Regulatory Decision Summary for Emapalumab (*Gamifant)
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: Emapalumab (*Gamifant)
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
January 29, 2021
What was the purpose of this submission?
The purpose of this New Drug Submission was to seek market authorization for Gamifant (emapalumab), an anti-interferon gamma monoclonal antibody, for the treatment of pediatric and adult patients with primary hemophagocytic lymphohistiocytosis (pHLH) who are refractory or intolerant to, or have relapsed after conventional treatment.
What did the company submit to support its submission?
The sponsor submitted quality, non-clinical, clinical and labelling components.
The pivotal evidence for safety and efficacy were provided in the form of a phase 2/3, single-arm, open-label trial of Gamifant, administered on a background of dexamethasone, in pediatric patients with pHLH who had received prior treatments. The study enrolled 27 treatment-experienced patients and 7 treatment-naïve patients. Patients from this study could enroll in a long-term follow-up study, which was also submitted. Limited treatment experience within the context of a compassionate use program was also included in 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, Health Canada had completed the assessment of the non-clinical data package and the review of the pivotal evidence supporting the safety and efficacy of Gamifant among treatment-experienced patients with pHLH. The review of clinical pharmacology information was on-going. Health Canada had identified deficiencies in the clinical data that would have precluded issuing an approval. The sponsor chose to cancel the submission and may 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 accessing Gamifant through SAP or in clinical trials.
Requests for special access to Gamifant 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:
http://www.healthcanada.gc.ca/sap.
Additional information
*Proposed Brand Name:
Gamifant