (dummy drug, 42 participants). After 15 months, children who received Spinraza showed improvements in completing activities related daily living, such as sitting, rolling and standing. This was measured by the Hammersmith Functional Motor Scale Expanded for SMA (HFMSE).  Over half (57%) of children who received Spinraza increased their HFMSE score by at least three points from the start of the trial to month 15, compared to 26% of children who received placebo.

Last update: 18/02/2025