Spinraza (nusinersen) has been approved in China for the treatment of 5q spinal muscular atrophy (SMA) in a move that expands Biogen’s presence in East Asia.
Spinraza is the first approved treatment in China for SMA, a leading genetic cause of death in infants that is marked by progressive, debilitating muscle weakness.
Approximately 95% of all SMA cases are 5q SMA, making it the most common form of the disease.
The China National Medical Products Association (NMPA) evaluation of Spinraza was based on the largest clinical data set currently available in SMA – including more than 300 patients with infantile and later-onset SMA.
A global Biogen study, NURTURE, demonstrated unprecedented efficacy in treating patients pre-symptomatically.
The study included infants up to six weeks of age at time of first dose, who were genetically diagnosed with SMA and had not experienced any symptoms by the time of first dose.
Data showed that earlier treatment of pre-symptomatic infants allows for progressive gains in motor function and milestones that are more consistent with normal development.
In May 2018, the China National Health Commission included SMA in the country’s first national list of rare diseases, which was developed to support diagnosis and treatment of rare conditions.
In July of that year, the NMPA announced a priority review process that would evaluate innovative treatments using clinical evidence from trials conducted in advanced markets.
In September, Spinraza was accepted by the NMPA for priority review approval as a clinically urgent new drug that has been approved overseas for a rare disease.
“Spinraza has the largest clinical data set in SMA with findings that demonstrate its efficacy and safety in a broad range of individuals, including significant improvements in motor development and reduction in mortality in infants,” said Professor Yi Wang, head of the National Rare Diseases Group of Chinese Paediatrics Society, Chinese Medical Association.
He added: “As the first approved therapy for SMA in China, Spinraza is a breakthrough within the rare disease space. The clinical treatment for SMA is entering a new historic stage.”