Negative opinion for Duchenne drug Raxone

Today, the Committee for Medicinal Products for Human Use (CHMP) has returned a second negative opinion for the Duchenne drug Raxone. This is disappointing news for people with Duchenne muscular dystrophy who could benefit from Raxone.

Raxone is developed by Santhera Pharmaceuticals and is thought to slow the decline of respiratory function in patients Duchenne muscular dystrophy who are not taking steroids. In June 2017, Raxone was approved via the Early Access to Medicines Scheme (EAMS). However, in September 2017, the CHMP, provided a negative opinion for the use of Raxone in patients with Duchenne. Santhera resubmitted the clinical trial data again but the CHMP has delivered a negative opinion again.

Muscular Dystrophy’s UK’s Director of Campaigns, Care and Information, Nic Bungay, said:


This is disappointing news for the people with Duchenne muscular dystrophy who could benefit from Raxone. With no alternative treatment to help preserve respiratory function when steroids stop working, adults with Duchenne can struggle to cough and clear their airways.

Raxone offered many people with Duchenne hope when it was made available via the Early Access to Medicines Scheme.  We are in close contact with Santhera about what happens next for those patients in the UK currently receiving Raxone as part of the EAM scheme.  We will today write to the MHRA urging them to ensure that patients who are benefiting from the drug do not have it withdrawn.

Duchenne has few treatment options and no cure. We must continue to look at innovative ways of bringing promising Duchenne treatments at the earliest possible stage to those who could benefit from them.

You can read more about the decision in Santhera's press release. 


For more information contact our Campaigns team