Roche’s Evrysdi (risdiplam) tablets have been approved by the Medicines and Healthcare products Regulatory Agency (MHRA) to treat spinal muscular atrophy (SMA).
The decision applies to patients living with SMA type 1, type 2 or type 3, or one to four SMN2 copies.
Unlike the currently available liquid version of Evrysdi, which needs to be kept in the fridge, the tablet formulation can be stored at room temperature, offering patients greater freedom and flexibility about how and where they can take the drug.
The 5mg tablets can either be swallowed whole or dispersed in water and are suitable for patients aged two years and older who weigh at least 20kg and are able to swallow without the use of a feeding tube.
The original oral solution will also remain available for those on other doses of Evrysdi and for those who may prefer the oral solution.
Affecting approximately one in every 10,000 babies worldwide, SMA is a severe and progressive neuromuscular condition characterised by muscle weakness and wasting that can significantly impact patients’ physical strength and their ability to walk, eat or breathe.
The disease is caused by a mutation of the SMN1 gene, which leads to a deficiency of the SMN protein found throughout the body.
Roche’s Evrysdi is designed to treat SMA by increasing levels of full-length functional SMN protein in the blood. Eligible patients in England, Northern Ireland and Wales can receive Evrysdi tablets under the existing managed access agreement, while those in Scotland are able to access the drug via the NHS.
The MHRA’s latest decision was based on data from a bioequivalence study, in which the 5mg tablet and original oral solution were bioequivalent, meaning patients taking the new formulation can expect the same efficacy and safety profiles as the oral solution.
Gwyn Jones, neuromuscular lead, Roche Products, said: “The marketing authorisation for the Evrysdi tablet marks a significant step forward in simplifying disease management and care for people living with SMA.”
