Cerliponase alfa
| Clinical data | |
|---|---|
| Trade names | Brineura |
| License data | |
| Routes of administration | Intraventricular |
| ATC code | |
| Identifiers | |
| CAS Number | |
| DrugBank | |
| UNII | |
| KEGG | |
| Chemical and physical data | |
| Formula | C2657H4042N734O79S11 |
| Molar mass | ~59000 |
Cerliponase alfa, marketed as Brineura, is an enzyme replacement treatment for Batten disease, which is a form of neuronal ceroid lipofuscinosis.[1]
On 27 April 2017, it was the approved by the United States Food and Drug Administration to slow loss of walking ability in symptomatic children over three years old with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2). The disease is also known as tripeptidyl peptidase-1 (TPP1) deficiency.[2] Also, it was approved by European Medicines Agency on 30 May 2017.[3] In the United Kingdom NICE evaluated Cerliponase alfa for the treatment of CLN2 and deemed it not cost-effective.[4][5]
It can be given by an intraventricular route which allows significant uptake into the brain.
It was developed by BioMarin Pharmaceutical.
References
- ↑ Cerliponase alfa
- ↑ "Press Announcements - FDA approves first treatment for a form of Batten disease". www.fda.gov. Retrieved 23 July 2017.
- ↑ European Commission Approves Brineura™ (cerliponase alfa), the First Treatment for CLN2 Disease, a Form of Batten Disease and Ultra-Rare Brain Disorder in Children
- ↑ "Evaluation consultation document: Cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2". NICE. Retrieved 9 August 2018.
- ↑ McKee, Selina (13 February 2018). "NICE deems Batten disease therapy too costly for NHS use". Pharma Times. Retrieved 9 August 2018.