BioMarin Pharmaceuticalsaid today that it got from the European Commission (EC) a grant for Brineura (cerliponase alfa), the first treatment approved in the European Union (EU) for tripeptidyl peptidase 1 (TPP1) deficiency, neuronal ceroid lipofuscinosis type 2 (CLN2).
BioMarin said that on April 21, 2017, the Committee for Medicinal Products for Human Use (CHMP), the scientific committee of the European Medicines Agency (EMA), adopted a positive opinion for the company’s Marketing Authorization Application (MAA) for BrineuraTM (cerliponase alfa) following an accelerated review procedure, reserved for medicinal products expected to be of major public health interest. The EMA revised process for accelerated assessment came into effect June 1, 2016. Brineura is one of the first therapies to go through this process.
CLN2 disease is an ultra-rare, rapidly progressive fatal brain condition, which affects an estimated 1,200 to 1,600 children worldwide, many of whom are undiagnosed. These affected children completely lose the ability to walk and talk around 6 years of age. During the later stages of the disease, feeding and tending to everyday needs become very difficult with death usually occurring between 8 and 12 years of age.
The principal investigator of the Brineura studies, Angela Schulz, from Department of Paediatrics at Children’s Hospital, University Medical Center Hamburg-Eppendorf, said: “Today represents several important milestones for the medical and patient communities, and also for me, both professionally and personally. For the first time since entering this field nearly 15 years ago, I can now tell families affected by CLN2 disease that there is a meaningful treatment that may help their child, and provide hope.”