Agamree

Pharmaceutical company: Santhera

Corticosteroids are thought to reduce damage to muscles by binding to receptors which reduce inflammation. However, when corticosteroids bind to these receptors, they also activate lots of other pathways which lead to a wide range of side effects.

Agamree binds to the same receptors as corticosteroids but avoids activating some pathways linked to corticosteroids side effects. This means Agamree can help reduce inflammation but may have fewer side effects. Agamree comes as a liquid that can be swallowed.

Agamree has been recommended as a treatment option for people aged four and over with Duchenne muscular dystrophy in the UK. Agamree should be available on the NHS from March 2025, please contact your (or child’s) clinical team for more information.

England, Wales and Northern Ireland

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

Scotland

Read the full Scottish Medicines Consortium (SMC) guidance.

We are proud to have played a key role in making sure Agamree is available for people with Duchenne. Working in partnership with other Duchenne charities, we made sure the experience and views of the Duchenne community were heard.

We also supported patient experts to share their children’s experiences with the assessment committees:

• Mandy Roe shared her 12-year-old son’s experience of receiving Agamree through a clinical trial
• Gerry McMenemy shared his son’s experience of living with Duchenne

Evidence from the phase 2b VISION-DMD clinical trial was used in the UK assessment of Agamree. This trial compared treatment with Agamree against both a placebo (dummy drug) and a corticosteroid often used to treat DMD (prednisone). 121 boys aged between four and six years old took part in the trial. They were randomly assigned to receive either one of two doses of Agamree (60 participants), prednisone (31 participants) or placebo (30 participants).

After 24 weeks, boys who received Agamree were able to stand quicker compared to when they started the study, as measured by the time to stand from supine velocity (TTSTAND) test. They also showed improvements compared to the boys who received the placebo.

Boys who received Agamree scored similarly to those who received prednisone on movement measurements, including the NorthStar Ambulatory Assessment, six-minute walk test (6MWT) and TTSTAND. This suggests that Agamree and prednisone have a similar effect in boys over four years with DMD.

Results from the trial have also suggested that Agamree has less of an impact on growth than other corticosteroids. However, other side effects were still reported.

Last updated: 18/02/2025