Evrysdi® – BiotechReality – Information Intelligence System for Biopharma

Roche has announced that the European Commission (EC) has granted label expansion to Evrysdi® (risdiplam), launching a new, room-temperature stable 5mg tablet formulation for the treatment of spinal muscular atrophy (SMA). The tablet may be taken whole or dissolved in water and can be administered with or without food and does not need refrigeration, providing more convenience and flexibility for patients. Evrysdi is the first non-invasive, disease-modifying treatment for SMA, intended for administration once daily at home. The new tablet formulation is for patients who are two years of age and older with a minimum weight of 20kg (44 lbs) who can swallow without the need for a feeding tube. The original oral solution will be continued for those on other dosages or who prefer the liquid form. The EC approval relies on the results of a bioequivalence study proving that the tablet dosage form is equivalent in efficacy and safety to the original oral solution. The results of research published at SMA Europe's 4th Scientific International Congress in 2024 demonstrated that two dosage forms are bioequivalent, yielding uniform treatment outcomes. We welcome the development of new treatment formulations that have the potential to further simplify disease management and care for people living with SMA,, 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. Nicole Gusset About Evrysdi® (risdiplam) To treat SMA brought on by chromosome 5q mutations that result in survival of motor neuron (SMN) protein shortage, Evrysdi is a survival motor neuron 2 (SMN2) splicing modifier. Every day, at home or on the go, Evrysdi is taken either as a tablet that may be swallowed whole or dissolved in water, or as a liquid that can be taken orally or through a feeding tube. By boosting and maintaining the synthesis of SMN protein in the central nervous system and peripheral tissues, Evrysdi is intended to cure SMA. The body's SMN protein is essential for the maintenance of healthy motor neurons and other bodily processes. Evrysdi received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) in 2017 and PRIME designation from the European Medicines Agency (EMA) in 2018. Evrysdi received the Society for Medicines Research Award for Drug Discovery and the British Pharmacological Society's Drug Discovery of the Year award in 2021. More than 18,000 SMA patients are currently receiving treatment worldwide thanks to Evrysdi's approval in more than 100 countries. Source: Hoffmann-La Roche Last Modified:

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Roche’s Evrysdi® Tablet Receives European Commission Approval, Enhancing Treatment Flexibility for Spinal Muscular Atrophy Patients

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