Ongoing study shows durable benefit of Brineura for three years for neuronal ceroid lipofuscinosis type 2 (Batten disease)- BioMarin

BioMarin announced that twenty-three patients in the ongoing open-label extension study treated with Brineura (cerliponase alfa) showed continued maintenance of stabilization of disease for three years or more as measured by the CLN2 Clinical Rating Scale as compared to untreated patients from a natural history cohort. Patients were assessed for decline in the motor domain of the CLN2 Clinical Rating Scale. The scale measures performance of mobility with normal function being a score of 3 and no function being a score of 0. Decline was defined as having a sustained 2-point decline or an unreversed score of 0 in the motor domain of the CLN2 Clinical Rating Scale.

Twenty-four patients aged 3-8 years were enrolled in the clinical study. One patient withdrew after week 1 due to inability to continue with study procedures; 23 patients were treated with Brineura every other week for three years or more. Two Brineura treated patients, who at study initiation had a maximum score, were excluded from the analyses; they maintained that score throughout the study period. Both children had motor scores of 3 after three years of treatment.

Comment: the European Commission (EC) on 1 June 2017 granted marketing authorization for Brineura (cerliponase alfa), the first treatment approved in the European Union for the treatment of neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase 1 (TPP1) deficiency. The dosing administration includes all ages from birth.The FDA on 28 April 2017 approved Brineura (cerliponase alfa) to slow the loss of ambulation in symptomatic pediatric patients 3 years of age and older with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase 1 (TPP1) deficiency. Brineura is the first treatment approved to treat children with CLN2 disease, a form of Batten disease.