| Summary: | Sapropterin (SAP) (Kuvan) is indicated in conjunction with a phenylalanine (Phe)-restricted diet to reduce blood Phe levels in patients with hyperphenylalaninemia (HPA) due to tetrahydrobiopterin (BH4)-responsive phenylketonuria (PKU). The initial dosage is 20 mg/kg/day administered orally for a period of up to 1 month. Once responsiveness to sapropterin (SAP) has been established, the dosage may be adjusted within the range of 5 to 20 mg/kg/day according to response to therapy. The confidential price per 100 mg tablet is $33.00. Based on representative body weight values obtained from trials of SAP, and depending upon dosage, annual costs for an 11 kg patient were estimated at $12,000 to $36,000; for a 29 kg patient, $24,000 to $72,000; and for a 68 kg patient, $48,000 to $169,000. SAP was originally submitted to the CADTH Common Drug Review (CDR) in 2010, and in January 2011, the Canadian Expert Drug Advisory Committee (CEDAC) issued a Final Recommendation that Kuvan not be listed. The key reason for the recommendation was that patient details were insufficient to identify a subpopulation for whom SAP may provide a significant clinical benefit that is cost-effective. A Request for Advice regarding SAP was submitted to CDR by CDR-participating drug plans in October 2011, which did not result in any changes to the recommendation. The basis for the current resubmission is the availability of new clinical evidence. Following the 2011 CEDAC recommendation, provincial reimbursement of Kuvan has occurred in Ontario (as of February 2013) and Saskatchewan (as of September 2013). The manufacturer states that reimbursement criteria in these provinces were developed with the understanding that new data would be forthcoming about the effectiveness and appropriate use of SAP to treat patients with PKU. The submitted price for SAP is the same as in the 2010 submission; however, a revised cost-effectiveness model was provided as part of the resubmission
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