China National Medical Products Association Approves SPINRAZA

Biogen announced that SPINRAZA (nusinersen) has been approved by the China National Medical Products Association (NMPA) for the treatment of 5q spinal muscular atrophy (SMA), expanding the company’s presence in China. Approximately 95 percent 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 Michel Vounatsos, Chief Executive Officer at Biogen. "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.

"This is great news. 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 Pediatrics Society, Chinese Medical Association. "These improvements provide new hope to the community where there previously were no approved treatments. 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."

Register as Visitor to CPhI China 2019!

-----------------------------------------------------------------------
Editor's Note:

To apply for becoming a contributor of En-CPhI.cn,

welcome to send your CV and sample works to us,

Email: Julia.Zhang@ubmsinoexpo.com.