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Canadian Drug Review Reimbursement Decision

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Generic sapropterin dihydrochloride
Company BioMarin Pharmaceutical (Canada) Inc.
Indication PKU
Condition Endocrine, nutritional and metabolic diseases
Submission Type Initial
Patient Population Sapropterin is a synthetic form of tetrahydrobiopterin (BH4), the cofactor for Phe hydroxylase. It is approved by Health Canada in conjunction with a Phe-restricted diet to reduce blood Phe levels in patients with hyperphenylalaninemia due to BH4-responsive PKU. The recommended starting dose is 10 mg/kg per day. Once responsiveness has been established, the dosage may be adjusted within the range of 5 mg/kg to 20 mg/kg daily, based on response to therapy. It is available as 100 mg tablets.
Status Complete
Date of Recommendation 2011-01-26
Recommendation Summary Do not list
Recommendation Details The Canadian Expert Drug Advisory Committee (CEDAC) recommends that sapropterin not be listed.
Reason for Recommendation 1. Patient details were insufficient to identify a subpopulation for whom sapropterin may provide a significant clinical benefit that is cost-effective. Of Note: Although the Committee found sufficient evidence that sapropterin lowers blood phenylalanine (Phe) levels in certain patients with phenylketonuria (PKU), the submission did not provide sufficient details of how to identify the patients who would benefit in a cost-effective manner. The proposed Kuvan Starter Program is suitable only to screen patients to identify “responders,” but such a response in the clinical trials did not differentiate low response from clinically important response. Starting and stopping rules, beyond the screening stage, that are linked to substantive benefit are needed. It would be advisable for the manufacturer to work with provinces to establish these requirements with respect to age, PKU classification, and specific benefits to be achieved that support the price of this product.
Clinical Report:
Pharmacoeconomic Report:
Final Recommendation Report: CDR clinical report  CADTH-CDR Final Recommendation report

†The information referenced on this page is compiled from publicly available documents published by CADTH and is available through the embedded links.

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