The Japanese Ministry of Health, Labour and Welfare (MHLW) granted approval of BioMarin Pharmaceutical's Vimizim (elosulfase alfa) for the treatment of patients with mucopolysaccharidosis type IVA (MPS IVA), also known as Morquio A syndrome. Vimizim is the first treatment in Japan approved for this condition. Vimizim was reviewed under the Orphan Drug programme.

Professor Torayuki Okuyama from the National Center for Child Health and Development, who is currently using Vimizim on patients in a clinical trial said, "Morquio A syndrome is an ultra-rare and difficult condition to treat. Vimizim is the only specific treatment available and offers improved endurance to these patients."

"We are pleased to be able to deliver the first drug therapy for Morquio A to patients in Japan. Vimizim will be the first product approval that BioMarin will market in Japan," said Jean-Jacques Bienaimé, CEO of BioMarin. "Now that BioMarin has established its presence in Japan, we look forward to delivering future therapies to treat patients who have rare genetic diseases with unmet medical needs."

Morquio A syndrome is an ultra-rare, severely debilitating disease affecting an estimated 3,000 patients in the developed world. The disease occurs as a result of a deficiency of activity in an enzyme involved in glycosaminoglycan (GAG) metabolism. The pervasive and progressive accumulation of GAGs leads to significant morbidities and multisystemic clinical impairments resulting in diminished functional capacity, impaired quality-of-life and early mortality. The most common features of the disease are 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 US Food and Drug Administration (US FDA) approved the Vimizim licence application for the treatment of patients with Morquio A syndrome on February 14, 2014. The therapy is also approved in Australia, Canada, Brazil and the European Union. Marketing applications have been submitted in several other countries.

"The approval of Vimizim in Japan underscores our commitment to providing this much needed therapy to patients with Morquio A syndrome across the globe," said Hank Fuchs, MD, chief medical officer of BioMarin. "We will continue to seek approvals in other countries so that more patients within the MPS community have access to the treatments they deserve."

Vimizim (elosulfase alfa) is a treatment for patients with Morquio A syndrome, or mucopolysaccharidosis IVA (MPS IVA). Vimizim is the first approved enzyme replacement therapy (ERT) designed to target the underlying cause of Morquio A Syndrome—a deficiency in the enzyme N-acetylgalactosamine-6 sulfatase (GALNS). Vimizim is intended to provide the exogenous enzyme GALNS that will be taken up into the lysosomes and increase the catabolism of GAGs. Morquio A syndrome is a rare, severely debilitating and progressive disease that previously had no approved, standard-of-care treatment other than supportive care.