Roche’s Evrysdi (risdiplam) tablets have been approved by the European Commission (EC) to treat spinal muscular atrophy (SMA).
The new, room-temperature stable formulation of Evrysdi may offer patients greater freedom and independence compared to the currently available oral solution, according to the company.
The 5mg tablets, which can either be swallowed whole or dispersed in water, are suitable for use in patients aged two years and older who weigh more than 20kg and are able to swallow without the use of a feeding tube.
The original formulation will remain available for patients on other doses of the drug and for those who may prefer the oral solution.
SMA is a severe and progressive neuromuscular disease that affects approximately one in every 10,000 babies worldwide. The disease is caused by a mutation of the SMN1 gene, which leads to a deficiency of SMN protein, and can significantly impact patients’ physical strength and their ability to walk, eat or breathe.
Given daily at home or on the go, 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 label extension makes Evrysdi the first and only tablet approved in the EU for SMA and was supported by 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 the new Evrysdi tablet “represents progress toward more versatile SMA disease management”, adding that the drug’s “proven efficacy, safety and convenience has significantly improved the course of disease for people living with SMA”.
Also welcoming the approval, Nicole Gusset, chief executive officer of non-profit organisation SMA Europe, said: “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.”
The EC’s decision comes four months after the US Food and Drug Administration approved Roche’s Evrysdi tablets for the same patient population.