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EMA has recommended that Evrysdi be granted marketing authorization in the EU for the treatment of patients with certain types of SMA.
The European Medicines Agency (EMA) has issued its recommendation that Evrysdi be granted marketing authorization in the European Union for the treatment of patients with certain types of spinal muscular atrophy (SMA).
According to a Feb. 26, 2021 press release, the regulatory agency’s decision has been based on two clinical studies, which investigated the effects of Evrysdi on patients with infantile-onset SMA and later-onset SMA. The trial results demonstrated a beneficial effect on the motor development and survival at 12 months in young patients when compared with the natural course of the disease.
Evrysdi is a non-invasive oral treatment that contains the active substance, risdiplam. As a part of the recommendation, EMA’s Committee for Medicinal Products for Human Use (CHMP) has asked Roche to perform a post-authorization efficacy study to further evaluate disease progression in treated patients versus untreated patients.
“Our close partnership with the SMA community has enabled the development of the first ‘at-home’ treatment for SMA in infants, children, and adults with varying levels of disease severity, the majority of whom remain untreated,” said Levi Garraway, Roche’s chief medical officer and head of Global Product Development, in a company press release issued on Feb. 26, 2021. “If approved, Evrysdi would represent a much-needed therapeutic option and we expect it to become the treatment of choice for people living with SMA and their families.”