Apitegromab is a muscle-directed therapy that aims to reverse or restrict the muscle atrophy and weakness. On Friday 11 September 2026, the US Food and Drug Administration (FDA) approved apitegromab for adults and children aged two and over with SMA who are already receiving an SMN2-targeted treatment, namely nusinersen or risdiplam in the US. 

For the SMA community, this was an important milestone. Many people and families have followed the development of apitegromab for a long time, and the US approval represented another potential treatment option for people living with SMA. 

What is happening in Europe? 

The situation in Europe is different from the UK. Scholar Rock previously withdrew its Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) following an issue relating to a third-party manufacturing facility. The company has said it plans to resubmit the application using an alternative manufacturing facility, with the aim of progressing apitegromab through the European regulatory review process as quickly as possible. 

This means that, while apitegromab does not currently have European approval, the regulatory pathway in the EU is still being pursued. No confirmed timeline for resubmission or a decision from the EMA has yet been announced. 

But there is now difficult news for people in the UK. 

On 28 September 2026, Scholar Rock wrote to SMA UK to confirm that, following its assessment of the UK reimbursement landscape, it has made the difficult decision not to submit a Marketing Authorisation Application (MAA) to the UK Medicines and Healthcare products Regulatory Agency (MHRA) at this time. This means that apitegromab will not be coming to the UK for now.

Scholar Rock has said that its ambition remains to bring apitegromab to more patients in the UK and that it will continue to work with SMA UK and the SMA community across the UK towards that goal. The company has also confirmed that people currently receiving apitegromab through clinical trials in the UK will continue to have access to treatment. 

Why is access different in the UK and US? 

It can be confusing to see a medicine approved in the US while there is no route to routine access in the UK. The two countries have different systems for approving and paying for medicines. 

In the US, FDA approval establishes that a medicine can be marketed, but access then depends on individual insurers, public programmes and other payers deciding whether and under what conditions they will cover it. The US system is more fragmented, with many different public and private payers. 

In the UK, regulatory approval and NHS funding are separate steps. A medicine generally needs UK regulatory authorisation and then, for routine NHS access, needs to go through the relevant health technology assessment and funding processes. In England, NICE assesses evidence such as clinical benefit and cost effectiveness, and there are mechanisms for companies and the NHS to agree confidential discounts or other commercial arrangements where appropriate. 

This more centralised system can provide a clearer route to nationally funded access once the necessary decisions have been made, but it also means that a company needs to take part in the UK regulatory and reimbursement process. In Scholar Rock’s case, the company has said that its assessment of the UK reimbursement landscape was a factor in its decision not to submit an MHRA application at this time. 

What does this mean for the UK SMA community ? 

This is understandably disappointing news for people living with SMA, their families and everyone who has followed apitegromab’s development and hoped it would become another treatment option in the UK. 

At the same time, this is not the same as saying that apitegromab will never be available in the UK. Scholar Rock has explicitly said that its ambition is to bring apitegromab to more patients and that it will continue to work with the SMA community towards that ambition. There is currently, however, no confirmed UK application or timeline for access. 

For everyone in the SMA community who has taken part in research, shared their experiences, advocated for better treatments, or continued to hope for more options, your involvement has helped shape the development of treatments like apitegromab. SMA UK will continue to follow developments closely and share confirmed information with the community. 

Read the community statement from Scholar Rock here >

Read more on SRK-015 / Apitegromab, including trials and the timeline of access here >