DrugSapropterin dihydrochloride (Kuvan)
IndicationIn conjunction with a phenylalanine (Phe)-restricted diet to reduce blood Phe levels in patients with hyperphenylalaninemia (HPA) due to tetrahydrobiopterin-(BH4)-responsive phenylketonuria (PKU).
Listing requestOngoing funding of sapropterin (Kuvan) for non-pregnant patients and patients actively planning pregnancy who have a diagnosis of PKU and who have demonstrated a response to the initial 6 month trial of sapropterin and who meet ALL of the following criteria:
  1. Compliance with low protein diet, formulas, and treatment with sapropterin; AND
  2. Has achieved
    a)

    normal sustained blood Phe levels [Greater than 120 μmol/L and less than 360 μmol/L] (At least 2 levels measured at least 1 month apart); OR

    b)

    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 less than 1200 μmol/L; OR

    c)

    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 greater than 1200 μmol/L; AND

  3. Demonstrated increase of dietary protein tolerance based on targets set between the clinician and patient; OR
  4. Clinically meaningful age-appropriate improvement in:
    a)

    neurobehavioural or neurocognitive function or impairment for patients with such impairments as determined by peer reviewed clinically validated scales; OR

    b)

    demonstrated improvement in Quality of Life using peer reviewed validated scales; AND

  5. Managed by a physician specialized in metabolic/biochemical diseases.
Dosage form(s)100 mg oral tablets
NOC dateApril 30, 2010
ManufacturerBioMarin Pharmaceutical Inc.

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Cover of Clinical Review Report: Sapropterin dihydrochloride (Kuvan)
Clinical Review Report: Sapropterin dihydrochloride (Kuvan) [Internet].
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