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Risdiplam: First Approval

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Abstract

Risdiplam (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.

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Acknowledgements

During the peer review process, the manufacturer of risdiplam was also offered an opportunity to review this article. Changes resulting from comments received were made on the basis of scientific and editorial merit.

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The preparation of this review was not supported by any external funding.

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  1. Springer Nature, Mairangi Bay, Private Bag 65901, Auckland, 0754, New Zealand

    Sohita Dhillon

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  1. Sohita Dhillon
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Correspondence to Sohita Dhillon.

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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.

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for this AdisInsight Report can be found at https://doi.org/10.6084/m9.figshare.12960173.

This profile has been extracted and modified from the AdisInsight database. AdisInsight tracks drug development worldwide through the entire development process, from discovery, through pre-clinical and clinical studies to market launch and beyond.

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Dhillon, S. Risdiplam: First Approval. Drugs 80, 1853–1858 (2020). https://doi.org/10.1007/s40265-020-01410-z

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