US-based BioMarin Pharmaceutical has received approval from the US Food and Drug Administration (FDA) to use its Vimizim (elosulfase alfa) for treatment of patients with mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome).
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.
BioMarin chief executive officer Jean-Jacques Bienaimé said: "Vimizim is the first and only therapy designed to address the condition at the cellular level, fulfilling a large unmet medical need for patients and their families.
"With the approval of VIMIZIM, BioMarin firmly establishes its leadership in advancing therapies to treat MPS diseases.
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"We have developed three therapies to treat three different MPS diseases and continue to build on our extensive scientific and clinical knowledge of lysosomal storage disorders to develop therapies for other rare genetic diseases."
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.
The company will begin shipments of Vimizim to the distribution channels, as well as start promotion of the drug in the US immediately.
Marketing applications for Vimizim have also been submitted by the company in the European Union, Brazil, Australia, Canada, and Mexico.
The safety and effectiveness of the drug were evaluated in a clinical trial involving 176 participants with Morquio A syndrome, ranging in age from five to 57 years.
In the 24-week, randomised, double-blind, placebo-controlled trial, patients treated with Vimizim showed greater improvement in a six-minute walk test compared to patients treated with placebo.
The primary endpoint of the trial, change in six-minute walk distance at 24 weeks, was statistically significant in patients receiving weekly infusions of Vimizim 2mg/kg with a mean increase of 22.5m compared to the patients who received placebo.
The most common adverse events observed in patients treated with Vimizim during clinical trials included fever, vomiting, headache, nausea, abdominal pain, chills and fatigue.
Image: Morquio’s syndrome is an autosomal recessive mucopolysaccharide storage disease. Photo: courtesy of freedigitalphotos.net.