Duchenne's Muscular Dystrophy is a genetic disorder, mainly affecting boys, and is one of the most common disorders causing progressive muscle weakness and degradation. It causes the deterioration of the respiratory muscles which eventually can cause respiratory failure and death.

In recent years there has been a movement within the physician community to drive the understanding that respiratory disease in DMD is treatable in nature, which has meant a belief shift in the way DMD patients with respiratory problems were managed. Traditionally, many caregivers had a more non-interventional approach when managing respiratory disease, but generally take a more aggressive, supportive approach.

Puldysa is the next step in this management approach, in phase III trials, when compared to placebo, patients showed a reduced loss in respiratory function delaying the need for assisted ventilation for three years. Not only does this drastically improve the quality of life for both the patients and their families it also helps to reduce the time that a patient has to remain in hospital. In a retrospective trial (SYROS) the patients had their respiratory function measured for up to 6 years. The results showed that Puldysa reduced respiratory decline by 50%.

The CHMP will now review the submission to determine whether DMD patients should be allowed early access to this potentially life-altering medication, prior to the completion of the necessary clinical trials required for it's full marketing authorisation. The company expects to have a decision by March 2020.