AdCom for Translarna (ataluren) by the FDA

Advisory Committee (AdCom) for Translarna (ataluren) by the FDA

On September 28, the US Food and Drug Administration (FDA) will hold an AdCom meeting for PTC Therapeutics Translarna (ataluren). This committee allows stakeholders to present additional data on treatment. It is important to ensure an in-depth analysis because Translarna (ataluren) acts on a small population of patients with DMD. It is effective on nonsense mutations, accounting for 13% of people with the disease.

An important event for the DMD community

This is an important opportunity for the DMD community to demonstrate its support for the cause. La Force will table a written testimonial of the effects of the drug on a number of Canadian patients being treated with Translarna (ataluren). Our team is participating in this advisory committee to support access to this treatment because approval for new drugs in Canada are closely linked to decisions made in the United States.

What is an FDA Advisory Committee?

FDA Advisory Committees present recommendations from independent experts on a range of complex scientific, technical and policy issues.

Within the framework of this specific committee, testimonials from patients benefiting from treatment will be presented, as well as those of patient associations, foundations, and independent specialists. This opportunity offered by the FDA allows for the evaluation of data added to those usually observed. In the case of a rare disease such as DMD, it is necessary to evaluate in depth, as only a small population of patients is concerned.

Prior to any advisory committee meeting, it is possible to present a verbal or written request indicating that you wish to intervene at the meeting. In general, advisory committees consist of a chair, several members, consumers, industry stakeholders, and sometimes, patient groups. Other specialists with knowledge in specific areas may be invited to individual committee meetings, as required. Finally, the committees present their recommendations to the agency, and the final decisions are made by the FDA.

Our team will be on-site on September 28 to share the results of this day with you live. Stand by and join our community for fresh news about this advisory committee.

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