Strict adherence to a Phe-restricted diet can prevent the consequences of elevated Phe, but many patients don’t maintain target levels

  • The US Collaborative Study proved that a Phe-restricted diet, if adhered to consistently, is effective in helping to prevent mental retardation and other consequences associated with PKU11
  • Dietary compliance becomes more difficult with age, however, and the percentage of managed patients with Phe levels above target range is high6

*Adapted from a 7-year, 4-center, retrospective study evaluating Phe levels in 330 diet-managed PKU patients <20 years old. Target range varied by center from 350–400 μmol/L for children <5 years of age, 400–700 μmol/L for children 5–10 years of age, and 450–700 μmol/L for children >10–19 years of age.6

Even patients who claim to follow their diet may still have elevated Phe

*Adapted from a prospective study evaluating bone mineral density of 31 adult PKU patients (mean age 25±5.3 years), including 17 diet-adherent and 14 nonadherent patients by self-report7

  • In a recent study, more than half of adult patients with classic PKU who claimed to be compliant with diet did not achieve blood Phe target level7
    • 53% (9/17 patients) were above the target level of 726 μmol/L

Long-term studies of neurocognitive outcomes with KUVAN treatment have not been conducted. BioMarin is implementing a trial to evaluate long-term neurocognitive outcomes in PKU patients treated with KUVAN.

Indication

KUVAN® (sapropterin dihydrochloride) Tablets is indicated to reduce blood phenylalanine (Phe) levels in patients with hyperphenylalaninemia (HPA) due to tetrahydrobiopterin- (BH4-) responsive Phenylketonuria (PKU). KUVAN is to be used in conjunction with a Phe-restricted diet.

Important Safety Information

Prolonged exposure to elevated blood Phe levels in PKU patients can result in severe neurologic damage. The initiation of KUVAN therapy does not eliminate the need for careful monitoring of blood Phe levels and ongoing dietary management.

Some patients receiving KUVAN can experience significant drops in blood Phe levels. Patients should be monitored closely to ensure that blood Phe levels do not fall too low.

Not all patients with PKU respond to treatment with KUVAN. Response to treatment can only be determined by a therapeutic trial of KUVAN.

KUVAN has not been studied in patients with liver or renal impairment. Patients who have these conditions should be carefully monitored when receiving KUVAN. Caution should be used with the administration of KUVAN to patients who are receiving levodopa and drugs that affect nitric oxide–mediated vasorelaxation or folate metabolism.

The most serious adverse reactions reported during KUVAN administration (regardless of relationship to treatment) were gastritis, spinal cord injury, streptococcal infection, testicular carcinoma, and urinary tract infection. Mild to moderate neutropenia was also noted. The most common adverse reactions were headache, diarrhea, abdominal pain, upper respiratory tract infection, pharyngolaryngeal pain, vomiting, and nausea.

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