Roche has won approval in Europe for a tablet formulation of its spinal muscular atrophy (SMA) treatment Evrysdi (risdiplam), as the drugmaker looks to extend its growing dominance in the neuromuscular disorder market.
The new 5mg tablet approved by the European Commission (EC) is room-temperature stable and is set to offer another administration option for patients with SMA. Until now, Evrysdi was only available as a liquid formulation, originally approved in Europe in 2021.
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Unlike the liquid, the tablet does not need to be taken after a meal, offering patients more flexible dosing. When stored at room temperature, it does not refrigerate and can be swallowed whole or dispersed in water.
The EC approval was based on a bioequivalence study (NCT04718181) in 131 healthy participants that demonstrated the tablet performed just as well as the existing oral solution. Roche said that SMA patients can expect the same efficacy and safety from the new form of Evrysdi.
While the tablet offers a more convenient option, it will not be available for all SMA patients. Those eligible must be aged two and above, weigh at least 20kg, and be able to swallow without a feeding tube. The oral solution, which can be administered via a feeding tube, will remain available for patients who require it, along with those on doses other than 5mg.
SMA is a genetic disease with severe and progressive neuromuscular effects. It is caused by a mutation of the survival motor neuron 1 (SMN1) gene, which leads to a deficiency of SMN protein. This protein is essential in muscle control and movement – without it, muscle weakness builds over time. The disease can be fatal if the respiratory muscles are affected.
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By GlobalDataEvrysdi, which was developed as part of a collaboration between Roche, US-based PTC Therapeutics, and the SMA Foundation, works by increasing the production of SMN protein via a second SMN gene, leading to functional levels throughout the central nervous system and peripheral tissues.
“We welcome the development of new treatment formulations that have the potential to further simplify disease management and care for people living with SMA,” said Nicole Gusset, CEO of SMA Europe, a non-profit umbrella organisation of SMA patient organisations.
“This is a disease requiring daily management, and it is paramount that people living with SMA, and those who care for them, are given options to optimise treatment administration.”
Roche’s market lead grows
The tablet form doubles down on Roche’s market advantage of offering the only non-invasive treatment specifically approved for SMA. Biogen’s Spinraza (nusinersen) became the first approved treatment for SMA in Europe in 2017. Despite early success, the antisense oligonuceltotide – administered via an injection into the spinal canal – has seen sales weaken in the wake of Evrysdi’s more convenient formulation.
Spinraza still managed to clock up $1.57bn worth of sales in 2024, though this was down 10% from the $1.74bn generated in 2023. In comparison, Roche’s Evrysdi’s grew 18% to around $1.8bn in full-year sales in 2024. Analysis by GlobalData predicts that the fast growth for the drug will continue, with sales forecast to reach $2.9bn by 2031.
GlobalData is the parent company of Pharmaceutical Technology.
It has been the same story for the third approved product for SMA – Novartis’ gene therapy Zolgensma. This treatment’s financial performance has also suffered amid pressure from Evrysdi, peaking at sales $1.4bn in 2022 and declining in the years since.
