Scholar Rock Sees Phase III Path For SMA Drug
Apitegromab Being Studied As Monotherapy And In Combination
While the drug may be well-positioned for registrational development, some important caveats remain after mixed data in Phase II study cohorts.
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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 introductions of Spinraza and Zolgensma in SMA offer new insights into how to address neurodegenerative diseases. But more real world evidence is needed.
Together with a similar delay for AbbVie’s Rinvoq, the delays appear to stem from a study that raised concerns about the class. But the potential sales impact is less clear.