Tous les comptes rendus municipaux disponibles sur Pappers Politique !

Pappers Politique vous permet d'accéder et d'explorer les comptes rendus, délibérations et procès verbaux de conseils municipaux :
  • Anticipez les besoins et projets dans les municipalités et plus particulièrement les appels d'offre à venir.
  • Identifiez les communes actives sur vos thématiques de prédilection
Réserver une démo avec un expert

Duchenne muscular dystrophy: authorisation for the marketing of Translarna as requested by sufferers and their families

Question écrite de M. Alessandro PANZA - Commission européenne

Question de M. Alessandro PANZA,

Diffusée le 13 février 2024

Subject: Duchenne muscular dystrophy: authorisation for the marketing of Translarna as requested by sufferers and their families

Duchenne muscular dystrophy is a very rare genetic disease that appears in early infancy and leads to death at around the age of 25. The severest symptoms are already occurring by the age of ten, and include an inability to walk and then loss of use of the arms, followed by lung complications and then heart complications.

The disease is incurable, but a drug called Translarna (active substance Ataluren), which is currently on the market, slows the advance of the disease and can help ensure those afflicted remain mobile for up to three and a half more years.

On 26 January 2024, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) issued a negative opinion on renewal of the marketing authorisation for Translarna in Europe.

According to clinical evidence, this medicine does not present a risk to patients’ health.

Besides this, the trial shows taking this medicine to have extended patients’ ability to walk by as much as three and a half years.

The medicine and those three and a half years of better quality of life offer real hope not just to sufferers, but also to their families.

Can the Commission state whether it will grant an extension for the marketing of Translarna and support further and prolonged clinical trials on Translarna?

Submitted:14.2.2024

Réponse - Commission européenne

Diffusée le 18 avril 2024

Answer given by Ms Kyriakides on behalf of the European Commission (19 April 2024)

The European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) considered the totality of data available on Translarna during the renewal of the marketing authorisation, including various clinical studies, patient registry data and contributions received from families, individual physicians, patients and healthcare professional organisations.

The CHMP concluded that the effectiveness of Translarna has not been confirmed and the benefit-risk balance is negative and recommended not renewing its marketing authorisation. More information on Translarna is available on the EMA website (1).

EU legislation requires that a marketing authorisation is maintained if a positive benefit/risk balance is confirmed by the EMA. This is no longer the case for Translarna. In accordance with the EMA scientific opinion, the Commission is preparing a decision to be applicable in all Member States.

The current marketing authorisation holder for Translarna can decide to carry out additional studies to show a positive benefit/risk balance of this medicine for the treatment of Duchenne muscular dystrophy and submit them to the EMA for review in the context of a new marketing authorisation procedure.

Moreover, the EU pharmaceutical legislation supports through incentives and free scientific advice, the development of medicines for diseases where there is an unmet medical need, such as the Duchenne muscular dystrophy.

1 ∙ ⸱ https://www.ema.europa.eu/en/news/ema-recommends-non-renewal-authorisation-duchenne-muscular-dystrophy-medicine-translarna;

https://www.ema.europa.eu/en/news/ema-confirms-recommendation-non-renewal-authorisation-duchenne-muscular-dystrophy-medicine-translarna | | ( | | )

Questions similaires

Déposée le 22 février 2022 à 23h00
Similarité de la question: 65% Similarité de la réponse: 71%
Déposée le 11 mars 2021 à 23h00
Similarité de la question: 46% Similarité de la réponse: 71%
Déposée le 17 février 2021 à 23h00
Similarité de la question: 55% Similarité de la réponse: 67%
Déposée le 7 février 2019 à 23h00
Similarité de la question: 66% Similarité de la réponse: 69%
Déposée le 9 mars 2021 à 23h00
Similarité de la question: 63% Similarité de la réponse: 69%
Déposée le 24 août 2021 à 22h00
Similarité de la question: 59% Similarité de la réponse: 69%