BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that Vimizim® (elosulfase alfa) has been approved by the National Medical Products Administration (NMPA) for the treatment of patients with mucopolysaccharidosis type IVA (MPS IVA), also known as Morquio A syndrome. Vimizim is the first treatment in China approved for this condition.
In May 2018, the Chinese government issued the country’s first national list of rare diseases, which included MPS. The list also includes phenylketonuria (PKU), Tetrahydrobiopterin Deficiency, and hemophilia—diseases that BioMarin either has an approved therapy for or is developing one. The list gives priority to rare diseases with a relatively high prevalence, that pose a heavy burden and that are highly treatable. Also, in August 2018, the Chinese Drug Evaluation Center posted a list of 48 drugs already approved in the U.S., EU or Japanthat could be eligible for Priority Review in China, which included Vimizim.
“Vimizim is the first, and currently only, disease-specific treatment option for this very rare, progressively degenerative, autosomal-recessive lysosomal storage disorder,” said Luo Xiaoping, Vice Chairman of the Pediatrics society of the Chinese Medical Association and head of Endocrine Genetic Metabolism Group of Chinese Medical Association.
“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,” said Gu Xuefan, Deputy Director of Shanghai Institute of Pediatrics, Director of Pediatric Endocrinology and Inherited Metabolic Diseases Research Office of Xinhua Hospital, Director of Pediatric Genetic Diseases Diagnosis and Treatment Center, and Director of Pediatric Endocrinology and Inherited Metabolism Group of Shanghai Medical Association.
