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:

Drug

Medicinal ingredient(s):

Nitisinone

Therapeutic area:

Other Alimentary Tract and Metabolism Products

Type of submission:

Priority New Drug Submission (New Active Substance)

Control number:

193226
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