Product Details

Kuvan

Sapropterin Dihydrochloride
100 mg
Tablet


DIN/PIN/NPN

02350580

Manufacturer

BioMarin International Ltd.

Formulary Listing Date

2013-02-20  

Unit Price

33.0000

Amount MOH Pays

33.0000

Coverage Status

Exceptional Access Program Product

ODB Formulary Therapeutic Classification

Therapeutic Note

NO

ATC Code

A16AX07

Interchangeable Products

NO  

LU Clinical Criteria

NO  

EAP Criteria

Therapeutic Class Reimbursement Criteria
Metabolic/Genetic Modifiers

Sapropterin

  • Brand(s): Kuvan
  • Dosage Form/Strength: 100 mg tablet
  • Updated December 2, 2020

Ongoing funding of sapropterin (Kuvan) will be considered through the EAP for non- pregnant patients and patients actively planning pregnancy who have a diagnosis of Phenylketonuria (PKU) and who have demonstrated a response to the initial 6-month trial of sapropterin [generally reimbursed through the Biomarin, the manufacturer of Kuvan]. 

Initial Criteria for the Trial Period (First 6 months):

For the management of patients with the diagnosis of hyperphenylalaninemia (HPA) due to tetrahydrobiopterin (BH4)-responsive phenylketonuria (PKU) who meet ALL of the following criteria: 

  1. Diagnosis of PKU confirmed through an approved test. 

  1. Compliance with low protein diet and formulas. 

  1. Documented baseline blood phenylalanine (Phe) levels, which have been measured at least twice during a 3-to-6-month period, are greater than 360 μmol/L despite compliance with a low protein diet; 

  1. Baseline protein intake assessment by a dietitian. Ability to comply with their medication regimen 

  1. Managed by a prescriber specialized in metabolic/ biochemical diseases. 

  1. the Patient’s blood Phe tolerance levels will be documented at months 1 to 2 and 4 to 6 during the Trial Period. 

Modified Criteria for Pregnant Patients during the 6-month trial period: 

  1. Patient has a diagnosis of PKU confirmed through an approved test 

  1. Patient’s treatment is being managed by a prescriber specialized in metabolic/biochemical diseases; and 

  1. Patient’s baseline blood Phe level is greater than 360 μmol/L despite compliance with all recommendations for dietary intervention and monitoring or compliance with a low protein diet. 

In the case of Patients who are eligible for but do not utilize the Patient support program for a 185-day or 6-month trial. Executive Officer will approve a request to reimburse claims for Kuvan at a dosage of up to 20mg/kg per day for the trial period of up to 6 months provided that the above conditions are met.

Approval Duration: 6 months

Funding Criteria for Kuvan in the Post Trial Period: Patients must have demonstrated the response as per the trial criteria to be funded following the 6-month trial period. 

Initial Criteria Post Trial: 

For the management of patients with the diagnosis of hyperphenylalaninemia (HPA) due to tetrahydrobiopterin (BH4)-responsive phenylketonuria (PKU) who meet ALL of the following criteria: 

  1. Compliance with low protein diet, formulas, and Kuvan; AND 

  1. During the 6-month trial period under EAP or PSP patient has achieved one or more of the following:
    i)     a demonstrated response to the Kuvan responsiveness test or PKU clinical protocol, based on the following information:
    ii) the clinic’s definition for response; AND
    iii) all relevant laboratory results used to determine that the Patient was a responder to Kuvan

  1. Patient meets one of the following:
    i)     normal sustained Blood Phe levels [ > 120 μmol/L and below 360 μmol/L] (At least 2 levels measured at least 1 month apart);
    OR
    ii)     sustained blood Phe reduction of at least 30% (At least 2 levels measured at least 1 month apart) compared to baseline if the Phe baseline level is below 1200 μmol/L;
    OR
    iii)     sustained blood Phe reduction of at least 50% (At least 2 levels measured at least 1 month apart) compared to baseline if the Phe baseline level is > 1200 μmol/L; 

  1. Demonstrated an increase in dietary protein tolerance based on targets set between the clinician and patient 

  1. Managed by a prescriber specialized in metabolic/biochemical diseases.

Dosage: Up to a maximum of 20 mg/kg per day 

Approval Duration: 1 year

Renewal Criteria: 

Renewals will be considered for patients meeting the following criteria: 

  • Demonstrates ongoing response to treatment; AND 

  • Complies with and will continue to comply with a low protein diet, formulas, and treatment with Kuvan; AND 

  • the request for extended coverage includes a recent follow-up from a prescriber specialized in metabolic/biochemical diseases. 

Exclusion Criteria for both Initial (Trial and Post Trial period) and Renewal criteria:
(Patients meeting any of the following criteria will not be funded) 

  • the Patient is not on a low protein diet or is not compliant with their low protein diet; or 

  • the Patient has a baseline blood PHe level less than 360 μmol/L prior to initiating therapy with Kuvan. 

Dosage: Up to a maximum of 20 mg/kg per day

Approval Duration: 1 year

EAP Drug Request Form:

Standard Form for EAP Drug Requests

Product Monograph

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