Brineura Approved for Late Infantile Neuronal Ceroid Lipofuscinosis Type 2
April 27, 2017 - The U.S. FDA approved Brineura™ (cerliponase alfa), manufactured by BioMarin Pharmaceuticals. It is an enzyme replacement therapy for children at least three years old who have a rare form of Batten disease known as late infantile neuronal ceroid lipofuscinosis type 2 (CLN2). It helps affected children maintain the ability to crawl or walk. Brineura is infused under sterile conditions directly into cerebrospinal fluid of the brain using an implanted reservoir and catheter. Dosage will be 150mg/5mL (30mg/mL) solution, two single-dose vials per carton co-packaged with Intraventricular Electrolytes Injection 5mL in a single-dose vial for intraventricular infusion. The recommended dose is 300mg administered over four and one-half hours every two weeks. BioMarin Pharmaceuticals plans to begin sales in early June 2017. Distribution will be through hospitals only.
Batten disease includes a collection of hereditary neurological disorders that affect only two to four babies in every 100,000 live births. Patients with CLN2 lack the enzyme tripeptidyl peptidase-1 (TPP1), which causes developmental delays, muscle spasms, seizures, vision loss and other neurological symptoms. Only about 20 infants per year are born with CLN2 in the United States. Symptoms typically begin between the ages of two and four years, patients gradually lose function, including speech and most cases are fatal before the age of 12 years. Brineura is the first drug FDA approved to treat CLN2. Continuing studies will test its use for children under two years of age and its long-term effects over 10 years. Before Brineura is given, patients should be pre-treated with an antihistamine, a fever reducer and/or a corticosteroid. After administration, electrolyte infusions are recommended.
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