Final NICE rejection for Biomarin’s childhood rare disease drug
Pharmaphorum Media Limited | February 22, 2019
After a year of negotiations, NICE has confirmed that it is still unable to recommend Biomarin’s Batten disease drug, saying that the company is unable to address concerns about long-term effectiveness. Brineura (cerliponase alfa) is designed to treat children with Batten disease, also known as neuronal ceroid lipofuscinosis type 2 (CLN2) – a very rare inherited condition affecting between one and six babies each year in the UK. In its earlier draft guidance, published last year, NICE’s independent committee had agreed that, although Brineura is not a cure for Batten disease, it is an important development for treating the condition and it has shown substantial short-term benefits in slowing the rate at which it progresses.
But NICE said that in the negotiations since then, BioMarin was unable to price Brineura at a level that would have addressed the problems highlighted during its assessment of the drug. Costing over £500,000 per person for each year’s treatment, Brineura is an enzyme replacement therapy administered directly into the brain via a surgically implanted permanent access device. In the absence of long-term evidence about its effectiveness in stabilizing the disease and preventing death, the independent committee considered that the drug was not valued for money within the context of highly specialized service.