The FDA has approved cerliponase alfa (Brineura, BioMarin Pharmaceutical) to slow loss of walking ability in symptomatic pediatric patients aged 3 years and older with a specific form of Batten disease. Brineura is the first FDA-approved treatment for late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase-1 (TPP1) deficiency.
CLN2 disease is a rare, inherited disorder and a specific version of Batten disease. In the late infantile form, symptoms typically include language delay, recurrent seizures, and difficulty coordinating movements in children between the ages of 2 and 4. The disorder affects essential motor skills, and causes muscle twitches and vision loss.
The approval is based on data demonstrating the efficacy of Brineura in a non-randomized, single-arm dose escalation clinical trial. The study included 22 symptomatic pediatric patients with CLN2 disease compared to 42 untreated patients with CLN2 disease who were at least 3 years old and had motor or language symptoms. Patients treated with Brineura experienced fewer declines in walking ability compared to untreated patients.
Brineura’s safety was evaluated in 24 patients with the disorder aged 3 to 8 years old who received at least 1 dose of the drug in clinical studies. The treatment’s safety and efficacy has not been established in patients less than 3 years old.
Brineura is an enzyme replacement therapy that is administered into the cerebrospinal fluid by infusion. The recommended dose in pediatric patients aged 3 years an older is 300 mg administered once every other week by intraventricular infusion, followed by an infusion of electrolytes.
Common adverse effects include fever, ECG abnormalities such as slow heart rate, hypersensitivity, decrease or increase in CSF protein, vomiting, seizures, hematoma, headache, irritability, increased CSF white blood cell count, device-related infection, feeling jittery, and low blood pressure.
Additionally, Brineura should not be given to patients with signs of acute intraventricular access device-related complications or ventriculoperitoneal shunts.
FDA approves first treatment for a form of Batten disease [news release]. FDA’s website. . Accessed April 27, 2017.