Santhera's attempt to win approval for Raxone as a treatment for Duchenne Muscular Dystrophy (DMD) in Europe has hit a giant setback after receiving a negative opinion from European Medicines Agency advisors.

The Committee for Medicinal products for Human Use (CHMP) concluded that an approval for Raxone in DMD cannot be granted on current evidence.

It acknowledged the positive outcome of the Phase III DELOS trial - which the firm said offers statistically significant and clinically relevant evidence that Raxone (idebenone) slows the decline of respiratory function in DMD patients - but said it needed more data to further link the observed treatment effects on respiratory function outcomes to patient benefit.

Respiratory decline is one of the leading causes of death in patients with DMD and there is currently no approved treatment. Santhera stressed that it remains “fully committed to addressing this unmet need” and remains convinced of its’ drug’s benefit in this setting.

The firm said it would now “collect further evidence to strengthen the clinical data package for Raxone” so that it can refile its marketing application in Europe in the future.

“Across Europe about half of patients with DMD aged 10-20 years old are not able to take steroids or have stopped because of side effects. These patients are currently left with no treatment option to slow the progressive decline in their respiratory function,” noted Thomas Voit, MD and Professor of pediatrics at the Great Ormond Street Hospital for Children and University College London, further highlighting the high level of unmet need.

“We will continue supporting Santhera in obtaining additional evidence required to have this medicine approved for this older group of DMD patients,” he added.

Muscular Dystrophy’s UK’s director of Campaigns, Care and Information, Nic Bungay, said the news of the CHMP’s rejection is “disappointing”, as with no alternative treatment to preserve respiratory function when steroids stop working, patients 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.”

He said the charity would now write to the MHRA “urging them to ensure that patients who are benefiting from the drug do not have it withdrawn”.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.”

Raxone won European approval in 2016 as the first treatment for visual impairment in adolescent and adult patients with Leber's Hereditary Optic Neuropathy (LHON), an inherited mitochondrial disease which, if untreated, usually leads to rapid, profound and permanent blindness.