If you or your son is currently taking Translarna, and you would like to express your views on the recent EMA announcement please contact MDI
Dear Member,
We understand you may be affected by the recent decision from the European Medicines Agency (EMA) “Committee for Medicinal Products for Human Use” (CHMP) not to continue authorisation of Translarna for the treatment of nonsense mutation Duchenne muscular dystrophy.
PTC therapeutics have asked for a re-examination of the data with a final decision expected in January 2024. Muscular Dystrophy Ireland is working collaboratively with clinicians in Ireland and other muscular dystrophy patient organisations from Europe to support people during this time.
If you would like to share your experience of taking Translarna, and how CHMP’s decision may impact you as a parent or patient please get in touch with Dympna Mulroy (MDI Research Officer) on mdiresearch@mdi.ie or phone 086 606 6109.
Further information about Translarna and the process to re-examine the decision can be found below.
Kind Regards
Dympna Mulroy
MDI Research Officer
When was Translarna approved?
· The European Medicines Agency (EMA) “Committee for Medicinal Products for Human Use” (CHMP) granted Translarna a ‘conditional marketing authorisation’ in 2014 because the medicine addressed a clear unmet medical need.
· Translarna (Ataluren) has been available in Ireland for people diagnosed with nonsense mutation Duchenne muscular dystrophy since 2019.
What does conditional marketing authorisation mean?
· Conditional marketing authorisation for Translarna was imposed with the condition that the pharmaceutical company (PTC Therapeutics) carry out a further clinical study to evaluate the effectiveness of the product.
· The authorisation of Translarna was granted because the benefit of immediate availability of the product outweighed the risk from less comprehensive clinical data than normally required.
· The company was also required to develop a patient registry to collect data on the use of Translarna in normal clinical practice.
· As a result, PTC Therapeutics have continued to provide the EMA with additional data from the clinical trial and the patient registry, looking at health outcomes of patients who had been treated with Translarna.
What recommendation did the CMHP make based on this additional information?
· On 15th September 2023, EMA announced that the CMHP had recommended not to grant full marketing authorisation of Translarna for the treatment of nonsense mutation Duchenne muscular dystrophy.
· The negative opinion also applies to the renewal of the existing conditional authorisation, meaning Translarna would come off the market.
· This decision was based on all the available data on the benefits and risks of Translarna.
What are the next steps?
· PTC Therapeutics have requested a re-examination of the decision.
· This re-examination process is likely to last until January 2024, and the European Commission have 67 days to enforce the recommendation, that is, by the end March 2024.
· Translarna will remain on the market until the re-examination process is complete and the European Commission (EC) makes a formal decision accepting the results of the re-examination process.
Will Translarna be available during the re-examination process?
· Importantly, during the re-examination process, people currently receiving Translarna may continue to get their medication without interruption. It is important that people do not make any changes to dosing or stop taking Translarna without consulting their physician.
What steps can the DMD community take?
· If you as a parent or patient would like to express your opinion on this decision and share your experiences of what, if any, impact Translarna has had on your quality of life, please contact MDI. All responses must be gathered by 17th November, in line with the re-examination process.