When family and societal expectations outweigh science. The case of ataluren for Duchenne.

Duchenne muscular dystrophy is a neurodegenerative disease with no current cure.

In 2014, the FDA and EMA evaluated a new drug for this condition: ataluren. Ataluren was not approved by the FDA due to a lack of efficacy in Duchenne muscular dystrophy, while the EMA granted conditional approval.

After marketing authorization in the EU, the EMA allowed the company to continue performing trials to demonstrate efficacy.

The Spanish National Health System issued a resolution denying reimbursement of this drug due to lack of efficacy. In Spain, virtually all Regional Health Services overruled the Ministry of Health and reimbursed ataluren to Duchenne patients using their own resources, yielding to social pressure and/or seeking political benefit.

A study on the effectiveness of ataluren within Spain National Health System was started, but couldn´t be completed due to lack of involvement from the Regional Health Services.

The cost per patient is estimated at approximately €200,000 annually.

Eleven years later, the EMA withdraws the conditional authorization of the drug after repeated failure to prove efficacy.

• When family and societal expectations outweigh science. The case of ataluren for Duchenne. Gérvas J. Madrid, Spain. December 2025. Download article here.