FDA Approves New Treatment for Form of Batten Disease

The US Food and Drug Administration recently approved Brineura (cerliponase alfa) to treat the slow loss of walking ability in pediatric patients aged 3 years and older with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase-1 (TPP1) deficiency.

Brineura is an enzyme replacement therapy that includes the recombinant form of TTP1 (the active ingredient cerliponase alfa), which is the enzyme that is deficient in patients with CLN2 disease.

For pediatric patients 3 years of age and older, the recommended dose of Brineura is 300 mg administered once every other week by intraventricular infusion into the cerebrospinal fluid (CSF), followed by an infusion of electrolytes. The drug should be administered by health care professionals knowledgeable in intraventricular administration and must be administered under sterile conditions to reduce the risk of infections. The entire process lasts approximately 4.5 hours, and patients taking antihistamines with or without antipyretics or corticosteroids should receive appropriate treatment 30 to 60 minutes before Brineura is administered.

A non-randomized, single-arm dose escalation clinical study that included 22 pediatric patients with CLN2 disease and a historical control group of 42 patients found that patients treated with Brineura showed fewer declines in walking ability. In addition, the safety of Brineura was evaluated in 24 patients with CLN2 disease aged 3 to 8 years of age who received at least 1 dose of Brineura in clinical studies. The drug has not been established as safe or effective for children younger than 3 years of age.

The most common adverse events reported in patients associated with Brineura included fever, ECG abnormalities, hypersensitivity, decrease or increase in CSF protein, vomiting, seizures, hematoma, headache, irritability, pleocytosis, device-related infection, feeling jittery, and low blood pressure. Hypersensitivity reactions have been reported in patients treated with Brineura.

—Melissa Weiss

Reference:

FDA approves first treatment for a form of Batten disease [press release]. FDA. April 27, 2017. https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm555613.htm?source=govdelivery&utm_medium=email&utm_source=govdelivery.