News Feature | May 2, 2014

BioMarin Receives EC Approval For Ultra-Rare Genetic Disease Drug

By Estel Grace Masangkay


BioMarin Pharmaceutical announced that it has received marketing approval from the European Commission for Vimizim (elosulfase alfa) for the ultra-rare genetic disease Morquio A syndrome in patients of all ages.

Vimizim is the first treatment to receive approval in the EU for Mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome). The drug was given orphan drug status in the EU and gains ten years of market exclusivity.

Jean-Jacques Bienaimé, CEO of BioMarin, said, “This approval of Vimizim in Europe is a key milestone because we estimate that 85 percent of Morquio A or MPS IVA patients live outside of the United States. BioMarin is a leader in advancing therapies to treat MPS diseases with three therapies to treat three different MPS diseases. We continue to build on our extensive scientific and clinical knowledge of lysosomal storage disorders to develop therapies for other rare genetic diseases.”

Morquio A syndrome is a disease characterized by the absence of an enzyme essential in the breakdown and removal of the glycosaminoglycans (GAGs) called keratan sulfate (KS) and chondroitin-6-sulfate (C6S). The incompletely broken down GAGs remain stored in cells in the body and causes systemic skeletal dysplasia, short stature, and joint abnormalities. The disease seriously affects both the length and quality of life of patients. According to the company, the rate of incidence of Morquio A syndrome is not yet confirmed and varies among different populations. Estimates vary between 1 person affected in 200,000 or 1 in 450,000 live births.

Vimizin is the first enzyme replacement therapy designed to target the underlying cause of Morquio A Syndrome. The drug provides the exogenous enzyme N-acetylgalactosamine-6 sulfatase (GALNS) that will be taken up into the lysosomes and increase the GAGs catabolism.

Christian J. Hendriksz, Salford Royal NHS Foundation Trust and lead investigator for the Phase 3 clinical trial, said, “VIMIZIM addresses the condition at the cellular level, fulfilling a large unmet medical need and represents an advance for Morquio A patients and their families. As a treating physician, I am encouraged that the therapy shifts the treatment beyond supportive care to treating the underlying cause of the disease, potentially changing the course of this devastating disease.”

The FDA previously approved Vimizim last February 14, 2014. The company has also filed applications for the drug in different countries including Japan, Brazil, Mexico, Canada, and Australia.