SMA therapy Spinraza approved in China

The China National Medical Products Association (NMPA) has approved Spinraza (nusinersen) for the treatment of 5q spinal muscular atrophy (SMA), thus paving the way to expand Biogen’s (Nasdaq: BIIB), the drug’s developer, presence in China.

Around 95% of all SMA cases are 5q SMA, making it the most common form of the disease. 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.

The 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. The NURTURE 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.

“We commend China’s regulatory authorities for their expedited review and approval of Spinraza, the first and only treatment for SMA,” said Biogen chief 3executive Michel Vounatsos, adding: “SMA is the most common genetic cause of infant mortality and a major cause of disability in adults. We are working diligently with the government agencies, patient groups and physicians who all have an incredible sense of urgency to expand access to a broad group of patients in China.”

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 2018 the NMPA announced a priority review process that would evaluate innovative treatments using clinical evidence from trials conducted in advanced markets. In September 2018 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.