BioMarin Pharmaceutical Inc. recently announced that the European Medicines Agency (EMA) has approved accelerated assessment for BioMarin’s cerliponase alfa Marketing Authorization Application (MAA).
Accelerated assessments are only used if products can meet a currently unmet medical need. The products must also fulfill a significant interest for the public health and the therapeutic innovation industries.
Approving the MAA will reduce the review procedure that the EMA typically uses, though the agency can choose to carry on the assessment within standard assessment timelines; most of the accelerated assessments are finalized with a standard timeline.
Cerliponase alfa, a recombinant human tripeptidyl peptidase 1 (rhTPP1), is designed to treat children who have CLN2 disease -- a kind of Batten disease that is neurodegenerative and fatal. Cases of CLN2 disease progress quickly. As of today, the FDA has not approved any treatments.
"We are pleased that the EMA has recognized the need to bring a therapy to children with this particular form of Batten disease as quickly as possible," Jean-Jacques Bienaimé, chairman and CEO of BioMarin, said. "This could be the first therapeutic option available for these children. We look forward to entering the regulatory review phase for cerliponase alfa on a global basis. CLN2 disease represents a significant unmet medical need for those affected, and our hope is to offer a life-altering treatment option to patients worldwide."
