While the EMA’s Committee for Medicinal products for Human Use (CHMP) recommended a spree of treatments over the past week, Santhera’s Raxone (idebenone) was left out, rejected by the advisory panel and given a negative opinion in the treatment of Duchenne muscular dystrophy (DMD).

Current evidence could not form an adequate basis for approval, the panel argued. While it recognised the Phase 3 evidence put forward in support of the application – evidence which Santhera argued provides statistically significant and clinically relevant support that Raxone (idebenone) slows the decline of respiratory function in DMD patients – the panel judged that more data was necessary to better illustrate the link the treatment’s effects to patient benefit.

Despite the decision, Santhera noted that it remains "fully committed to addressing this unmet need", confirming that it would continue to "collect further evidence to strengthen the clinical data package for Raxone".

Nic Bungay, Director of Campaigns, Care and Information at Muscular Dystrophy’s UK, also remarked: "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," he continued. "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 We will today write to the MHRA urging them to ensure that patients who are benefiting from the drug do not have it withdrawn.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."