The European Commission has deferred the negative opinion of the EMA's Committee for Medicinal Products for Human Use (CHMP) on the drug TranslarnaTM (ataluren).
May 20, 2024: The European Commission (EC) has decided against the adoption of the negative opinion and has returned the opinion to the CHMP for re-evaluation. This action allows Translarna to remain on the market and available for patients in Europe, consistent with its current marketing authorization. The EC has directed the CHMP to further consider the totality of evidence, including data from patient registries and real-world evidence, in a revised opinion. This is a positive step to help maintain access to Translarna for boys and young men living with nonsense Duchenne muscular dystrophy mutations in Europe and reinforces the importance of the totality of evidence gathered from clinical trials and the STRIDE real-world patient registry.
In addition, the EMA informed PTC that a decision was made to consider the Scientific Advisory Group meeting for Translarna on September 5, 2023, and all procedural steps that followed as invalid.
This is a great victory for the international Duchenne and Becker community, which will be able to continue using the drug.
What Happened:
On September 15, 2023, after 9 years of use under conditional authorization, the EMA's Scientific Committee for Medicinal Products for Human Use gave a negative opinion to the request to convert Translarna's conditional authorization to full authorization and to renew the existing conditional authorization.
From that day on, Parent Project APs and all other international associations mobilized so that the voices of families and children would be heard. One could not sit on the sidelines and observe; it was necessary to bring all of Europe together in a united front.
Ataluren is a safe and easy drug to use with children. The slowing of disease progression is commonly observed and often exceeds expectations.
The families and caregivers of the children who participated in the study have sufficient evidence that ataluren works:
1. Many families have noticed a slowing of the decline
2. It is showing effects across the range of patients
3. It is showing effects even in the most severely affected patients.
The families' reports on ataluren are in line with data published by the STRIDE registry, which offers a unique opportunity to directly observe the long-term benefits of ataluren in terms of delayed gait loss.
The campaign:
That's why Parent Project aps launched Buy Some Time, a campaign that brings together associations, families, and patients from across Europe under one claim, along with a petition on Change.org that has gathered more than 15,000 signatures.
On January 25, 2024, nonetheless, the CHMP issued its second negative opinion for the renewal of the conditional marketing authorization of Translarna™ (ataluren) following the review procedure.
The choice, therefore, was to appeal to the European Commission. Thus, together with all other international associations Parent Project aps wrote a letter to the European Commission and the EMA asking them to review the decision regarding Translarna™ (ataluren).
The leaders of Duchenne muscular dystrophy (DMD) Clinical Networks in Italy, through the Italian Myology Association, and many other countries also mobilized to express their support for maintaining Translarna™ (ataluren) as a therapeutic option in the European Union and called for a suspension of the decision on the non-renewal of conditional approval.
The voice of the Duchenne community was also heard in the European Parliament thanks in part to the personal efforts of some MEPs.
Ataluren is not the cure, we know that, but it buys us time. And time, for patients with DMD, is crucial as the disease, in the absence of specific treatments, progresses.
Parent Project renews its happiness at this wonderful news and its commitment to Buy Some Time.