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EURORDIS Reacts to Translarna Review: Trust in Scientific Evidence

Maggio 2024

31 May 2024, Paris – EURORDIS-Rare Diseases Europe calls for a public stakeholder meeting following recent developments concerning the conditional marketing authorisation for Translarna.

In January 2024, the Committee for Medicinal Products for Human Use (CHMP) recommended not renewing the conditional marketing authorisation for Translarna based on its evaluation of all available data. However, the European Commission has requested a reassessment of this decision.

Today, the CHMP announced it has restarted the evaluation of an application to renew the conditional marketing authorisation for Translarna (ataluren), a medicine used to treat Duchenne muscular dystrophy.

EURORDIS shares the disappointment of patients and healthcare professionals following the results of three randomised clinical trials that did not demonstrate the clinical benefits of Translarna. Additionally, the data registries submitted by the marketing authorisation holder were not considered methodologically robust by the European Medicines Agency (EMA).

EURORDIS strongly supports the use of real-world data as complementary evidence to clinical trial data, especially when no other type of evidence can be generated. Data from children who have been treated with Translarna over the years can be valuable in decision-making, provided appropriate methods are used for their analysis. However, regulatory decisions must be based on robust scientific evidence to maintain public trust. There is no benefit for patients when decisions are not evidence-based.

This development prompts EURORDIS to suggest holding a public stakeholder meeting to discuss the matter further and reflect on the potential influence of non-scientific factors on regulatory decisions. EURORDIS is calling on the EMA and CHMP to organise this meeting to facilitate exchanges with the patient community and support the CHMP’s decision-making process. This would enable a transparent review of all data related to Translarna, allowing patients and citizens to understand the reasoning behind the decisions and to share their perspectives.

Since its inception, the EMA has been regarded as a body committed to evidence-based decision-making without political influence. Upholding this principle is essential for maintaining patient trust and welfare.

EURORDIS is, therefore, calling on the EMA and CHMP to organise a public stakeholder meeting to facilitate exchanges with the patient community and support the CHMP’s decision-making process.

*Translarna has had conditional approval since 2014 for treating patients aged two years and older with Duchenne muscular dystrophy who are able to walk.


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Contact

Julien Poulain 
Communications Manager 
EURORDIS-Rare Diseases Europe 
Julien.poulain@eurordis.org 
+33 6 42 98 14 32