Other safety alerts
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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 human medicines committee (CHMP) has recommended not renewing the marketing authorisation for Translarna (ataluren), a medicine for treating patients with Duchenne muscular dystrophy 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 recommendation follows the full re-evaluation of the benefits and risks of the medicine during the renewal of its marketing authorisation, including results of a new study which failed to confirm Translarna's effectiveness.
Because Translarna was meant to address an unmet medical need for a serious disease, it received a conditional marketing authorisation in July 2014. This type of authorisation allows a medicine to be authorised on the basis of less comprehensive (complete) data than are normally required, and when the benefits of having it available earlier outweigh any risks associated with using the medicine while waiting for further evidence. Medicines with a conditional marketing authorisation are subject to specific post-authorisation obligations that aim to generate comprehensive data.
In 2016, at the time of the renewal of Translarna’s marketing authorisation, the CHMP assessed new data from a study requested as part of the specific obligations and considered that there were still uncertainties about the medicine’s beneficial effects. The committee therefore requested the company to carry out another study to evaluate effectiveness primarily in a sub-group of patients with a progressive decline in their ability to walk, as they were expected to benefit more from Translarna treatment than the broader patient population.
In this sub-group of patients, the study did not show a statistically significant difference between Translarna and placebo (a dummy treatment) in terms of the distance patients could walk in six minutes after 18 months of treatment; this means that the difference observed may be due to chance. The results in the broader patient population failed to confirm the effect seen in the initial study that supported the marketing authorisation.
In addition, as part of the current marketing authorisation renewal, the CHMP re-evaluated all the available data on the benefits and risks of Translarna. This included an analysis of patient registry data comparing health outcomes of patients who had been treated with Translarna for an average of 5.5 years with those of patients who had not received Translarna. The CHMP noted the results with Translarna in terms of delaying the loss of walking ability, however, the committee could not draw conclusions from these data due to methodological issues and uncertainty linked to the indirect comparison. In addition, there was no clear benefit of Translarna for other outcomes assessed by the CHMP. While re-assessing all the available data, the committee considered that the results of the two studies carried out after authorisation, which were more robust than the patient registry data, did not confirm a beneficial effect of Translarna.
During this re-evaluation, the CHMP consulted a group of experts in neurology as well as patient representatives and took their views into account in its final recommendation.
The CHMP considered that the data now available on Translarna are comprehensive. Based on the totality of the evidence, the committee concluded that the effectiveness of Translarna has not been confirmed in patients with nonsense mutation Duchenne muscular dystrophy, including those who were expected to have a better response to treatment. The committee concluded that Translarna’s benefit-risk balance is negative and 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
In Hong Kong, the above product is not a registered pharmaceutical product.
Ends/Saturday, Sep 16, 2023
Issued at HKT 13:15
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