KOL Views: Sizing up the bogeys on Spinraza's radar in SMA

Spinraza (nusinersen) has helped revolutionise treatment of spinal muscular atrophy (SMA) and to the benefit of patients – but chagrin of Biogen and Ionis Pharmaceuticals – multiple differentiated competitors are drawing nearer to market. The first of these is Novartis' gene therapy, Zolgensma (onasemnogene abeparvovec), which could be approved later this month, followed by Roche and PTC Therapeutics' oral risdiplam, which is in Phase III testing.

To provide FirstWord readers with rapid feedback on how the newer therapies may change how SMA is treated and Spinraza is used, 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 do the risk/benefit profiles of Spinraza, Zolgensma and risdiplam stack relative to one another; how would the commercial availability of Zolgensma change how you treat patients with SMA; how sensitive will your prescribing patterns be to Zolgensma's price; would you be willing to compromise at all on efficacy for the oral convenience of risdiplam; do you expect that all three will eventually be approved for SMA types 1, 2 and 3; if all three make it to market, roughly what proportion of new patients would you start on each; how optimistic are you about combining more than one of the nagents and do you foresee this being feasible given economic realities?

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 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.

To ensure access to all KOL Views content, click here to upgrade your FirstWord Pharma service or contact us to discuss your market intelligence needs.

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

To read more KOL Views articles, click here.