PTC has provided a community statement regarding the recently decision by the CHMP/EMA to not renew the marketing authorisation for Translarna (ataluren), and what this means for availability of the treatment in Great Britain:
Licensed Indication
Translarna is indicated for the treatment of Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene, in ambulatory patients aged 2 years and older. The presence of a nonsense mutation in the dystrophin gene should be determined by genetic testing (Translarna Summary of Product Characteristics (SmPC) for respective countries).
Ataluren in the European Union
The Committee for Medicinal Products for Human Use (CHMP) of the EMA confirmed its original recommendation to not renew the conditional marketing authorisation for ataluren in the European Union on 25 January 2024. Per European regulations, the European Commission has 67 days to adopt the opinion. During this time, there are no changes to the marketing authorization for ataluren. Once this recommendation is confirmed by the European Commission, the medicine will no longer be authorised in the European Union.
Ataluren in Great Britain
The CHMP and European Commission decision does not affect the marketing authorization of ataluren in other countries with country specific independent regulations and marketing authorizations, such as in Great Britain. The MHRA has informed PTC Therapeutics of its intention to perform an independent review of the data. This independent action could allow for the continued authorization of ataluren in Great Britain, despite the decision by CHMP. This process is expected to begin before the end of March 2024 and its duration is unknown for the time being. During this process, ataluren will continue to be licensed and so available to all eligible patients in Great Britain. Patients and their families should discuss management options with their treating clinician. In the EU and Great Britain, ataluren received a conditional marketing authorisation. This medicinal product is subject to additional monitoring. This will allow identification of new safety information. Healthcare professionals are asked to report any suspected adverse reactions.
Action Duchene remains fully committed to supporting the treatments’ continued availability within Great Britain. We are steadfastly dedicated to providing support and advocating for individuals affected by Duchenne muscular dystrophy. As we receive further information, we will keep you updated on developments.
Would you like to know more about Duchenne muscular dystrophy? Increase your knowledge and understanding of Duchenne with our bite-sized science video series.
Section 1 – Facts about Duchenne muscular dystrophy
Section 2 – Signs and Symptoms of Duchenne muscular dystrophy
Section 3 – Diagnosis of Duchenne muscular dystrophy
Section 4 – Crucial Genetic Terminology
Section 5 – Genetics – Blueprint of Duchenne muscular dystrophy
SAVE THE DATE
Our 2024 Annual International Conference will be held on Friday 8th and Saturday 9th November 2024. The conference brings together families, clinicians, researchers and experts for 2 days of sharing knowledge and experience. It is an amazing opportunity to come together as part of the Duchenne community
