EMA Recommend Against Renewing Translarna for DMD

The Committee for Medicinal Products for Human Use (CHMP), part of the European Medicines Agency, has again advised against renewing approval for Translarna (ataluren), a drug used to treat Duchenne muscular dystrophy (DMD) caused by a nonsense mutation in the dystrophin gene.

The European Commission, which makes the final decision on medications in the EU, is expected to give a final ruling in about two months. In the meantime, the company that makes Translarna, PTC Therapeutics, is working to keep the drug available.

“As Translarna will remain authorised in Europe pending review by the European Commission, we will continue to ensure Translarna is available to boys and young men living with nonsense mutation Duchenne muscular dystrophy. In addition, PTC will ensure the European Commission has all possible evidence to support continued authorisation.” Matthew B. Klein, MD, CEO of PTC, said in a company press release.

“The CHMP acknowledges the high unmet medical need for an effective treatment for patients with Duchenne muscular dystrophy. However, based on all the evidence accumulated, it concluded that the effectiveness of Translarna has not been confirmed in patients with Duchenne muscular dystrophy caused by a nonsense mutation, including those who were expected to have a better response to treatment,” the agency stated in a press release.

MDI acknowledge that this news is devastating for those impacted by this decision. If you or your child is receiving Translarna, you should speak to your doctor about this decision and what it means for you or your child’s treatment.  Information about the review process is available here.