Spinal muscular atrophy (SMA) is a rare and severe neuromuscular disorder. It is a leading genetic cause of mortality in infants under the age of two. No medication was available until the first disease modifying treatment SPINRAZA was developed in 2016. The drug was approved in China in 2019 and was included in the National Reimbursement Drug List (NRDL) at the end of 2021.
This move has improved access to and availability of the drug, resulting in a significant rise in the treatment rate. China became the No.1 market for SPINRAZA four months after the NRDL inclusion.