Evrysdi Adverse Reactions

Summary of the safety profile: In infantile-onset SMA patients, the most common adverse reactions observed in Evrysdi clinical studies were pyrexia (54.8%), rash (29.0%) and diarrhoea (19.4%).
In later-onset SMA patients, the most common adverse reactions observed in Evrysdi clinical studies were pyrexia (21.7%), headache (20.0%), diarrhoea (16.7%), and rash (16.7%).
The adverse reactions previously listed occurred without an identifiable clinical or time pattern and generally resolved despite ongoing treatment in infantile-onset and later-onset SMA patients.
Based on the primary analysis of RAINBOWFISH, the safety profile of Evrysdi in pre-symptomatic patients is consistent with the safety profile of symptomatic infantile-onset and later-onset SMA patients. The RAINBOWFISH study enrolled 26 patients with pre-symptomatic SMA between 16 and 41 days of age at the time of the first dose (weight range 3.1 to 5.7 kg). The median exposure duration was 20.4 months (range: 10.6 to 41.9 months). Limited post-marketing data are available in neonates <20 days of age.
See also Pharmacology: Toxicology: Preclinical safety data under Actions for the effects of Evrysdi observed in nonclinical studies.
Tabulated list of adverse reactions: The corresponding frequency category for each adverse drug reaction is based on the following convention: very common (≥1/10), common (≥1/100 to <1/10), uncommon (≥1/1,000 to <1/100), rare (≥1/10,000 to <1/1,000), very rare (<1/10,000). Adverse drug reactions from clinical studies (Table 7) are listed by MedDRA system organ class. (See Table 7.)

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Safety profile in patients previously treated with other SMA-modifying therapies: Based on the primary analysis of the JEWELFISH study, the safety profile of Evrysdi in SMA treatment non-naive patients who received Evrysdi for up to 59 months (including those previously treated with nusinersen [n=76] or with onasemnogene abeparvovec [n=14]) is consistent with the safety profile in SMA treatment-naive patients treated with Evrysdi in the FIREFISH, SUNFISH and RAINBOWFISH studies (see Pharmacology: Pharmacodynamics under Actions).
Post-marketing experience: Cutaneous vasculitis was reported during post-marketing experience. Symptoms recovered after permanent discontinuation of Evrysdi. The frequency cannot be estimated based on available data.
Reporting of suspected adverse reactions: Reporting suspected adverse reactions after authorisation of the medicinal product is important. It allows continued monitoring of the benefit/risk balance of the medicinal product. Healthcare professionals are asked to report any suspected adverse reactions.