BioMarin Pharmaceutical Reports the US FDA’s Approval of Brineura (cerliponase alfa) to Treat Neuronal Ceroid Lipofuscinosis Type 2
Shots:
- The US FDA’s approval has expanded Brineura’s use to slow ambulation loss in children (all ages) with neuronal ceroid lipofuscinosis type 2, whether symptomatic or presymptomatic
- Approval was based on P-II (Study 190-203) trial of Brineura in children (1-6yrs.; 8 were >3yrs.) that showed slowing of motor function decline & delayed disease onset, confirming reduced disease progression & delay onset with the treatment after & before 3yrs. of age, respectively
- Data also showed 2-point decline or a score of zero in 0% (>3yrs.) at wk.169; 7/8 matched with 18 untreated from a natural history cohort with 61% having an unreversed 2-point decline or score of zero; all 7 (>3yrs.) maintained a motor score of three, indicating delayed disease onset
Ref: BioMarin | Image: BioMarin
Related News:- BioMarin Receives the EMA’s CHMP Positive Opinion to Expand Use of Voxzogo (vosoritide) for Children Aged ≥4 Months with Achondroplasia
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Disha is a content writer at PharmaShots. She is passionate and curious about recent updates and developments in MedTech and Pharma industry. She covers news related to clinical trial results and updates. She can be contacted at connect@pharmashots.com.
