Positive FIREFISH trial results to bolster risdiplam as favored treatment option for SMA patients

Following the news that Roche reported positive results from Part 2 of the pivotal FIREFISH trial for spinal muscular atrophy (SMA) patients;

Vinie Varkey, Senior Analyst of Neurology & Ophthalmology at GlobalData, a leading data and analytics company, offers her view on the implications of this trial for the SMA market:

“From a mechanistic point of view, risdiplam works similarly to Biogen’s Spinraza which was the first-to-market therapy for SMA; both of them are understood to modulate the splicing of the defective gene SMN2. Risdiplam has the added advantage in that it is being developed for use as an oral agent. Key opinion leaders interviewed by GlobalData pointed out that an oral route of administration will be received with much more enthusiasm compared to Spinraza which needs to be administered via an intrathecal route.

“From a cost perspective, it is hard to see any new agents or therapies surpassing the $2.1m tag set by Novartis’ gene therapy Zolgensma. Nonetheless, Roche recently announced their plan to launch risdiplam at a price point that will help them win more patients from other treatment options and ensure a predictable market access outcome. As the third-to-market treatment option for SMA, all these developments for risdiplam weighs heavily in favor of its use and quick uptake in the market.

“According to GlobalData, the *SMA market is expected to grow at a compound annual growth rate of 16% to reach a global value of US$6.6bn in 2028, with the top three drugs by sales anticipated to be Zolgensma, Spinraza, and risdiplam, respectively.

“For a rare disease such as SMA, risdiplam exhibits a favorable profile in terms of its mechanism of action, route of administration, and anticipated cost of therapy. All these factors are expected to bolster its standing as a strong treatment option for SMA patients.”

*Information taken from GlobalData’s report: Spinal Muscular Atrophy (SMA): Opportunity Analysis and Forecasts to 2028

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