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SMA Market Snapshot: Future Spinraza Patent Loss Makes Room For Promising Myostatin Inhibitors

 
• By Ayisha Sharma

While spinal muscular atrophy therapies Evrysdi and Zolgensma have gained ground at the expense of pioneer Spinraza, emerging myostatin inhibitors promise a complementary mechanism that could enhance functional outcomes.  

Children With Type I SMA Do Not Often Make It Past Their Second Birthday • Source: Alamy

The reign of Biogen, Inc.’s Spinraza in spinal muscular atrophy (SMA) has been challenged by Novartis AG’ Zolgensma and Roche Holding AG’s Evrysdi, but experts say the complementary approach of myostatin inhibition could shift the treatment paradigm even further.

The SMA market took off in 2016 with the US Food and Drug Administration approval of Spinraza (nusinersen), an antisense survival motor neuron (SMN) upregulator. Subsequent approvals further validated the...

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