Zolgensma

Pharmaceutical company: Novartis Gene Therapy

Zolgensma targets the genetic cause of SMA by delivering a functional copy of the SMN1 (survival motor neuron) gene into nerve cells. This leads to the production of the SMN protein, which is missing in SMA. This protein is essential for the health of nerve cells which help to control muscles.

Zolgensma is a single-dose treatment, meaning it is only administered once. It comes as a liquid and is administered into the bloodstream (known as intravenous infusion).

Zolgensma has been recommended as a treatment option for both symptomatic and pre-symptomatic (where a baby has a genetic change associated with SMA but is not yet showing symptoms) SMA in the UK and is available on the NHS. Only people with type 1 SMA are eligible for Zolgensma. Please consult your child’s clinical team for more information.

England, Wales and Northern Ireland

Read the full National Institute for Health and Care Excellence (NICE) guidance:

• For children showing symptoms
• For pre-symptomatic babies

Scotland

Read the full Scottish Medicines Consortium (SMC) guidance

We are proud to have played a key role in making sure Zolgensma is available for children with type 1 SMA. Throughout the assessment processes, we worked closely with other SMA charities, and patient groups to make sure the experience and views of the SMA community were heard.

Zolgensma has been tested in many clinical trials. The key evidence used during its assessment for use in the UK was from the phase 1 START trial and phase 3 STR1VE-US. These studies generally enrolled babies with type 1 SMA who were six months or younger when they received Zolgensma and had not received other SMA treatments.

In these trials, babies who were given the now-approved dose of Zolgensma lived longer and had improved motor function (being able to control and coordinate muscle movement). In the STR1VE-US trial, most children (82%) did not require ventilation support by age 18 months, and more than half (59%) were able to sit unsupported for at least 30 seconds. Zolgensma was also found to be safe.

Children who received Zolgensma in the phase 1 START trial have been found to maintain the improvements in motor function for an average of 6.9 years. Further data is still being collected to monitor the long-term effects of Zolgensma.

Zolgensma has also been tested in babies who have a genetic change associated with SMA but are not yet showing symptoms (pre-symptomatic). These babies would be expected to develop SMA, so were treated with Zolgensma before 6 weeks of age. After two years, all children were still alive and did not need ventilation or feeding tube support. Many of the children reached developmental milestones, such as being able to stand or walk independently, within timeframes that are typical for children without SMA.

Last updated: 18/02/2025