NICE rejects BioMarin's Batten disease drug

The UK’s National Institute for Health and Care Excellence (NICE) has published draft guidance that does not recommend Brineura (cerliponase alfa) made by US rare disease drug developer BioMarin Pharmaceutical (Nasdaq: BMRN) for children with neuronal ceroid lipofuscinosis type 2 (CLN2) – a very rare inherited condition affecting between 1 and 6 babies each year in the UK.

This preliminary decision, which is now the subject of public consultation until March 5, comes as the company confirmed it is to negotiate with NHS England terms that could allow the drug to be made available while uncertainties in the evidence of its long term benefits are addressed.

Brineura was approved in Europe last June and in in April 2017 in the USA, where BioMarin executives said the drug would have an average net price of $486,000 per patient per year.