BLAINJECTIONINJECTABLEPriority Review
Approved
Apr 2017
Lifecycle
Peak
Competitive Pressure
30/100
Clinical Trials
1
Mechanism of Action
CLN2 disease is a neurodegenerative disease caused by deficiency of the lysosomal enzyme tripeptidyl peptidase-1 (TPP1), which catabolizes polypeptides in the CNS. TPP1 has no known substrate specificity. Deficiency in TPP1 activity results in the accumulation of lysosomal storage materials…
Clinical Trials (1)
Cerliponase Alfa Observational Study in the US
Started Aug 2020
35 enrolled
Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2