Cerliponase Alfa: First Global Approval

  title={Cerliponase Alfa: First Global Approval},
  author={Anthony Markham},
  • A. Markham
  • Published 6 June 2017
  • Medicine, Biology
  • Drugs
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), a paediatric neurodegenerative disease caused by a deficiency in TPP1. CLN2 is characterised by progressive impairment of motor function, language deficiencies, seizures, ataxia, blindness and early death, and intracerebroventricular infusion of cerliponase alfa has been shown to reduce the… 
Cerliponase alfa for CLN2 disease, a promising therapy
Current research indicates treated children have better outcomes in comparison to their natural history counterparts allowing preserved ambulation and language, and future research should be directed toward the discovery of therapies that may allow the delivery or promote production of functional CLN2 protein such as gene therapy.
Changing Times for CLN2 Disease: The Era of Enzyme Replacement Therapy
Administration of cerliponase alfa in patients with CLN2 disease has led to significant reductions in the rate of decline of motor and language functions in comparison with a natural history population, highlighting the need to understand different patterns of disease progression and clinical needs in treated patients.
Pharmacological approaches to tackle NCLs.
The LINCE Project: A Pathway for Diagnosing NCL2 Disease
LINCE proved to be a simple, useful, and reliable tool for the diagnosis of NCL2, enabling clinicians to diagnose NCL1 faster, and the presence of N CL2-positive cases in the authors' population and availability of treatment may facilitate the inclusion of Ncl2 in neonatal screening programs for early diagnosis.
Presymptomatic treatment of classic late-infantile neuronal ceroid lipofuscinosis with cerliponase alfa
Cerliponase alfa is able to delay the onset of symptoms when treatment is started in a presymptomatic stage of CLN2 disease and the results advocate the start of treatment at an early age before symptom onset, but should be confirmed in a larger cohort study.
Genetics and Therapies for GM2 Gangliosidosis
Current knowledge pertaining to the pathobiology as well as potential innovative treatments for the GM2 gangliosidoses are outlined.
Advances in the treatment of neuronal ceroid lipofuscinosis
Much progress has been made in NCL research, including better animal models, biomarkers and improved therapeutics with many of them reaching the clinical trial stage, with emphasis on the last decade.
Neuronal Ceroid Lipofuscinosis: Potential for Targeted Therapy
An overview of genetics for each CLN disease is provided, and the current understanding from pre-clinical and clinical study of potential therapeutics is discussed, to slow or even halt disease progression.