The Day SMA Treatment Finally Arrived
For decades, families affected by Spinal Muscular Atrophy, also known as SMA, lived without an FDA-approved treatment capable of addressing the underlying biology of this rare genetic neuromuscular disease. SMA can cause progressive muscle weakness, breathing difficulties, swallowing challenges, loss of mobility, fatigue, and increasing dependence on caregivers and medical equipment. That changed on December 23, 2016, when the U.S. Food and Drug Administration approved Spinraza, also known as nusinersen, for pediatric and adult patients with SMA. The FDA records December 23, 2016 as Spinraza’s marketing approval date, while Cure SMA describes it as the first-ever FDA-approved therapy for Spinal Muscular Atrophy.
Spinraza changed SMA treatment by targeting the backup SMN2 gene and helping the body produce more functional survival motor neuron protein, which motor neurons need to remain healthy and communicate with muscles. Years of laboratory research, clinical trials, patient participation, advocacy, and collaboration between researchers and biotechnology companies ultimately made that breakthrough possible. Spinraza began as early as 2003, and pivotal clinical development eventually demonstrated enough benefit for FDA approval in 2016. The arrival of Spinraza also transformed expectations surrounding SMA research, helping shift conversations from primarily managing decline toward preserving function, improving outcomes, treating earlier, and continuing to search for therapies that target the disease from different biological angles.
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