According to a story from Market Screener, the biotechnology company BioMarin Pharmaceutical Inc. recently announced that the Chinese government has approved the company’s drug elosulfase alfa (marketed at Vimizim) as a treatment for Morquio A syndrome, a very rare lysosomal storage disease. This is the first time that a treatment for the disease has been approved in China. This drug was first approved by the US Food and Drug Administration (FDA) in early 2014.
About Morquio Syndrome (MPS IV)
Morquio syndrome, which is also known as mucopolysaccharidosis IV (MPS IV) is a rare metabolic disorder in which the body is unable to process certain molecules in the body as a result of a genetic mutation that causes the necessary enzyme to be absent. The two type of Morquio syndrome (type A and type B) are distinguished from each other by which gene is affected. The disorder is usually inherited. Symptoms vary in severity and can include short stature, enlarged liver, dwarfism, large fingers, wide spaced teeth, heart murmurs, enlarged heart, compressed spinal cord, scoliosis, vision problems, thin tooth enamel, and joint abnormalities. The standard approach to treatment is enzyme replacement therapy. This disease can considerably shorten life span, with females generally living longer than males; respiratory failure is the leading cause of death. Incidence is about 1 in every 300,000 births in the US. To learn more about Morquio syndrome, click here.
Rare Diseases in China
MPS was included on a list of rare diseases that the government of China released in May 2018. In this list, priority was given to diseases that impose a heavy burden, are of greater prevalence, and are treatable. Later in the year the government also released a list of drugs that it considered eligible for priority review, which included Vimizim. The drug can have a significant positive impact on respiratory function, which is a major cause of death in Morquio syndrome patients.
Morquio syndrome is estimated to impact only about 3,000 people, at least in the developed world. Now, with this approval, this patient group in China will finally have access to a disease modifying treatment for the first time.