According to a story from BioSpace, the biopharmaceutical company CANBridge Pharmaceuticals announced that it has recently submitted a New Drug Application with China’s National Medicinal Products Administration (NMPA) for its product idursulfase beta (marketed as Hunterase) as a treatment for Hunter syndrome, a rare and often fatal genetic disorder. Hunterase is an enzyme replacement therapy that can reduce symptoms and improve patients quality of life. This disease is substantially more prevalent in Asian countries than in the west. CANBridge is committed to the development of innovative therapies for unmet medical needs in China and other parts of the world.
About Hunter Syndrome
Hunter syndrome, which is also known as mucopolysaccharidosis II (MPS II), is a rare lysosomal storage disease. This X-linked disorder is caused by mutations of the IPS gene and affects males almost exclusively. This mutation causes a deficiency of a certain enzyme which allows other substances to accumulate in the body and cause symptoms. These symptoms include ear infections, abdominal hernia, enlarged tongue, distinct facial features, developmental declines, joint stiffness, enlarged spleen and liver, heart problems, skin lesions, behavioral abnormalities, retinal degeneration, and hearing loss. Treatment for Hunter syndrome includes enzyme replacement therapy and bone marrow or stem cell transplantation; these treatments cannot address neurological symptoms. Research into gene therapy for this disease is also ongoing. There are around 2,000 people worldwide that are known to have the disorder. To learn more about Hunter syndrome, click here.
Rare Diseases in China
CANBridge successfully licensed Hunterase from GC Pharma, who was the original developer of the drug. This license gives the company control over commercialization and development in China. There is currently no approved treatment for Hunter syndrome or other forms of mucopolysaccharidosis in the country, but the government has placed this group of diseases on its “First National List of Rare Diseases” as a priority target.
About CANBridge Pharmaceuticals
The company has begun to gain recognition as a leading innovator for rare and orphan diseases in China. It has entered a partnership with another Chinese company, called WuXi Biologics, for the development and commercialization of treatments for genetic rare diseases and disorders.