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| European Union: EMA recommends non-renewal of authorisation of Duchenne muscular dystrophy medicine Translarna |
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European Medicines Agency (EMA) announces that its Committee for Medicinal Products for Human Use (CHMP) has recommended not renewing the conditional marketing authorisation for Translarna (ataluren), a medicine for treating patients with Duchenne muscular dystrophy. Translarna is used in patients whose disease is caused by a type of genetic defect called a ‘nonsense mutation’ in the dystrophin gene and who are able to walk.
The CHMP issued an initial negative opinion on the renewal of the marketing authorisation 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) may impact the CHMP’s conclusion. In addition, following the appellate judgment of the Court of Justice of the European Union of 14 March 2024 in Case C-291/22 P, 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.
In relation to the request from the European Commission, the CHMP reviewed the recent publications, one of which analysed the combined data of three clinical studies with Translarna already assessed by the CHMP (meta-analysis); a second evaluated the level of agreement amongst 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 received from parents or caregivers of boys affected by Duchenne muscular dystrophy, patient organisations, healthcare professional organisations, and treating doctors, as well as reports on individual patients treated with Translarna.
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.
For the present opinion, the CHMP also considered the advice from the new scientific advisory group on neurology. This groups comprised experts, including neurologists and people with lived experience of Duchenne muscular dystrophy, whoprovided 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.
In reaching its opinion, the CHMP took all the above information into account as well as the evidence accumulated on the medicine since its marketing authorisation in 2014. This evidence includes data from the main study that supported the authorisation, and from two post-authorisation studies that were requested by the CHMP to confirm the medicine’s effectiveness.
The two post-authorisation studies failed to confirm the benefits of the medicine, including in patients with a progressive decline in their ability to walk who were expected to be more sensitive to treatment with Translarna. The CHMP also reviewed data from a study comparing two patient registries. However, due to differences between the two registries and uncertainty linked to the indirect comparison, no firm conclusion on the effectiveness of the medicine could be drawn from these real world data; and, the Committee considered that this study cannot counterbalance the findings from the failed post-authorisation studies. After a thorough assessment of the totality of the data, the Committee concluded that the effectiveness of Translarna has not been confirmed in patients with nonsense mutation Duchenne muscular dystrophy.
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, as described above, are comprehensive and concluded that the benefit-risk balance for this medicine is negative. It therefore recommended not renewing the marketing authorisation in the EU.
EMA will now send the CHMP opinion to the European Commission, which will issue a final legally binding decision applicable in all EU Member States.
Please refer to the following website in EMA for details:
http://www.ema.europa.eu/en/news/ema-recommends-non-renewal-authorisation-duchenne-muscular-dystrophy-medicine-translarna-0
In Hong Kong, Translarna (ataluren) is not a registered pharmaceutical product. Related news was previously issued by EMA, and was posted on the Drug Office website on 27 Jan 2024 and 16 Sep 2023.
Ends/Saturday, Jun 29, 2024
Issued at HKT 12:00
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