Brineura

Описание товара

About Brineura

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. Brineura contains the active substance cerliponase alfa.

What it is used for

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

How it is used

Brineura is infused directly into the brain. Before the first infusion, the patient will need to have surgery to implant a device, which runs from the outside of the skull through to the fluid cavity in the brain where the medicine is delivered to. The infusions are given once every two weeks by a healthcare professional knowledgeable about giving medicines into the brain. To reduce the risk of infusion-related reactions, patients may be given other medicines before or during treatment with Brineura or the infusion may be slowed down. The treatment can continue for as long as the patient benefits from it. Brineura can only be obtained with a prescription.

How it works

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.

Active substance: cerliponase alfa

Source: EMA EPAR