For neuronal ceroid lipofuscinosis type 2 (CLN2 disease). Sold as Brineura. Every FDA application for this active ingredient.
As of Nov 15, 2026, FDA lists 1 application for Cerliponase Alfa (sold as Brineura), first approved Apr 27, 2017, with 1 new use approved and 0 published complete response letters.
| Intravitreal ERT to Prevent Retinal Disease Progression in Children With CLN2Neuronal Ceroid Lipofuscinosis Type 2 · David L Rogers, MD | Phase 1 / Phase 2 | Active, not recruiting | Dec 10, 2021 |
| Natural History and Longitudinal Clinical Assessments in NCL / Batten Disease, the International DEM-CHILD DatabaseNeuronal Ceroid Lipofuscinosis, Batten Disease · Universitätsklinikum Hamburg-Eppendorf | — | Recruiting | Nov 3, 2020 |
| Cerliponase Alfa Observational Study in the USLate-Infantile Neuronal Ceroid Lipofuscinosis Type 2 · BioMarin Pharmaceutical | — | Active, not recruiting | Jul 20, 2020 |
| Examining Developmental Outcomes of Children Diagnosed With CLN2 DiseaseBatten Disease, CLN2 · Jessica Scherr | — | Enrolling by invitation | Mar 5, 2019 |
| Approved | Application | Sponsor | Supplements | Marketing |
|---|---|---|---|---|
| Apr 27, 2017 | BrineuraBLA 761052 · New molecular entity | Biomarin Pharm | 5 | Marketed |
Applications, approvals and products come from FDA's Drugs@FDA data files, refreshed weekly, grouped by active ingredient as FDA spells it. Complete response letters are FDA's published letters. Medicare spending is CMS's Part D and Part B dashboard, matched to this ingredient by generic name; shortages are FDA's shortage list, matched by application number; clinical trials are ClinicalTrials.gov studies naming the ingredient as an intervention, refreshed daily; safety programs are FDA's REMS list, matched by application number or, for shared programs, by ingredient. Drugs@FDA
