Following the news that Biogen will initiate a new clinical trial DEVOTE that will evaluate higher doses of Spinraza;

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

“The results of this trial, if positive, are likely to help bolster treatment rates for a broad SMA population; this implication is likely to be most pronounced in patients with late-onset SMA. According to GlobalData’s recent report, ‘Spinal Muscular Atrophy (SMA): Opportunity Analysis and Forecasts to 2028, the treatment rates for type 4 SMA patients in the seven major markets (7MM*) were found to be the lowest compared to other sub-types of SMA.

“If the new trial from Biogen is able to establish the efficacy and safety of higher-dose Spinraza, then this will likely result in more adult patients to opt for Spinraza as well continue building physicians’ and payers’ confidence in this therapy. It is worth noting that this patient population is currently an unchartered territory for Novartis’ Zolgensma due to a lack of studies evaluating this therapy in such patients.

“However, the results of the trial for other sub-types of SMA, particularly those with early-onset may be less pronounced for a number of reasons. While Spinraza has the advantage of being first-to-market approved for all types of SMA and continues to build on real-world safety and efficacy data, Zolgensma’s prospect for pediatric patients less than two years of age is one that excited many key opinion leaders (KOLs) interviewed by GlobalData; although many voiced their concerns regarding the long-term safety and efficacy potential of this curative gene therapy. While it’s recent FDA approval is a testimony to the potential of Zolgensma in the treatment of SMA, macro factors such as its high-price and reimbursement mechanisms seems to be slowing its uptake among the patient population.

“Despite these barriers, there is no denying that a significant factor for an SMA therapy to establish itself as a market leader is to become first-line treatment for the most severe sub-types of SMA, including type 1 and type 2 SMA which collectively account for 69.4% of all diagnosed prevalent in the 7MM, as per GlobalData’s report.

“Even if positive results from this new trial are able to increase Spinraza’s uptake among late-onset SMA patients, the fact that this sub-type of SMA represents a much lesser population base means that Zolgensma is likely to establish itself as the market leader in the long-term with expected peak year sales of $3bn.”

*7MM = US, France, Germany, Italy, Spain, UK and Japan