Regulatory Decision Summary for Orfadin
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:
Medicinal ingredient(s):
Therapeutic area:
Type of submission:
Control number:
What was the purpose of this submission?
This New Drug Submission was filed to obtain market authorization for Orfadin for the treatment of patients with hereditary tyrosinemia type 1 (HT-1), in combination with dietary restriction of tyrosine and phenylalanine.
Why was the decision issued?
This submission was based on demonstration of the efficacy and safety of nitisinone [(2-(2-nitro-4-trifluoromethylbenzoyl)-1,3-cyclohexanedione; NTBC] in the NTBC Study, supported by the published results of the Québec NTBC study and post-market safety data.
Uncertainties included the effects of advanced age, chronic treatment, renal impairment, hepatic impairment, race, and genetic polymorphisms on the pharmacokinetics of nitisinone, and safety when used during pregnancy and lactation.
A Risk Management Plan was reviewed by the Marketed Health Products Directorate. Risk minimization measures include ongoing post-marketing surveillance, commitment to a Canadian post-approval safety study, and adequate labelling of all identified safety issues, including the lack of data in some subpopulations, the potential risk from use during pregnancy and lactation, and the potential risk to the fetus and mother from discontinuing nitisinone during pregnancy.
The evidence of the benefit provided by nitisinone for the treatment of hereditary tyrosinemia type 1 outweighs the associated risks.
Decision issued
Approved; issued Notice of Compliance in accordance with the Food and Drug Regulations
Related Drug Products
| Product name | DIN | Company name | Active ingredient(s) & strength |
|---|---|---|---|
| ORFADIN | 02459736 | SWEDISH ORPHAN BIOVITRUM AB (PUBL) | NITISINONE 20 MG |
| ORFADIN | 02459698 | SWEDISH ORPHAN BIOVITRUM AB (PUBL) | NITISINONE 2 MG |
| ORFADIN | 02459701 | SWEDISH ORPHAN BIOVITRUM AB (PUBL) | NITISINONE 5 MG |
| ORFADIN | 02459728 | SWEDISH ORPHAN BIOVITRUM AB (PUBL) | NITISINONE 10 MG |