The SMA Market: Assessing The Unknowns
Treatment Progress Leads To New Challenges In Spinal Muscular Atrophy
The introductions of Spinraza and Zolgensma in SMA offer new insights into how to address neurodegenerative diseases. But more real world evidence is needed.
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While the drug may be well-positioned for registrational development, some important caveats remain after mixed data in Phase II study cohorts.
Risdiplam’s efficacy across two clinical trials and oral dosing made it a credible threat before its US FDA approval, but its weight-based pricing falls below Biogen’s Spinraza and Novartis’s Zolgensma.
The introduction of gene therapies has been the highest-profile new modality to reach the market, but new RNA-based therapeutics hold significant potential and could be more accessible for patients. In Vivo reviews the leading drug developers in this space.