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BioMarin announced that its drug, Brineura (cerliponase alfa), received recommendation by NICE’s Highly Specialized Technology committee.
Pharma Analyst Kajal Jaddoo of GlobalData, a data and analytics company, offered her view on the potential the drug has. According to GlobalData, Brineura global sales are predicted to reach $358m by 2025.
“Brineura has the potential to be commercially successful as currently there is no cure or life-extending treatments for neuronal ceroid lipfuscinosis type 2 (CLN2), a rare inherited disease and GlobalData forecasts that Brineura will generate global sales of $358m by 2025.
“Though Brineura has received approval, it will still need to be evaluated by an ongoing judicial review and payers before it gains widespread use, meaning the drug may face a slow uptake.
“Brineura is a recombinant human tripeptidyl peptidase enzyme. It is administered by intracerebral route through patented technology. The drug was indicated to slow loss of movement ability in symptomatic pediatric patients aged three and older with infantile CLN2. Brineura is tripeptidyl Peptidase 1 (TPP1) replacement agent. It restores TPP1 enzyme activity and breaks down the storage materials that cause CLN2 disease.”
To read more about Brineura, you can read the comprehensive article we wrote on the drug here in May.