Regulatory Decision Summary for Esperoct
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:
Contact:
Medicinal Ingredient(s):
Antihemophilic Factor VIII (Recombinant, B-Domain Truncated), PEGylated
Control Number:
268221
Therapeutic Area:
Antihemorrhagics
Type of Submission:
Supplement to a New Drug Submission
Decision issued:
Authorized; issued a Notice of Compliance in accordance with the Food and Drug Regulations
What was the purpose of this submission?
The purpose of this submission was to update the product monograph (PM) for Esperoct including safety and efficacy data based on the completed trial NN7088-3908 in previously untreated patients with hemophilia A.
Why was the decision issued?
NN7088-3908, was an open label single-arm multicenter non-controlled phase 3a trial investigating the safety and efficacy of Esperoct in prophylaxis and treatment of bleeding episodes in previously untreated pediatric patients (PUPs) with severe hemophilia A. The study enrolled 88 patients, of which 80 were included in the primary analysis.
The primary study endpoint evaluated the incidence of FVIII inhibitors. FVIII inhibitor development was observed in 26.3% of patients. This is consistent with the expected frequency of FVIII inhibitor development for recombinant FVIII products in PUPs of approximately 24-45%.
The most common adverse drug reactions (ADR) observed in PUPs were rash, erythema, injection site reaction, drug hypersensitivity, and factor VIII inhibition. Low incremental recovery (IR) was observed in 17 of the 59 patients without inhibitors. The majority of ADRs were mild to moderate and there were no fatal events. Overall, the safety profile of Esperoct remained consistent with what has been previously reported. Updates with respect to PUPs have been added to the Warnings and Precautions and Clinical Trial Adverse Reactions sections of the PM.
The key efficacy endpoint was the annualized bleeding rate (ABR; number of breakthrough bleeding episodes during prophylaxis). The median ABR for bleeds in patients on prophylaxis throughout the study was 1.35 bleeds/patient/year. This is consistent with what has been reported for previously treated patients. The efficacy results were considered supportive, but due to limitations with the data (e.g., study not adequately designed to assess efficacy), the sponsor agreed not to update the efficacy results in the product monograph.
Overall, the benefit-risk of Esperoct remains positive with the additional clinical data related to PUPs.
For further details about Esperoct, please refer to the Product Monograph, approved by Health Canada and available through the Drug Product Database.
Date of Decision:
2023-09-11
Manufacturer / Sponsor:
Drug Identification Number(s) Issued:
N/A
Prescription status:
Schedule D drug
Date Filed:
2022-09-27
Related Drug Products
| Product name | DIN | Company name | Active ingredient(s) & strength |
|---|---|---|---|
| ESPEROCT | 02490064 | NOVO NORDISK CANADA INC | ANTIHEMOPHILIC FACTOR VIII (RECOMBINANT, B-DOMAIN TRUNCATED), PEGYLATED 500 UNIT / VIAL |
| ESPEROCT | 02490080 | NOVO NORDISK CANADA INC | ANTIHEMOPHILIC FACTOR VIII (RECOMBINANT, B-DOMAIN TRUNCATED), PEGYLATED 1500 UNIT / VIAL |
| ESPEROCT | 02490099 | NOVO NORDISK CANADA INC | ANTIHEMOPHILIC FACTOR VIII (RECOMBINANT, B-DOMAIN TRUNCATED), PEGYLATED 2000 UNIT / VIAL |
| ESPEROCT | 02490102 | NOVO NORDISK CANADA INC | ANTIHEMOPHILIC FACTOR VIII (RECOMBINANT, B-DOMAIN TRUNCATED), PEGYLATED 3000 UNIT / VIAL |
| ESPEROCT | 02490072 | NOVO NORDISK CANADA INC | ANTIHEMOPHILIC FACTOR VIII (RECOMBINANT, B-DOMAIN TRUNCATED), PEGYLATED 1000 UNIT / VIAL |