23 December 2016 - The U.S. FDA today approved Spinraza (nusinersen), the first drug approved to treat children and adults with spinal muscular atrophy, a rare and often fatal genetic disease affecting muscle strength and movement. 

Spinraza is an injection administered into the fluid surrounding the spinal cord.

The FDA granted this application fast track designation and priority review. The drug also received orphan drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.

The sponsor is receiving a rare paediatric disease priority review voucher under a program intended to encourage development of new drugs and biologics for the prevention and treatment of rare paediatric diseases. A voucher can be redeemed by a sponsor at a later date to receive priority review of a subsequent marketing application for a different product. This is the eighth rare paediatric disease priority review voucher issued by the FDA since the program began.

Read FDA press release