BioMarin will be celebrating news that regulators on both sides of the Atlantic have agreed to review Vimzin under accelerated review processes.
Vimzin is an enzyme replacement therapy for which the drugmaker is seeking approval as a treatment for the rare lysosomal storage disorder Mucopolysaccharidosis Type IVA (MPS IVA), also known as Morquio A Syndrome.
During an initial review of the Biologics License Application the US Food and Drug Administration requested extra Chemistry, Manufacturing and Controls information, and following receipt of this from BioMarin it has extended the PDUFA action date by three months to February 28 next year.
In the EU, assuming the marketing application remains on the accelerated assessment timeline, a CHMP opinion is anticipated in December this year, which means a decision from the European Commission could be handed down in the first quarter of 2014, the firm said.
The estimated prevalence of MPS IVA is around 3,000 patients in the developed world, and it is believed that around 20% or patients are in North America and 50% in Europe.
"MPS IVA represents a significant unmet medical need for those affected, and our hope is to offer a life-altering treatment option to patients worldwide," said Jean-Jacques Bienaimé, Chief Executive Officer of BioMarin.