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NICE refuses to endorse Biomarin’s Batten disease drug pharmaceutical-technology
February 26, 2019
The UK National Institute for Health and Care Excellence (NICE) has published a draft guidance saying that Biomarin’s therapy for Batten disease, Brineura (cerliponase alfa), cannot be recommended for use in the National Health Service (NHS)....
NICE says drug to treat Batten disease cannot be recommended for NHS use because company is unable to address concerns about long-term effectiveness firstwordpharma
February 23, 2019
NICE has published draft guidance which confirms its earlier decision not to recommend cerliponase alfa (also called Brineura and made by Biomarin) for children with Batten disease - a very rare inherited condition affecting between 1 and 6 babies each ye
FDA approves first treatment for a form of Batten disease worldpharmanews
May 02, 2017
The U.S. Food and Drug Administration approved Brineura (cerliponase alfa) as a treatment for a specific form of Batten disease.