Brineura (cerliponase alfa), by BioMarin, is now approved in Europe to treat neuronal ceroid lipofuscinosis type 2 (CLN2), one of a group of disorders known as Batten disease. The therapy covers all ages from birth.
The marketing authorization granted by the European Commission (EC) follows a positive review by the Committee for Medicinal Products for Human Use (CHMP) — part of the European Medicines Agency (EMA) recommending the treatment’s approval. Brineura was granted accelerated review.
“Today represents several important milestones for the medical and patient communities, and also for me, both professionally and personally. For the first time since entering this field nearly 15 years ago, I can now tell families affected by CLN2 disease that there is a meaningful treatment that may help their child, and provide hope,” Angela Schulz, a doctor at Children’s Hospital at University Medical Center Hamburg-Eppendorf, in Germany, and a principal investigator of the Brineura studies, said in a press release. “We are committed to furthering the study of Brineura and believe that the medical knowledge gained from the Brineura studies can help us learn more about treating other neurodegenerative diseases.”
CLN2 type Batten disease affects the nervous system and typically cause worsening problems with vision, movement and thinking ability. The disorder is inherited, with symptoms typically beginning between ages 2 and 4 and including recurrent seizures (epilepsy) and difficulty coordinating movements (ataxia). Affected children also develop muscle twitches (myoclonus) and vision loss, and may lose the ability to talk and walk by age 6.
Brineura is the first enzyme replacement therapy directly administered into brain fluid through a catheter. It treats the main cause of CLN2 disease by helping to replace the deficient TPP1 (tripeptidyl peptidase 1) enzyme missing in these children.
Brineura’s approval was based on the results of an open-label, dose-escalation study (NCT01907087) conducted in 24 patients with CLN2 disease, ages 3 to 8, and results from an ongoing extension study (NCT02485899). These clinical trials looked at the safety and tolerability of Brineura administered in patients’ brains and assessed the therapy’s effectiveness as measured by a CLN2 disease-specific rating scale score, in comparison with natural history data, after 48 and 72 weeks of treatment.
Data at 48 weeks showed that 87 percent, or 20 of the 23 children who concluded the dose-escalation study, continued to have motor-language scores showing substantially slower disease progression compared to 42 patients in the natural history group.
In the ongoing study, the rate of decline in Brineura-treated patients continues to demonstrate durability.
Brineura was approved by the U.S. Food and Drug Administration (FDA) in April to slow the loss of walking ability in symptomatic pediatric patients age 3 and older with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as TPP1 deficiency.
“Thank you to the European Commission for recognizing the medical benefits of Brineura for children affected by CLN2 disease. We are also grateful to the families and study investigators and their teams, who dedicated their time to the clinical program to make this treatment a reality for affected children in the EU,” said Jean-Jacques Bienaimé, chairman and CEO of BioMarin. “The physicians and patient families that form the Batten disease community have been integral to the approval of this treatment. We are also working to reduce the diagnostic journey for patients, who are often not diagnosed until the age of 5, nearly two years after their first seizure. With a treatment now available, accelerating early diagnosis will be critical.”