First treatment for CLN2 disease approved in Europe

The European Commission has granted marketing authorization for Brineura (cerliponase alfa), the first treatment approved in the European Union for the treatment of neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase 1 (TPP1) deficiency.

The dosing administration includes all ages from birth, noted rare disease drug developer BioMarin Pharmaceutical (Nasdaq: BMRN), whose shares gained 1.4% to $88.86 on the announcement.

On April 21, 2017, the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for the company's Marketing Authorization Application (MAA) for Brineura 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.