Roche has announced that a tablet formulation of its spinal muscular atrophy (SMA) treatment Evrysdi (risdiplam) has been approved by the US Food and Drug Administration (FDA).
Evrysdi was originally authorised by the US regulator in 2020 to treat the progressive neuromuscular disease in patients aged two months and older, before being granted expanded approval in 2022 for infants aged under two months.
Until now, the drug has only been available as an oral solution, administered daily at home by mouth or feeding tube.
The new 5mg tablet is expected to be available in the coming weeks and is suitable for use in patients aged two years and older who weigh more than 20kg, Roche said, adding that the original formulation will remain available for patients on other doses of the drug and for those who may prefer the oral solution.
It is hoped that the tablets, which can be stored at room temperature and either swallowed whole or dispersed in water, will provide more freedom and independence for patients.
SMA affects approximately one in every 10,000 babies worldwide and is caused by a mutation of the SMN1 gene, which leads to a deficiency of SMN protein. Depending on the form of the disease, patients’ physical strength and their ability to walk, eat or breathe can be significantly diminished or lost.
Roche’s Evrysdi is designed to treat SMA by increasing and sustaining the production of SMN protein in the central nervous system and peripheral tissues.
The FDA’s latest decision on Evrysdi, which makes it the first and only tablet approved for SMA, was based on results from a bioequivalence study demonstrating that the 5mg tablet and original oral solution provided comparable exposure to the drug.
Levi Garraway, chief medical officer and head of global product development at Roche, said: “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.
“The Evrysdi tablet combines established efficacy with convenience, providing an additional flexible option for SMA management.”