An estimated 30 to 40 thousand people with spinal muscular atrophy will soon have access to the first-ever effective treatment for this life-limiting disorder as China’s health authorities granted marketing authorisation to nusinersen (Spinraza®).

Normally, China’s law requires that medicines are first trialled in China in order to receive approval. As last year China made combating rare diseases a national priority, since summer 2018 approvals for orphan drugs can be issued solely on the basis of foreign data.

Nusinersen’s approval was based on a large set of such international data gathered from over 300 participants of Spinraza clinical trials conducted worldwide.

The expedited approval came as China pledged to dedicate significant financial resources to facilitate access to therapies in rare diseases.