UK – NICE approves BioMarin’s Brineura after initial setback

First treatment for rare disease

BioMarin and NICE had a rocky start with Batten disease therapy Brineura, but the two have now come to an agreement. 

The positive recommendation of Brineura was agreed by NICE’s Highly Specialised Technology committee. The disease, known as neuronal ceroid lipfuscinosis type 2 (CLN2), is a very rare inherited condition. According to NICE, it is thought to affect between one and six babies every year in the UK.

Although Brineura does not does not cure the disease, NICE agreed that it is an important development in the treatment of the condition. There is currently no cure or life-extending treatments for CLN2, so the approval is likely to be met with elation from patient groups…