Roche secures EU approval for convenient Evrysdi tablet to treat SMA

UK—Roche has secured European Commission approval for a new tablet formulation of its spinal muscular atrophy (SMA) treatment, Evrysdi (risdiplam), marking a significant advancement in the management of this rare neuromuscular disorder.

This 5mg tablet, which remains stable at room temperature, provides patients with a more convenient and flexible dosing option compared to the existing liquid formulation, first approved in Europe in 2021.

Unlike the oral solution that requires refrigeration and must be taken after a meal, the Evrysdi tablet can be swallowed whole or dispersed in water and taken with or without food.

This eliminates the need for refrigeration and allows for easier administration at home or on the go, enhancing patient independence and comfort.

 However, the tablet is intended for patients aged two years and older who weigh at least 20 kilograms (about 44 pounds) and can swallow without a feeding tube.

The oral solution will remain available for patients who require different dosages or who are unable to swallow tablets, including those who need feeding tube administration.

The European Commission’s approval was based on a bioequivalence study involving 131 healthy participants, which demonstrated that the tablet delivers the same efficacy and safety profile as the original oral solution.

This study was presented at SMA Europe’s 4th Scientific International Congress in 2024, confirming that patients can expect consistent treatment outcomes regardless of the formulation used.

SMA is a severe, progressive genetic disease caused by mutations in the survival motor neuron 1 (SMN1) gene, leading to a deficiency of SMN protein.

This protein is crucial for muscle control and movement; its lack results in muscle weakness that worsens over time, potentially affecting respiratory muscles and becoming fatal.

Evrysdi works by increasing the production of SMN protein through a second gene, SMN2, thereby improving muscle function throughout the central nervous system and peripheral tissues.

Roche’s Evrysdi is currently the only non-invasive, disease-modifying treatment for SMA that can be administered at home.

Over 18,000 people worldwide have been treated with Evrysdi, highlighting its growing role in SMA care.

The introduction of the tablet formulation further strengthens Roche’s market position against competitors like Biogen’s Spinraza (nusinersen), which requires spinal injections, and Novartis’ gene therapy Zolgensma, both of which have seen sales decline amid Evrysdi’s increasing adoption.

In 2024, Evrysdi generated approximately US$1.8 billion in sales, up 18% from the previous year, while Spinraza’s sales fell 10% to US$1.57 billion. Analysts forecast Evrysdi’s sales to reach US$2.9 billion by 2031.

Nicole Gusset, CEO of SMA Europe, a non-profit umbrella organisation for SMA patient groups, welcomed the new tablet formulation, emphasising the importance of treatment options that simplify daily disease management and improve quality of life for patients and caregivers.

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