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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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.
With top-line data showing efficacy in type 1 SMA patients, risdiplam may be able to obtain a label that would address significantly more patients than Novartis’ Zolgensma. Final efficacy data will show how drug’s efficacy compares to Biogen’s Spinraza.