An exciting new treatment option for PKU
BioMarin Pharmaceutical Inc. is pleased to announce the introduction of KUVANTM (sapropterin dihydrochloride) Tablets, the first and only FDA-approved prescription medication that reduces 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. KUVAN, a synthetic form of BH4, can activate the residual Phenylalanine hydroxylase enzyme to help improve Phe metabolism and reduce blood Phe levels in some patients. KUVAN was just approved by the US Food and Drug Administration.1
View a list of downloadable information about KUVAN for patients.
View a list of downloadable information about KUVAN for healthcare providers.
KUVAN Clinical Trials
The efficacy and safety of KUVAN was evaluated in four separate clinical studies in patients with PKU. In these studies, KUVAN reduced Phe levels in patients with mild, moderate, or severe PKU. KUVAN in doses of 5, 10, or 20 mg/kg/day was also well tolerated in over 579 patients for periods from 1 to 30 weeks of treatment.1
Dosing and Response
The recommended starting dose of KUVAN is 10 mg/kg/day taken once daily with food. Patients should be tested with 20 mg/kg /day before they are considered non-responders. Once response to KUVAN is established, the dosage may be adjusted within the range of 5 to 20 mg/kg/day according to response to therapy.1
It is not possible to predict who may benefit from treatment with KUVAN. KUVAN response is observed in some patients with PKU. Therefore, a trial of KUVAN is necessary to determine responsiveness to treatment.1
Patient and Clinic Reimbursement Support
The BioMarin Patient and Physician Support (BPPS) Program is designed to help patients gain coverage and access to KUVAN. It is a program intended to work with patients, clinics, and health insurance companies to easily and efficiently get patients started on KUVAN treatment. Case managers at BPPS will interface with insurance companies to remove the burden from your office staff and patients. For more information or to enroll a patient in BPPS, please call 1-877-MY-KUVAN (1-877-695-8826).
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.1
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.1
Not all patients with PKU respond to treatment with Kuvan. Response to treatment can only be determined by a therapeutic trial of Kuvan.1
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.1
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.1
- Reference: 1. KUVAN prescribing information, BioMarin Pharmaceutical Inc.
