| Announcements

June 30, 2020

The Committee for Medicinal Products for Human Use (CHMP), an arm of
the European Medicines Agency, has recommended a label update for Translarna (ataluren) to enable its use in Duchenne muscular dystrophy (DMD) patients who lost their ability to walk.

The committee has specifically voted to remove the statement “efficacy has not been demonstrated in non-ambulatory patients” from the therapy’s label. If accepted by the European Commission, which makes the final decision based on CHMP’s recommendation, the change is also expected to support continued reimbursement and access to the medication in patients who become non-ambulatory over the course of treatment.

Translarna is approved — with conditions — in the European Union for DMD
patients 2 years and older who carry nonsense mutations in the DMD gene and are able to walk. Butclinical trials and real-world evidence have demonstrated it is also able to improve walking ability and patient outcomes beyond the loss of walking ability.

Long-term data from Study 019 (NCT01557400), an open-label extension study, found that Translarna preserves lung function in non-ambulatory (unable to walk without assistance) DMD patients for an additional four years, compared to an
historical group of patients receiving standard treatment in the Duchenne Natural History Study (DNHS; NCT00468832).

The DNHS, conducted by the Cooperative International Neuromuscular Research Group, is the largest observational, longitudinal study assessing the natural course of the disease.

Results from the ongoing STRIDE registry (NCT02369731) — an observational study across more than 50 care centers in Europe and Israel — also demonstrated that boys receiving Translarna plus standard treatment retained their ability to walk independently for an additional 3.5 years compared to those receiving standard care alone in the DNHS.

Translarna also preserved lung function in boys receiving it in the STRIDE trial.
Compared to their counterparts in the natural history study, these boys gained healthy lung function for longer periods. Respiratory failure is a leading cause of death among DMD patients, so preserving lung function is a key therapeutic priority.

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