The Food and Drug Administration (FDA) has approved Brineura (cerliponase alfa; Biomarin) as a treatment to slow loss of ability to walk or crawl (ambulation) in symptomatic pediatric patients 3 years of age and older with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase 1 (TPP1) deficiency. This is the first treatment approved to treat pediatric patients with CLN2 disease, a form of Batten disease.

Brineura, an enzyme replacement therapy, is a recombinant form of human TPP1, the enzyme deficient in patients with CLN2 disease. It is administered into the cerebrospinal fluid (CSF) by intraventricular injection; an intraventricular access device or port must be placed at least 5–7 days prior to the first infusion. Infusion of Brineura lasts approximately 4.5 hours; this includes the required infusion of intraventricular electrolytes. Pre-treatment with antihistamines with or without antipyretics or corticosteroids is recommended 30–60 minutes prior to initiating the infusion. Due to the potential for anaphylaxis, appropriate medical support should be readily available. 

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The approval of Brineura was based on data from a 96-week, non-randomized, single-arm dose escalation clinical trial. The study included 22 symptomatic patients with CLN2 and compared them to 42 untreated patients with CLN2 disease from a natural history cohort who were at least 3 years old and had motor or language symptoms. Results from the analysis showed the odds of Brineura-treated patients not having a decline were 13 times the odds of natural history cohort patients not having a decline (odds ratio 13.1, 95% CI: 1.2, 146.9).

The most common adverse events reported during infusion included fever, ECG abnormalities including bradycardia, hypersensitivity, decrease or increase in CSF protein, vomiting, seizures, hematoma, headache, irritability, pleocytosis, device-related infection, jittery feeling, and low blood pressure.

Brineura is expected to be available by early June as a 150mg/5mL solution in cartons containing 2 single-dose vials co-packaged with intraventricular electrolytes injection.

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