Roche recently announced that its product, Evrysdi (risdiplam), which was approved by the US Food and Drug Administration (FDA) in August 2020, was recommended for approval for the treatment of spinal muscular atrophy (SMA) in patients two month of age and older by the European Medicines Agency (EMA). GlobalData, a leading data and analytics company, forecasts that Evyrsdi will generate $1.8bn in sales globally by 2026.

Biogen’s Sprinraza and Novartis’ Zolgensma are the leading treatments for SMA, however, there is an unmet need for SMA treatment that offers a favourable route of administration.
Christie Wong, Pharma Analyst at GlobalData, comments: “Sprinraza is associated with clinical challenges due to the need for a lumbar puncture (LP) each time the drug is administered via an intrathecal route. LP is a resource-intensive procedure and one that is risky for patients who have undergone spinal fusions to remedy indication specific scoliosis. All these factors limit the patient population that can receive this treatment option.”

In an effort to address this issue, companies such as Novartis and Roche are developing therapies that are proposed to have a similar mechanism of action (MOA) as Spinraza, but intended to be administered via an oral route, including Branaplam from the former and Evrysdi from the latter. Branaplam is currently in Phase II of development in US and EU, and GlobalData expects the drug to launch in 2024 in the US.

Wong continues: “Like Sprinraza, Evyrsdi has a similar MOA and targets the survival motor neuron protein. However, it is formulated as a powder and can be administered orally. With consideration of the ongoing COVID-19 pandemic and the infant patient population, the non-invasive oral treatment Evrysdi offers has the opportunity to provide a convenient at home treatment.
“Evrysdi is expected to compete directly with Spinraza due to the fact that the drug can be administered orally. This means that utilization of institutional infrastructure and resources can be minimized, in comparison to intrathecally administered Spinraza.”