Regulatory Decision Summary for Brineura

Review decision

The Regulatory Decision Summary explains Health Canada’s decision for the product seeking market authorization. The Regulatory Decision Summary includes the purpose of the submission and the reason for the decision.

Product type:


Medicinal ingredient(s):

cerliponase alfa

Therapeutic area:

Enzyme Replacement Therapy

Type of submission:

New Drug Submission

Control number:

What was the purpose of this submission?


The purpose of this submission is to seek marketing authorisation for Brineura (cerliponase alfa) in Canada for the treatment of neuronal ceroid lipofuscinosis type 2 (CLN2) disease.


Why was the decision issued?


The efficacy and safety of Brineura were assessed over 96 weeks in an open-label clinical study and an ongoing long-term extension study in patients with CLN2 disease compared to an untreated historical control group. Twenty-three patients, aged 3 to 8 years, received Brineura at 300 mg every other week to a maximum of 145 weeks.

The primary efficacy endpoint was the proportion of patients with an absence of an unreversed two-point decline on a disease-specific clinical rating scale for the motor and language functions. Efficacy was demonstrated at Week 48 of the open-label study where 87% of the patients met the primary endpoint. The proportion of patients with no decline at both Week 48 and Week 96 (Week 48 of the long-term extension study) was 83% and showed durability of treatment effect.

The major risks were severe or life-threatening device-related infections and meningitis. The most common adverse events (AEs) were pyrexia (71%), vomiting (63%) and seizure (58%). The most common serious AE was hypersensitivity (29%). There were no deaths during the clinical development program. Given the poor prognosis of this disease, the safety profile of Brineura is considered acceptable with the appropriate labelling and post-market monitoring that has been put in place through the Brineura Risk Management Plan.

Overall, the benefits of Brineura outweigh its risks for the indication sought. The benefit/risk profile of Brineura is favourable in the target patient population.

For more information on Health Canadas decision, please view the Summary Basis of Decision.


Decision issued

Approved; issued a Notice of Compliance in accordance with the Food and Drug Regulations.