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NICE nixes BioMarin's ultra-orphan therapy for lack of long-term data

February 22, 2019 9:09 PM UTC

The U.K.'s NICE did not recommend Brineura cerliponase alfa from BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) due to uncertainty around the therapy's long-term efficacy and concerns NHS would be unable to implement the rare disease biotech's proposed managed access agreement.

The U.K. agency's final evaluation determination confirmed a previous evaluation from February 2018 not to recommend the recombinant human tripeptidyl peptidase-1 (TPP1) enzyme replacement therapy to treat neuronal ceroid lipofuscinosis type 2 (CLN2) disease. NICE said the form of Batten disease is diagnosed in about 3-6 children per year in the U.K., with 30-50 children currently living with the condition. Symptoms begin around the age of two with a life expectancy of 10 years. Brineura is the only approved treatment for the disease...

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