Risdiplam: First Approval
online resourceposted on 2020-09-27, 21:04 authored by Sohita Dhillon
Funding The preparation of this review was not supported by any external funding.
Authorship and Conflict of interest Sohita Dhillon is a contracted employee of Adis International Ltd/Springer Nature, and declares no relevant conflicts of interest. All
authors contributed to the review and are responsible for the article content.
Additional information about this Adis Drug Review can be found here.
AbstractRisdiplam (Evrysdi™) is an orally administered, survival motor neuron 2 (SMN2)-directed RNA splicing modifier being developed by Roche, PTC Therapeutics Inc and the SMA Foundation for the treatment of the spinal muscular atrophy. The small molecule is designed to treat spinal muscular atrophy caused by mutations in chromosome 5q leading to SMN protein deficiency. The drug boosts the ability of an alternative gene SMN2 to produce full-length and functional SMN protein. In August 2020, Evrysdi™ (risdiplam) received its first approval in the USA for the treatment of spinal muscular atrophy in patients 2 months of age and older. Risdiplam is in pre-registration for this indication in numerous countries worldwide, including the European Union, Brazil, Chile, China, Indonesia, Russia, South Korea and Taiwan. This article summarizes the milestones in the development of risdiplam leading to this first approval for spinal muscular atrophy.