The EMA has granted BioMarin's request for accelerated assessment for the planned cerliponase alfa Marketing Authorization Application (MAA). Accelerated assessments are granted on the grounds that a product may satisfy an unmet medical need and is of major interest from the point of view of therapeutic innovation and public health.
Accelerated assessment has the potential to shorten EMA's review procedure. However, at any time during the MAA assessment, the EMA may decide to continue the assessment under standard assessment timelines, and most applications that initially qualify for accelerated assessment are ultimately reviewed on a standard timeline.
Cerliponase alfa is a recombinant human tripeptidyl peptidase 1 (rhTPP1) intended for the treatment of children with CLN2 disease, a form of Batten disease. CLN2 disease is a rapidly progressing, fatal neurodegenerative disease with no approved treatments.
The company expects to submit the cerliponase alfa MAA to the EMA and the Biologics License Application to the FDA by mid-year. If the cerliponase alfa MAA is accepted by the EMA, then an opinion from the Committee for Medicinal Products for Human Use (CHMP) is anticipated in the first quarter of 2017, and, if positive, a decision from the European Commission could be received in the first half of 2017.
"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. This could be the first therapeutic option available for these children," said Jean-Jacques Bienaimé, chairman and chief executive officer of BioMarin. "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."
In the U.S., BioMarin is seeking to shorten the regulatory review time by requesting Priority Review. Priority Review status is designated by the FDA to drugs that offer major advances in treatment or provide a treatment where no adequate therapy exists. The FDA will evaluate this request following submission of the planned Biologics License Application (BLA). Cerliponase alfa has been granted Orphan Drug designation by the FDA and EMA and Breakthrough Therapy designation by the FDA.