NEJM publishes updated results for Brineura treatment for children with CLN2 disease. - BioMarin

BioMarin Pharmaceutical Inc. announced that the New England Journal of Medicine (NEJM) published updated results from a multi-center, open-label, dose-escalation and ongoing extension study evaluating the efficacy and safety of Brineura (cerliponase alfa) in children with CLN2 disease .

The new data demonstrated that treatment with Brineura resulted in less decline in motor and language function compared to historical controls. Brineura was approved in April 2017 by the FDA 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 also was approved in June 2017 by the European Commission. Brineura is the first treatment approved to treat children with CLN2 disease, a form of Batten disease.

The data published in NEJM showed that patients treated with Brineura experienced a slower rate of decline in motor and language function than historical controls (the unadjusted mean rate of decline in the score on the motor�language scale was 0.27�0.35 points per 48 weeks among the 23 treated patients as compared with 2.12�0.98 among the 42 historical controls, a difference of 1.85�0.21 points (95% CI, 1.51 to 2.18; P<0.001). in addition to its publication in nejm, dr. emily de los reyes, m.d., director of the batten disease center of excellence at nationwide children's hospital in columbus, ohio and a clinical trial investigator involved in the study, presented an updated analysis of the study at 96 weeks, at the invitation of the aan science committee during the clinical trials plenary session at its 70th annual meeting on april 24th, 2018.the primary endpoint of the study�the time to a 2-point decline in motor-language score (range 0-6, 0 representing no function and 3 representing normal function for each of the two domains) assessed as mean change per 48 weeks compared to 42 historical controls�was met. median time to a 2-point decline in motor-language score was 345 days (49.3 weeks) for natural history and not reached for treated patients (9 percent of treated patients had declined by 2 points at 345 days).>

Brineura is the first and only therapy approved by the FDA and European Union to treat children with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), a form of Batten disease also known as tripeptidyl peptidase 1 (TPP1) deficiency. Due to the rapidly progressing nature of the disease and clinical efficacy of its phase 1/II study, Brineura was approved in just four years from first patient dosed to registration approval.

See- "Study of Intraventricular Cerliponase Alfa for CLN2 Disease"-Angela Schulz, M.D., Temitayo Ajayi, M.D., Nicola Specchio, M.D., Ph.D., Emily de Los Reyes, M.D., Paul Gissen, M.B., Ch.B., Ph.D., Douglas Ballon, Ph.D., Jonathan P. Dyke, Ph.D., Heather Cahan, M.D., Peter Slasor, Sc.D., David Jacoby, M.D., Ph.D., and Alfried Kohlsch�tter, M.D. for the CLN2 Study Group-April 24, 2018 DOI: 10.1056/NEJMoa1712649.