In its most severe form, spinal muscular atrophy is usually fatal before the age of two. Zolgensma, which was proven powerfully beneficial in clinical testing, won approval in Europe in 2020, one year after its clearance in the U.S.
Testing showed Zolgensma can keep infants alive for longer and help them reach developmental milestones, such as sitting upright without support, that they otherwise would be unlikely to achieve.
In the U.S., Zolgensma already carries a “black box” warning for acute serious liver injury and acute liver failure. After the two deaths last August, Novartis notified the FDA and regulators in other countries where the drug is used. The company defended its treatment, noting that it remains confident in the drug’s risk/benefit profile.
