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NICE recommend Brineura▼ (cerliponase alfa) for the treatment of neuronal ceroid lipofuscinosis type 2

Brineura▼, for Batten's disease (Neuronal ceroid lipofuscinosis) and Neuronal ceroid lipofuscinosis from BioMarin

The National Institute for Health and Care Excellence (NICE) have recommended that cerliponase alfa, brand name Brineura▼, as being suitable for patients with neuronal ceroid lipofuscinosis type 2.

This means that the medicine is available to patients by NHS England and Wales.

Neuronal ceroid lipofuscinosis type 2 (CLN2 disease), is an inherited condition in children that leads to progressive brain damage.

CLN2 disease is also known as Batten's disease.

NICE state: 'Cerliponase alfa is recommended as an option for treating neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase 1 (TPP1) deficiency, only if the conditions in the managed access agreement are followed.

This recommendation is not intended to affect treatment with cerliponase alfa that was started in the NHS before this guidance was published. People having treatment outside this recommendation may continue without change to the funding arrangements in place for them before this guidance was published, until they and their NHS clinician consider it appropriate to stop. For children or young people, this decision should be made jointly by the clinician and the child or young person, or the child's or young person's parents or carers.

Managed access schemes are agreements between the manufacturer and the NHS, usually specifying a set of conditions under which reimbursement for the medicine will be made.

You can follow developments on Brineura by using our Medicines Tracker service which provides users with updates about the medicines they are interested in.

About Brineura (source EMA)
Brineura is a medicine for treating neuronal ceroid lipofuscinosis type 2 (CLN2 disease), an inherited condition in children that leads to progressive brain damage.

Because the number of patients with CLN2 is low, the disease is considered ‘rare’, and Brineura was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 13 March 2013.

Patients with CLN2 lack an enzyme required for normal brain development called tripeptidyl-peptidase-1 (TPP1). The active substance in Brineura, cerliponase alfa, is a copy of TPP1 and is used as a replacement for the missing enzyme.

The medicine is infused directly into the brain in order to bypass the blood-brain barrier, a protective barrier that separates the blood stream from the brain, and prevents substances such as medicines, from entering brain tissue.

Brineura was first made available in the EU in 2016. It is manufactured by BioMarin.

European Medicines Agency website page for Brineura
Accessed 28/11/19
Links available in External Resources

© NICE [2019]

Cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2. Highly specialised technologies guidance [HST12]. Published date: 27 November 2019
Available from: See Link below. All rights reserved. Subject to Notice of rights
NICE guidance is prepared for the National Health Service in England. All NICE guidance is subject to regular review and may be updated or withdrawn. NICE accepts no responsibility for the use of its content in this product/publication.

The information provided by NICE was accurate at the time this article was issued.

NICE: Cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2

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