Evrysdi tablets approved as SMA treatment in Europe
Format designed to offer patients flexibility, convenience
The European Commission (EC) has approved a tablet formulation of spinal muscular atrophy (SMA) treatment Evrysdi (risdiplam).
“The new Evrysdi tablet with its flexible administration represents progress toward more versatile SMA disease management,” Levi Garraway, MD, PhD, chief medical officer and head of global product development at Roche, said in a company press release.
People with SMA ages 2 and older who weigh at least 20 kg (about 44 pounds) and are able to swallow independently will be able to use the tablet version of Evrysdi across the European Union. A liquid formulation of Evrysdi is available in the EU for SMA patients of all ages.
The U.S. Food and Drug Administration (FDA) in February approved the tablet formulation for use in the U.S. Evrysdi tablets are stable at room temperature, whereas the liquid version requires refrigeration for long-term storage. The tablets, which are 5 mg and approximately 6.5 mm across, can be swallowed whole or dissolved in water.
Oral solutions of Evrysdi will still be available for those who have feeding tubes, require different doses, or prefer the liquid formulation. The tablet option will give people with SMA greater freedom, according to Roche and its subsidiary Genentech.
Correcting gene splicing
SMA is caused chiefly by mutations in SMN1, a gene that provides the instructions to make the SMN protein. Low levels of SMN lead to degeneration of motor neurons, which control voluntary movement, ultimately resulting in SMA symptoms.
SMN2, a different gene, also contains instructions to make SMN. However, a process called alternative splicing prevents SMN2 from producing high levels of working SMN. Evrysdi works by correcting splicing of SMN2, which has the potential to increase levels of functional SMN. It is the only approved therapy that non-invasively targets the underlying causes of the disease, Roche noted.
“With over 18,000 people treated to date, Evrysdi’s proven efficacy, safety and convenience has significantly improved the course of disease for people living with SMA,” Garraway said.
The EC’s decision follows a Phase 1 clinical trial (NCT04718181) that established the bioequivalence of the liquid and tablet forms of the medication. This means that people taking the tablet can expect the same efficacy and safety of the oral solution, according to Roche.
“We welcome the development of new treatment formulations that have the potential to further simplify disease management and care for people living with SMA,” said Nicole Gusset, PhD, CEO of SMA Europe, an umbrella non-profit of patient organizations from across Europe. “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.”
Evrysdi was developed by Roche and Genentech, in collaboration with the SMA Foundation and PTC Therapeutics.
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