PTC Provides Update on Translarna™

PTC Provides Update on Translarna™ (ataluren) Application for Label Expansion

June 28, 2019 > Original press release <

PTC Therapeutics, Inc. announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has completed their review of a proposed indication extension of Translarna™ (ataluren) for the treatment of patients with nonsense mutation Duchenne muscular dystrophy (nmDMD) who are non-ambulatory. While the CHMP adopted a negative opinion of the extension, PTC was informed by EMA representatives that the European Public Assessment Report (EPAR) would be updated to clarify that patients who start Translarna while ambulatory are not required to discontinue treatment after the loss of ambulation.


Translarna is currently indicated for ambulatory Duchenne patients who are over two years of age; the requested extension would have allowed for the inclusion of non-ambulatory patients in the label. PTC plans to seek a re-examination of the procedure within the next two weeks and expects the new examination to last approximately four months. 


Marcio Souza, Chief Operating Officer, PTC Therapeutics, Inc – “While we are disappointed with the current outcome of the label expansion procedure and its impact on non-ambulatory patients with nonsense mutation Duchenne Muscular Dystrophy, we are pleased that patients on Translarna can remain on treatment after the loss of ambulation. We remain committed to work with the CHMP to clarify the open questions and are confident we will be able to demonstrate the pulmonary benefit of Translarna in non-ambulatory patients.”


The clinical data supporting the extension is based on results supporting the positive impact in the Force Vital Capacity (FVC) parameters for patients treated with Translarna in study 019, a long-term, open-label study, and study 025 (STRIDE Registry) when compared to matched natural history controls. This is in addition to currently approved labelling stating that the pharmacokinetics (PK) and safety profiles are comparable between ambulatory and non-ambulatory nmDMD patients and that no dose adjustment is necessary when patients become non-ambulatory. The CHMP opined that the comparable PK might not ensure efficacy in non-ambulatory patients since muscle mass is reduced in this patient group. PTC and members of the scientific community expressed disagreement with this understanding during the oral explanation.

Translarna received the annual renewal of its conditional marketing authorization in June 2019 for nonsense mutation Duchenne muscular dystrophy patients who are ambulatory and two years and over. In addition, in connection with the June 2019 renewal, PTC’s specific obligation for the submission of the results of Study 041, an ongoing clinical trial of ataluren, has been extended to September 2022.


Translarna™ (ataluren) in Canada

Translarna™ (ataluren) has not received marketing authorization in Canada. This treatment must, first and foremost, be evaluated and approved for the Canadian market by Health Canada. To approve a drug, Health Canada must ensure that it meets certain safety, efficiency and quality requirements.

La Force Foundation hopes that Translarna™(ataluren) will be marketed in Canada as soon as possible so that all young patients can benefit.

More about Translarna (ataluren)

Pioneer in the DMD therapy > Watch the Vlog

La Force & Max’s Big Ride in the parliament of Canada

A memorable day

Monday, June 10, was the day that closed the loop of the event of Max’s Big Ride to End Duchenne Muscular Dystrophy. La Force & Max’s Big Ride in the parliament of Canada.

Our two families are living with Duchenne muscular dystrophy. We share the same battle, that of access to treatment. Yesterday was a memorable day because the government has heard us. There is still a lot of work to be done, but now we know that the government is supporting us in our fight.

Andrew Sedmihradsky, Max’s father, invited Anakin and me to join him and his family for the 2019 Max’s Big Ride event. We shared our stories and raised awareness about DMD and access to treatment. Yesterday was a beautiful demonstration that we are stronger together.

Parliament Hill in Ottawa

Early Monday morning, we met the Sedmihradsky family on Parliament Hill. To begin the day, we were greeted by the Liberal MP, the Honorable Karina Gould. Subsequently, we had the pleasure of meeting the Parliamentary Secretary to the Minister of Health, Pam Damoff. We also met with the Minister of Health, the Honorable Ginette Petitpas Taylor. We had the chance to comply with the Right Honourable Prime Minister of Canada, Justin Trudeau. Raising awareness among key players in Quebec and across Canada is an essential element. We were sharing our knowledge of new treatments (currently unavailable in Canada), so that they may become available quickly to children and young adults with DMD.

See pictures on the Facebook page here

More about Max’s Big Ride

For the fifth year in a row, eight-year-old Max Sedmihradsky is taking his dad Andrew and three-year-old sister Isla on a 600 km cargo bike ride to help find a cure for Duchenne muscular dystrophy. Max’s Big Ride had kicked off in Hamilton on Monday, June 3 and had ended on Parliament Hill in Ottawa on Sunday, June 9.

Since 2015, Max’s Big Ride has raised over $170,000, met the Prime Minister of Canada twice and has helped to identify exciting new directions for Duchenne research, including the establishment of Max’s Big Fellowship with the Gunning Lab at the University of Toronto Mississauga. Learn more here: Watch a video about the Gunning Lab here.

The approval process in Canada

When you’re suffering from a fatal (orphan) disease for which there is no cure, such as Duchenne muscular dystrophy, each year, month, day, hour and second that separates you from a potential treatment is VITAL.

Nowadays, in an era when science and technology are advancing at the speed of light, new forms of treatment for Duchenne muscular dystrophy, about to be approved to represent real hope. However, these new forms of treatment must go through an approval process that takes between 2 and four years before becoming available to patients.

When rare diseases are involved, patients should be included in this process so that it occurs as quickly as possible and that all provinces across Canada reimburse the medication.

Here is a brief overview of the process


We are stronger together



Marie-Catherine Du Berger

An international study to learn about your preferences for DMD Treatments

La Force DMD is seeking your participation in a research survey for parents or guardians of sons with Duchenne muscular dystrophy and adult men (18+ years) with Duchenne. This is an international survey about your preferences and priorities for Duchenne treatments.

Regulatory agencies such as the EMA and FDA rely on the input of patients, families, and drug approval examples from other countries to inform treatment approval decisions. This preference survey will be the first study to measure preferences for Duchenne treatments internationally. The findings from this survey will be returned to participating patient advocacy organizations and may be used to inform policymakers about what you want out of treatments, and what risks you are willing to take.

The goal of the survey is to learn about your preferences and priorities for Duchenne treatments and to see if preferences vary internationally. Duchenne patient and advocacy organizations from Australia, Belgium, Canada, the Netherlands, the United Kingdom, and the United States have come together along with researchers at Johns Hopkins University to conduct this survey.

This survey is about your preferences and priorities for Duchenne muscular dystrophy treatments. This is the first study to measure Duchenne preferences internationally. DMD advocacy organizations globally have come together to launch this survey, and results will be returned to patient advocacy organizations and used to inform policymakers about what you want out of treatments, and what risks you are willing to take.

Survey info

  • Online
  • 45-60 minutes
  • No payment
  • Some questions might make you feel upset.
  • Treatments described include risks of severe side effects.
  • It is your choice to participate
  • You can stop the survey at any time
  • Results will be anonymous

Who can participate

  • Male adults (18 years+) with DMD
  • Parents of living sons with DMD
  • People currently living in Australia, Belgium, Canada, Netherlands, UK, or the US
  • Don’t match this description? Reach out, and we will accommodate

This research survey is online and will take 45-60 minutes. You will not be paid for completing the survey. It is possible that some of the questions in this survey might make you feel upset. The treatments that we describe include risks of serious side effects. It is your choice to complete the survey. You can stop the survey at any time. The results of the survey will be anonymous.

To participate, please email the survey@jessesjourney.com

If you have questions or concerns, please contact Norah Crossnohere at ncrossn1@jhu.edu

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