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news.US-based BioMarin Pharmaceutical has announced that the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion for the marketing authorization application (MAA) of its Vimizim (elosulfase alfa) for the treatment of Morquio A syndrome, also called Mucopolysaccharidosis Type IVA (MPS IVA).
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The CHMP’s recommendation is now referred to the European Commission (EC) and if approved, the company will receive marketing authorization for Vimizim in all EU Member States.
The EC is expected to render a final decision for Vimizim in the second quarter of 2014.
BioMarin chief executive officer Jean-Jacques Bienaimé said the positive CHMP opinion is an important milestone in the company’s mission to provide the first approved therapy to treat Morquio A patients across Europe.
"We will leverage our existing European infrastructure to ensure that these patients gain access to Vimizim as quickly as possible," Bienaimé said.
In February 2014, the US Food and Drug Administration (FDA) had approved Vimizim for the same indication.
Vimizim is the first enzyme replacement therapy (ERT) designed to target the underlying cause of Morquio A Syndrome – a deficiency in the enzyme N-acetylgalactosamine-6 sulfatase (GALNS).
Morquio A syndrome is a rare, severely debilitating and progressive disease that occurs as a result of a deficiency of activity in an enzyme involved in glycosaminoglycan (GAG) metabolism.
The drug is intended to provide the exogenous enzyme GALNS that will be taken up into the lysosomes and increase the catabolism of GAGs.
The most common features of Morquio A Syndrome include progressive skeletal dysplasia, the need for frequent surgical procedures related primarily to musculoskeletal or respiratory dysfunction, and significant limitations in mobility, endurance and breathing.