Exondys 51 (eteplirsen)

This treatment uses a specific exon-skipping technique to jump over a portion of genetic machinery that produces a non-working, mutated form of dystrophin in children with DMD. It aims to restore the machinery’s ability to read genetic code, so it can create a less mutated form of dystrophin that works in children with DMD.

The production of partly functional dystrophin may delay muscle destruction and extend mobility in children with this devastating, rare disease. More specifically, Exondys 51 (eteplirsen) triggers the skipping of exon 51, which occurs in 13% of children with DMD.

This drug is not currently available in Canada, as Health Canada must approve its use in the Canadian market.

For more information on the exon-skipping technique in the treatment of DMD as well as a glimpse into the U.S. approval process for Exondys 51 (eteplirsen), please consult the following links to the manufacturer and other websites:


2015

  • Completion of the rolling submission of a New Drug Application (NDA) to the United States Food and Drug Administration (FDA) for eteplirsen on June 26, 2015. Press release
  • Sarepta Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) has filed the New Drug Application (NDA) for eteplirsen for the treatment of Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. Press release
  • FDA has granted Rare Pediatric Disease Designation for eteplirsen. Press release

2016

  • European Medicines Agency (EMA) validated the previously submitted Marketing Authorization application (MAA) for eteplirsen to treat Duchenne muscular dystrophy amenable to exon 51 skipping. Sarepta is seeking conditional approval of eteplirsen in the EU through the centralized procedure. Validation of the MAA confirms that the submission is accepted and starts the formal review process by the EMA’s Committee for Human Medicinal Products (CHMP). The standard review period is 210 days (plus additional time for the applicant to respond to questions from the agency). Press release
  • Sarepta Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted accelerated approval for EXONDYS 51™ (eteplirsen). Press release, Read more here
  • At the end of 2016, Sarepta Therapeutics announced that the European Medicines Agency (EMA) validated the previously submitted Marketing Authorization Application (MAA) for eteplirsen to treat Duchenne muscular dystrophy amenable to exon 51 skipping. Sarepta is seeking conditional approval of eteplirsen in the EU through the centralized procedure. Validation of the MAA confirms that the submission is accepted and starts the formal review process by the EMA’s Committee for Human Medicinal Products (CHMP). The standard review period is 210 days (plus additional time for the applicant to respond to questions from the agency). Info from Business Wire see complete article here.
  • The FDA has just announced an unprecedented decision… The children with Duchenne muscular dystrophy were heard because it’s “yes” to Eteplirsen! Read more here.

2017

  • Sarepta Therapeutics, Inc., and Clinigen Group plc’s Idis Managed Access division have initiated a Managed Access Program (MAP) for eteplirsen in certain geographies to treat eligible Duchenne muscular dystrophy (DMD) patients amenable to exon 51 skipping.  Read more here.

2018

  • The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA), has adopted a negative opinion for EXONDYS® (eteplirsen), as expected. Press release, Read more here
  • European Regulators Reject Sarepta’s Exondys 51 for DMD. Read more on Bio Space.


Translarna™ (ataluren)

This treatment is the first to target an underlying genetic cause of DMD: a nonsense mutation (nmDMD). It aims to slow the progression of DMD by restoring the function of a specific protein, dystrophin. In DMD, a nonsense mutation corrupts the genetic machinery that builds a working version of this protein, which is essential for proper neuromuscular function.

In clinical trials, ataluren has shown some benefit in slowing the loss of motor skills in some children with nmDMD. Some boys treated with ataluren could more easily undertake a range of physical activities, including walking, climbing and descending stairs and other motor functions.


2014

  • The European Medicines Agency granted Translarna™ (ataluren) conditional marketing authorization to treat nmDMD in children of 5 years of age or more and still able to walk. Translarna™ (ataluren) is also approved for use in South Korea and Israel.

2015

  • The European Medicines Agency (EMA) has set up a “post-AMM” study by the PTC Therapeutics laboratory, which sells Translarna™ (ataluren). This study, still in progress, is today called the STRIDE register. Its goal is to evaluate the safety and efficacy of ataluren over five years as part of the usual neuromuscular consultation. At the end of 2016, Translarna™ (ataluren) received marketing authorization by the European Medicines Agency and is available in Europe and regions that reference that authorization. AFM Théléthon, Press Release

2016

  • NICE Recommends Translarna™ (ataluren) for the Treatment of Patients with Nonsense Mutation Duchenne Muscular Dystrophy in England. Press release
  • PTC Therapeutics, Inc. announced that it has received a Refuse to File letter from the U.S. Food and Drug Administration (FDA) with regard to its New Drug Application (NDA) for ataluren (trade name, Translarna), an oral, first-in-class, protein restoration therapy for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD). FDA Rejects PTC’s New Drug Application for Translarna as a Nonsense Mutation DMD Treatment
  • NHS England Enables Access to Translarna™ (ataluren) For Patients with Nonsense Mutation Duchenne Muscular Dystrophy. Press release
  • CHMP Recommends Renewal of Translarna’s™ Marketing Authorization for Nonsense Mutation Duchenne Muscular Dystrophy Based on Continued Positive Benefit-Risk Assessment. Press release
  • PTC Therapeutics, Inc. announced that the company intends to submit the results of the recently completed Phase 3 ACT DMD study for review by Health Canada as part of the New Drug Submission (NDS) for Translarna™ (ataluren) to treat nonsense mutation Duchenne muscular dystrophy (nmDMD). PR Newswire
  • No decision was issued by Health Canada. The company cancelled its submission before a final decision was issued. Regulatory Decision Summary – ataluren (*Translarna) – Health Canada

2017


2018

  • CHMP Adopts Positive Opinion for the Expansion of the Translarna™ (ataluren) Label to Include Patients as Young as 2 Years of Age. Read more here.
  • Health Canada > Drug and health product submissions under review (SUR). Health Canada
  • Analysis of data to date shows Translarna Preserved Ambulation for up to 5 Years Compared to Natural History. Read more here.

2019

  • Translarna™ (ataluren) is the first therapy approved in Brazil for DMD. Read more here.
  • 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. Besides, 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 Translarna™ (ataluren), has been extended to September 2022.


Translarna™ (ataluren) in Canada

En 2016, PTC Therapeutics fait le point sur l’examen par Santé Canada de Translarna™ (ataluren) pour le traitement de la dystrophie musculaire de Duchenne.

  • No decision was issued by Health Canada. The company cancelled its submission before a final decision was issued. Regulatory Decision Summary.

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. PR Newswire

In Septembre 2018 the new drug submission has been accepted. Ataluren is currently on the List of Drug and health product submissions under review (SUR) at Health Canada. Drug and health product submissions under review (SUR)

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

  • For specific updates about ataluren, you can consult and sign in on Duchenne and you