Genentech Celebrates Success with Evrysdi: Milestone in Spinal Muscular Atrophy

The drug market for spinal muscular atrophy (SMA) is experiencing remarkable developments: The Roche-developed therapy Evrysdi (active ingredient Risdiplam) shows significant success in treating pre-symptomatic infants in a groundbreaking Phase II study called Rainbowfish. These results were recently presented at the World Muscle Society Congress 2024 in Prague and promise a paradigm shift in dealing with this genetic disorder. The Rainbowfish study observed 26 babies from birth to six weeks old who showed no symptoms of SMA. After two years of treatment, all children were able to consume food orally and manage without future respiratory support—a notable improvement compared to untreated Type 1 SMA cases, where affected children rarely survive beyond two years. Furthermore, the children exhibited cognitive development comparable to healthy infants, as measured by the Bayley Scales of Infant and Toddler Development, Third Edition. Pleasingly, this is the first clinical study in SMA research to depict cognitive abilities as an exploratory endpoint with a standardized scale. Notably, previously announced one-year data had already raised hopes: 80% of patients could sit without support, and 81% could even sit for 30 seconds. Levi Garraway, CEO of the medical division of Genentech, emphasized that early interventions, combined with infant screening programs, can significantly improve the quality of life for children with SMA. Financially, 2023 was a successful year for the drug, generating substantial revenue of CHF 1.4 billion. Besides Evrysdi, parents have other treatment options such as Biogen's intrathecal therapy Spinraza and Novartis' innovative gene therapy Zolgensma. However, Evrysdi offers a crucial advantage: It can be administered non-invasively and orally, setting it apart from its competitors.