New medicines and treatments go through a rigorous process of testing during the development process (e.g. clinical trials). They must be proved to be safe and have some level of effectiveness at treating a condition before they can be licensed for use. In the UK, the organisation that makes this assessment and grants these licences is the Medicines and Healthcare products Regulatory Agency (MHRA). Its licences declare what health condition the medicine and treatment should be used for and the recommended dosage.

Givinostat was approved by the MHRA on 20 December 2024 as a treatment for DMD for patients aged six years and older who are ambulatory (walking) and conditional approval for people who are no longer ambulatory.

New treatments must also go through a health technology assessment to determine if they can be available for free through the NHS.

The National Institute for Health and Care Excellence (NICE) is the body that decides for NHS England, and the Scottish Medicines Consortium (SMC) is the body that decides for NHS Scotland. While there aren’t health technology assessments for the NHS in Wales and Northern Ireland, there is a process to decide on their use for them which often follows the NICE decision.

A first meeting of the NICE committee that is making a decision about givinostat took place on 10 July. The committee has chosen to seek additional information to be considered at a second committee meeting in October.

NICE has said that it wants to hold a second meeting to assess new evidence that it is requesting. It says that:

“The committee has identified specific areas where additional evidence is required to support a fully informed decision. As a result, NICE has launched a four-week targeted call for evidence from stakeholders, ahead of a further committee meeting in October.”

NICE has asked stakeholders and patient experts such as Duchenne UK to help them answer questions the committee still has about givinostat and DMD. We will work with the community to prepare our response.

The SMC is currently appraising givinostat. We are taking part in the process as a stakeholder and the deadline for submissions is 1 September 2025.

Givinostat is available for free to the NHS through an Early Access Programme (EAP) until NICE reaches a decision.

Several hospitals have commenced access programmes, including Leicester Royal Infirmary, the University Hospital of Wales and Singleton Hospital in Swansea, and Great Ormond Street Hospital. While it has been approved across Scotland.

Unfortunately, not all eligible Trusts have given access to givinostat through the EAP.

The decision to seek further answers at a second committee meeting does not affect access to givinostat through the EAP. The EAP will remain open until NICE reaches a final decision as to whether givinostat should be made available routinely on the NHS. We expect those Trusts that have already committed to delivering givinostat to patients through the EAP to continue to progress access.

Anyone already receiving the treatment at the point of a final decision will continue to do so, regardless of the outcome.

However, it means that we must put more pressure on Trusts that are eligible to take part in the EAP but have not yet given access to givinostat through it. We will be holding a drop-in event in parliament to keep up our engagement with MPs, and seek their help in securing access to givinostat through the EAP. It is taking place from: 4-6pm on Wednesday 3 September in Room W2, Westminster Hall, Palace of Westminster. We are inviting people with DMD and their families to come along and talk to MPs about our campaign. We also encourage you to invite your MP too. You can email [email protected] if you’d like to attend or know more about it.