FDA Approves Roche's Evrysdi as First Tablet for Spinal Muscular Atrophy

Roche announced that the U.S. Food and Drug Administration (FDA) has approved a New Drug Application (NDA) for an Evrysdi® (risdiplam) tablet for people living with spinal muscular atrophy (SMA). Evrysdi is the only non-invasive disease-modifying treatment for SMA. The 5 mg Evrysdi tablet can either be swallowed whole or dispersed in water.
""Evrysdi has robust potential to modify the SMA disease trajectory, and has already been used to treat thousands of patients to date. This approval marks another significant step forward,"" said Levi Garraway, M.D., Ph.D., Chief Medical Officer and Head of Global Product Development, Roche. ""The Evrysdi tablet combines established efficacy with convenience, providing an additional flexible option for SMA management.""

The approval of the Evrysdi tablet was based on the results of a bioequivalence study, which demonstrated that the 5 mg tablet, whether swallowed whole or dispersed in non-chlorinated drinking water (e.g., filtered water), and original oral solution provide comparable exposure to risdiplam. This means patients who take the tablet can expect the same established efficacy and safety as the Evrysdi oral solution. The Evrysdi oral solution will remain available for those on other doses of Evrysdi and for those who may prefer the oral solution.

""We cannot underestimate the value that comes with simplifying treatment administration and disease management for people who are living with SMA or those caring for them,"" said Kenneth Hobby, President of Cure SMA. ""This new room temperature stable formulation option offers an additional choice that may more conveniently fit into daily living activities such as working, traveling, and education.""

As part of the label extension, the Evrysdi prescribing information has been updated to include guidance on tablet administration and storage.

The new tablet, expected to be available in the coming weeks, is suitable for people two years of age or older who weigh more than 20 kgs (44 lbs).

Roche leads the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics.

About Evrysdi® (risdiplam)
Evrysdi is a survival motor neuron 2 (SMN2) pre-mRNA splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to survival of motor neuron (SMN) protein deficiency. Evrysdi is administered daily at home or on the go, either in liquid form (by feeding tube or by mouth) or in the form of a tablet (by mouth only, swallowed whole or dispersed in water).
Evrysdi is designed to treat SMA by increasing and sustaining the production of SMN protein in the central nervous system (CNS) and peripheral tissues. SMN protein is found throughout the body and is critical for maintaining healthy motor neurons and core functions.

Evrysdi was granted PRIME designation by the European Medicines Agency (EMA) in 2018 and Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) in 2017. In 2021, Evrysdi was awarded Drug Discovery of the Year by the British Pharmacological Society as well as the Society for Medicines Research Award for Drug Discovery. Evrysdi is currently approved in more than 100 countries, with more than 16,000 people with SMA treated globally.

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