Regulatory Decision Summary for Ataluren (*Translarna)
Review decision
The Regulatory Decision Summary explains Health Canada’s decision for the product seeking market authorization. The Regulatory Decision Summary includes the purpose of the submission and the reason for the decision.
Product type:
Summary of Cancellation: ataluren (*Translarna)
Medicinal ingredient(s):
Therapeutic area:
Type of submission:
Control number:
Overview
Decision issued
No decision was issued by Health Canada. The company cancelled its submission before a final decision was issued.
Date of cancellation
March 17, 2016
What was the purpose of this submission?
This New Drug Submission (NDS) was filed to seek approval under the Notice of Compliance with Conditions (NOC/c) Guidance for use of ataluren for the treatment of patients with Duchenne Muscular Dystrophy (DMD) resulting from a nonsense mutation in the dystrophin gene, who are ambulatory and 5 years of age and older.
What did the company submit to supports its submission?
The sponsor submitted drug quality, pre-clinical and clinical components. The pivotal clinical safety and efficacy information consisted of the following: a Phase 2a (Study 004) and a Phase 2b trial (Study 007).
Study 004, a 28 week study of 3 doses of ataluren in 38 patients, primarily assessed the effect of the drug on dystrophin expression, determined from patient muscle biopsies. Study 007, a 48 week trial which enrolled 174 patients, compared 2 doses of ataluren to placebo, with the primary efficacy endpoint being slowing of the rate of decline in ambulation in the patients, as measured by the 6 Minute Walk Distance (6MWD).
Phase 3 trial data was not part of the submission.
What was the status of the submission when it was cancelled? What was Health Canadas assessment of the submission at the time of cancellation?
At the time of the cancellation, the review of the submission was nearing completion. Health Canada had identified some deficiencies in the data that would have precluded issuing an approval. The sponsor chose to cancel their submission in order to re-file at a later date with additional data.
What consequences does the cancellation have for patients accessing the drug under the Special Access Programme (SAP), or via clinical trials?
There is no expected impact for patients using SAP or in clinical trials.
Requests for special access to ataluren will continue to be considered on a case-by-case basis. For more information about the Special Access Programme refer to the programmes web site: www.healthcanada.gc.ca/sap or www.santecanada.gc.ca/pas
There are two approved clinical trials underway in Canada for the use of ataluren in the treatment of nonsense mutation dystrophinopathy. No impact is expected for patients participating in these studies.
Additional information
*Proposed Brand Name:
Translarna