According to a publication from BioPortfolio, China’s National Medical Products Administration (NMPA — similar to the American FDA) recently approved BioMarin’s mucopolysaccharidosis type IVA drug Vimizim for use in the People’s Republic.
Last year, PRC leadership published a list of a number of rare diseases with no approved treatments in China. Chinese leadership promised accelerated review for drugs treating conditions on the list — Vimizim (approved for use in the United States back in 2014) represents one of the first rare disease drugs to receive accelerated approval in such a fashion.
About Mucopolysaccharidosis Type IVA (Morquio A Syndrome)
Mucopolysaccharidosis is a group of disorders characterized by the body’s inability to break down long sugar molecules called glycosaminoglycans. These glycosaminoglycans, essentially long chains of sugar molecules, can accumulate throughout the body in a variety of tissues — including blood, cells, and connective tissue.
The effects can be devastating if left untreated. MPS patients are more likely to develop heart disease, hearing and vision loss, or difficulty breathing. Estimates suggest that as many as one in between 200,000 and 300,000 births may be affected by Morquio A — though reliable figures aren’t available. These individuals often do not live beyond their 20s.
China Seeks to Expand Available Rare Disease Treatments
In a bid to improve the health of its citizens, and doubtlessly to advance its own healthcare infrastructure, China announced last year that it would offer expedited review of dozens of rare disease drugs that were available abroad, but not in the PRC.
Already the world’s second-largest drug market, China’s efforts to introduce new medications for currently untreated diseases suggest a larger effort to beef up the country’s competitiveness in the pharmaceutical industry. The accelerated review process, in addition to accelerated market approval for drugs offering “significant clinical value,” also offers clinical approval for generic drugs whose reference product is still patent protected for another three years (or fewer) and drugs under similar review in the United States and Europe.
By explicitly offering accelerated approval on the basis of pre-existing European and American research, China is taking advantage of its current technological deficit — it’s easier, and generally far more cost effective, to wait for someone to develop a new treatment and then acquire it rather than develop said treatment from the ground-up yourself.
Do you think China’s policy would be effective in other major nations? What might the drawbacks of such a program be? Share your thoughts with Patient Worthy!
