The Highly Specialised Technology committee of the U.K.'s National Institute of Health and Care Excellence (NICE) said it is supporting a positive recommendation for Biomarin Pharmaceutical Inc.'s Brineura (cerliponase alfa), for children with the rare and fatal neurodegenerative condition ceroid lipofuscinosis type 2 (CLN2), affecting between one and six babies each year in the U.K. The independent committee noted that although cerliponase alfa is not a cure for the disease, it is an important development for treating the condition, and that it has shown substantial short-term benefits in slowing the rate at which it progresses. Details of the managed access agreement will be worked out by NICE in consultation with NHS England, Biomarin, clinicians and the Batten Disease Family Association. A managed access agreement describes the patient eligibility criteria for access, as well as stopping rules and data collection arrangements. The agreement, NICE reported, is likely to include all new patients with CLN2 except those with advanced disease and very low motor and language scores of one or less, because they would be unlikely to benefit.

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