KOL Views: What do SUNFISH data say about risdiplam’s disruptive potential in SMA?

Roche and partner PTC Therapeutics reported detailed results from Part 2 of the SUNFISH trial showing that risdiplam improvement scores on the Motor Function Measure (MFM-32) scale by a statistically significant 1.55 points at 12 months versus placebo in 180 patients. The data seem do not throw up any major hurdles to approval and yet also raise some niggly questions about how the SMN2 splicing modifier will compete in an increasingly crowded marketplace for the treatment of spinal muscular atrophy (SMA).

To provide FirstWord readers with rapid feedback on the how risdiplam may change prescribing patterns in SMA, we are hosting an expert call with a key opinion leader (KOL) next week.

Key topics that will be discussed during the call include, among other things… how are you currently deciding between Spinraza (nusinersen) from Biogen and Ionis Pharmaceuticals and Novartis' Zolgensma (onasemnogene abeparvovec); how strong are the SUNFISH results; what are the biggest remaining questions about risdiplam safety and/or efficacy; does risdiplam look better or worse relative to other SMA therapies in light of this week's SUNFISH readout; if you were on an AdCom, would you recommend approving risdiplam with a broad label for type 1, 2 and 3 patients based on available data; if approved, when would you consider reaching for risdiplam and is it likely to result in less use of one or both of Spinraza and/or Zolgensma; how much will the price of risdiplam impact prescribing decisions; do you believe that risdiplam will ultimately be used in combination or sequentially with the available medicines, and if so might that be an option soon or will additional data (from studies like JEWELFISH) be needed before this is possible?

Ask the expert!

Furthermore, we invite FirstWord Pharma readers to submit their own questions for consideration. Please click here to do so. We can't guarantee that all questions submitted will be asked due to time constraints, but we will do our utmost to cover the important issues relating to the increasingly competitive SMA treatment landscape.

We endeavour to provide feedback from KOLs as quickly as possible. Interview content will be published for FirstWord Pharma PLUS subscribers to read. To be notified when the interview content is available please click here.

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As always, FirstWord would very much like to receive your feedback and suggestions.

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