Psychological issues may occur in PKU patients who do not maintain low Phe levels

  • Adults with PKU who were given a Phe-restricted diet exhibited more psychiatric disorders than controls (25.7% vs 16.1%, respectively)14

*Adapted from a study in which 35 PKU patients (mean age 22.2 years) were compared to a control sample of 181 subjects aged 18 years in a separate epidemiological study, based on standardized and highly structured 2-hour, face-to-face interviews with child psychologists and clinical psychiatrists.14 †P<0.05 for PKU patients compared to controls.14

  • PKU patients, despite being given diet to follow, had significantly more stimulant use (26%) for attentional symptoms compared to patients in the control group (6.5%)15
    • Higher mean blood Phe levels (792 μmol/L) were associated with greater stimulant use compared to patients with lower blood Phe (486 μmol/L) (P<0.02)

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.

Top

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.

Please read the full Prescribing Information by clicking here.

BIOMARIN