Roche Uses 5-Year Evrysdi Data in SMA Battle with Spinraza, Zolgensma

Roche has revealed five-year data demonstrating the continued efficacy and favorable safety profile of its oral spinal muscular atrophy (SMA) medication, Evrysdi (risdiplam). This development enables Roche to maintain competitive pressure on Biogen’s Spinraza (nusinersen) and Novartis’ gene therapy Zolgensma (onasemnogene abeparvovec).

Levi Garraway, Roche's chief medical officer, emphasized that the conclusion of the FIREFISH study has provided extensive insights and data, solidifying Evrysdi as a critical treatment option for SMA. Evrysdi received its initial approval in 2020 for treating SMA in adults and children aged two months and older, based on data from the FIREFISH and SUNFISH trials. In 2022, the approval was extended to include infants younger than two months.

The latest findings from the extension of the FIREFISH study reveal that after five years of Evrysdi treatment, 91% of children with Type 1 SMA—a severe and infantile-onset form—were alive. Historically, these patients would not have been expected to live beyond two years of age. Furthermore, 81% of these children were surviving without the need for permanent ventilation. Significant motor milestone achievements were also noted, with 59% of participants able to sit without support for 30 seconds, in addition to gains in standing and walking abilities. Beyond improvements in mobility, 96% of the children could swallow, and 80% could feed orally without the need for a feeding tube. Safety data were promising, showing no treatment discontinuations due to adverse events and a 66% reduction in overall event rates between the first and fifth years of treatment.

Industry experts recognize potential for Evrysdi to establish a strong presence in the SMA treatment landscape. According to Baird analysts, the SMA market is crowded yet lucrative. They noted that ultra-rare, progressive, and fatal diseases often offer substantial revenue opportunities for multiple products. However, the emergence of three highly effective treatments—Spinraza, Zolgensma, and Evrysdi—in quick succession is likely to continue pressurizing the pioneering therapy, Spinraza.

Spinraza was the first to be approved by the FDA in 2016, followed by Zolgensma over two years later. In 2023, Biogen’s Spinraza generated $1.7 billion in sales, while Evrysdi brought in CHF 1.4 billion ($1.6 billion), and Novartis recorded $1.2 billion from Zolgensma.

Baird analysts anticipate that while Evrysdi will exert "moderate pressure" on Spinraza's market dominance, the Biogen drug is expected to continue providing substantial revenue over the next decade. They also highlighted Spinraza as "one of the most effective drugs we have ever seen," despite the competitive landscape.

In summary, Roche's Evrysdi has shown sustained efficacy and safety over five years, with impressive survival and motor function improvements in children with Type 1 SMA. As the SMA treatment market evolves with multiple effective therapies, Evrysdi is positioning itself as a significant competitor to established treatments like Spinraza and Zolgensma.