The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion for BioMarin Pharmaceutical‘s marketing authorisation application (MAA) for Vimizim (elosulfase alfa), to treat patients with mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome).

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.

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The CHMP’s recommendation is now referred to the European Commission (EC) and if approved, the company would receive marketing authorisation for Vimizim in all the EU Member States.

"We will leverage our existing European infrastructure to ensure that these patients gain access to Vimizim as quickly as possible."

The company said that 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: "We will leverage our existing European infrastructure to ensure that these patients gain access to Vimizim as quickly as possible.

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Earlier this month, the US Food and Drug Administration (FDA) approved Vimizim for the same indication.

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Vimizim is the first enzyme replacement therapy (ERT) designed to target the underlying cause of Morquio A Syndrome, which is a deficiency in the enzyme N-acetylgalactosamine-6 sulfatase (GALNS).

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 pervasive and progressive accumulation of GAGs leads to significant morbidities and multisystemic clinical impairments, which results in reduced functional capacity, impaired quality of life and early mortality.

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.