Following the news (Thursday 12 September) that BioMarin’s Brineura (cerliponase alfa) received recommendation by NICE’s Highly Specialised Technology committee;

Kajal Jaddoo, Pharma Analyst at GlobalData, a leading data and analytics company, offers her view on the drug’s potential:

“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.”