The Committee for Medicinal Products for Human Use (CHMP), the scientific committee of the European Medicines Agency (EMA), has adopted...
BioMarin Pharmaceutical Inc. announced the European Commission (EC) has granted marketing authorization for Brineura (cerliponase alfa), the first treatment approved...
BioMarin Pharmaceutical Inc. announced that the New England Journal of Medicine (NEJM) published updated results from a multi-center, open-label, dose-escalation...
BioMarin announced that twenty-three patients in the ongoing open-label extension study treated with Brineura (cerliponase alfa) showed continued maintenance of...
BioMarin Pharmaceutical Inc. announced a program update for cerliponase alfa, a recombinant human tripeptidyl peptidase 1 (rhTPP1) to treat children...
Cerliponase alfa (Brineura™) is a recombinant human tripeptidyl peptidase-1 (TPP1) being developed by BioMarin Pharmaceutical Inc. for use in patients with neuronal ceroid lipofuscinosis type 2 (CLN2)...
This Phase 2 open-label, multicenter study will evaluate the safety, tolerability, and efficacy of BMN 190 intracerebroventricular (ICV) administration every other week (qow) for a period of 96 weeks, in patients with CLN2.
The Phase 1/2 study (190-201) evaluated the efficacy and safety of doses up to 300 mg/every other week (qow) BMN 190 in patients with CLN2. The dose and regimen for this study (190-202) are based on the results of the 190-201 study.
The National Institute for Health and Care Excellence (NICE) has published draft guidance that does not recommend the drug cerliponase...