Yesterday, the FDA announced that it has approved Spinraza (Nusinersen) to treat spinal muscular atrophy, making it the first-ever FDA-approved therapy for SMA, a product which addresses the underlying genetic cause of the condition by making the back-up gene, SMN2, produce more functional SMN protein. This milestone has been achieved as a result of the cooperation between all stakeholders: researchers, families & groups, companies and regulators, all working together as one community.
The approval from the FDA for all SMA (paediatric and adult), is the broadest possible label and has happened as a result of Biogen’s rigorous clinical development plan for Spinraza (Nusinersen).
Here in Europe, we eagerly await the decision from the European Medicines’ Agency (EMA).
Please read Biogen’s press release!