BioMarin Pharmaceutical has announced that European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has granted a positive opinion for Marketing Authorization Application (MAA) of Brineura (cerliponase alfa).

Brineura is intended to treat children affected with Neuronal Ceroid Lipofuscinosis Type 2 (CLN2), a form of Batten disease commonly known as tripeptidyl peptidase 1 (TPP1) deficiency.

Brineura is a recombinant form of human TPP1, which is administered as an enzyme replacement therapy designed to restore TPP1 enzyme activity and break down the storage materials that cause CLN2 disease.

The treatment is delivered directly into the fluid surrounding the brain (cerebrospinal fluid) using BioMarin’s patented technology in order to reach the cells of the brain and central nervous system.

The CHMP’s recommendation is now referred to the European Commission (EC), which is expected to provide its decision by the second quarter of this year.

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Following the approval of EC, BioMarin will receive marketing authorisation for Brineura in all 28 countries of the European Union along with Iceland, Norway and Liechtenstein.

CHMP delivered its positive opinion based on an accelerated review procedure.

BioMarin worldwide research and development managing director and president Hank Fuchs said: “A little less than four years ago, the first child was treated in a clinical trial, and today marks another important step forward in providing the first treatment option for children affected by CLN2 disease, a rapidly progressing and fatal pediatric brain disorder.

“We thank the CHMP and the CLN2 community for their continued support, including the children and families who gave their time to participate in the clinical trials with the goal of making treatment a reality for patients.

“The CHMP’s recommendation is now referred to the European Commission (EC), which is expected to provide its decision by the second quarter of this year.”

“It is a privilege and an honour to pioneer the successful delivery of an enzyme replacement therapy delivered directly to the brain and to show that the treatment can slow or stabilise the progression of this degenerative brain disease.”

The Brineura MAA was based on an open-label, dose-escalation study on 24 patients with CLN2 disease between three and eight years old and an open-label extension study.

The study evaluated the safety and tolerability of intracerebroventricular-administered Brineura and analyse its effectiveness using a CLN2 disease-specific rating scale score in comparison with natural history data after 48 and 72 weeks of treatment.

Brineura is also currently under review by the US Food and Drug Administration (FDA).

Image: Crystal structure of tripeptidyl-peptidase I (TPP1) with co-ordinated zinc (grey), chlorine (yellow) and calcium (red) ions. Rendered from PDB 3EE6. Photo: courtesy of Matthew Jenner via Wikipedia.