Tuesday, July 16, 2024

Duchenne Muscular Dystrophy Treatment Translarna Faces Non-Renewal Recommendation by EMA

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The European Medicines Agency’s (EMA) Human Medicines Committee (CHMP) has recommended not renewing the conditional marketing authorization for Translarna (ataluren), a medicine for treating patients with Duchenne muscular dystrophy. Translarna is used in patients whose disease is caused by a genetic defect called a ‘nonsense mutation’ in the dystrophin gene and who can walk.

The CHMP issued an initial negative opinion on the renewal of the marketing authorization for Translarna in September 2023, which was confirmed in January 2024 following a re-examination requested by the company marketing the medicine. Both rounds of assessment concluded that the effectiveness of Translarna had not been confirmed following a re-evaluation of the medicine’s benefits and risks.

In May 2024, the European Commission asked the CHMP to further consider whether the data available on Translarna were sufficiently comprehensive to conclude on the medicine’s benefit-risk balance, and whether additional real-world data brought to the attention of the Commission during its decision-making process (including three recent publications) might impact the CHMP’s conclusion.

EMA’s New Scientific Review on Translarna’s Effectiveness

Following the appellate judgment of the Court of Justice of the European Union on 14 March 2024, EMA decided to convene a new scientific advisory group on neurology (SAG) for Translarna. The assessment was therefore reset to this stage of the initial renewal procedure. The CHMP reviewed the recent publications, one of which analyzed the combined data of three clinical studies with Translarna (meta-analysis); a second evaluated the level of agreement among 12 clinicians on the use of Translarna; and a third described an initiative to compile data on rare neuromuscular disorders.

The Committee also considered additional information from parents or caregivers of boys affected by Duchenne muscular dystrophy, patient organizations, healthcare professional organizations, and treating doctors. The CHMP carefully reviewed this information and concluded that it did not bring sufficient evidence to confirm the effectiveness of the medicine. In particular, the Committee noted that the methods used to carry out the meta-analysis had several shortcomings and its results could not overrule the negative findings of the individual studies included in the meta-analysis.

The other two publications did not provide new data on the medicine’s effectiveness. The Committee therefore concluded that these additional data do not impact its previous conclusion on the benefit-risk balance of Translarna. The CHMP also considered the advice from the new scientific advisory group on neurology. This group comprised experts, including neurologists and people with lived experience of Duchenne muscular dystrophy, who provided their views on specific questions posed by the CHMP.  During the assessment, people with lived experience of Duchenne muscular dystrophy also presented their views to the CHMP during its plenary meetings.

Duchenne Muscular Dystrophy

EMA Recommends Non-Renewal of Translarna Due to Insufficient Evidence of Effectiveness

The Committee acknowledged the high unmet medical need for an effective treatment for patients with this rare disease. However, it considered that the data available on Translarna are comprehensive and concluded that the benefit-risk balance for this medicine is negative. It, therefore, recommended not renewing the marketing authorization in the EU. EMA will now send the CHMP opinion to the European Commission, which will issue a final legally binding decision applicable to all EU Member States.

Following a request from the European Commission, EMA’s CHMP has reviewed additional data about the renewal of the marketing authorization for Translarna, alongside all the available evidence on the medicine. These additional data included the results from three recent publications, information received from parents or caregivers, patient organizations, healthcare professional organizations, and treating doctors, as well as reports on individual patients treated with Translarna.

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The CHMP carefully reviewed all this information and concluded that it did not bring sufficient evidence to confirm the effectiveness of the medicine. The Committee noted in particular that the meta-analysis had several methodological shortcomings so its results cannot overrule the negative findings of the individual studies.

Duchenne Muscular Dystrophy Treatment Translarna Faces Non-Renewal by EMA

The publication concerning rare neuromuscular disorders did not discuss the effectiveness of Translarna, while the publication regarding the level of agreement among neurologists did not provide new data on the medicine’s effectiveness. In addition, the CHMP took into account the views of a scientific advisory group on neurology, which comprised experts, including neurologists and people with lived experience of Duchenne muscular dystrophy. The group responded to specific questions posed by the CHMP. The Committee acknowledged the high unmet medical need for an effective treatment for patients with this rare disease; however, considering all the available evidence it concluded that the effectiveness of Translarna has not been confirmed in patients with nonsense mutation Duchenne muscular dystrophy.

Therefore, the Committee recommended not renewing Translarna’s marketing authorization in the EU. This means that if this recommendation is confirmed by the European Commission, the medicine will no longer be authorized in the EU. Until then, the marketing authorization for Translarna remains valid. If you have any questions, please speak with your doctor or contact your national competent authority.

Translarna received conditional marketing authorization in the EU on 31 July 2014 for the treatment of patients with Duchenne muscular dystrophy caused by a ‘nonsense mutation’ in the dystrophin gene. Duchenne muscular dystrophy is a serious and rare condition with no authorized treatment, apart from Translarna.

It is a genetic disease that causes a gradually increasing weakness and loss of muscle function, leading to death due to respiratory muscle weakness or cardiomyopathy. Patients with this disease lack normal dystrophin, a protein found in muscles that helps protect muscles from injury as they contract and relax. EMA will now send the CHMP opinion on the renewal application to the European Commission for a final legally binding decision applicable in all EU Member States.

 

Resource: The European Medicines Agency, June 28, 2024

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