Potential embryo-foetal toxicity: Embryo-foetal toxicity has been observed in animal studies (see Pharmacology: Toxicology: Preclinical safety data under Actions). Patients of reproductive potential should be informed of the risks and must use highly effective contraception during treatment and until at least 1 month after the last dose in female patients, and 4 months after the last dose in male patients. The pregnancy status of female patients of reproductive potential should be verified prior to initiating Evrysdi therapy (see Use in Pregnancy & Lactation).
Potential effects on male fertility: Based on observations from animal studies, male patients should not donate sperm while on treatment and for 4 months after the last dose of Evrysdi. Prior to initiating treatment, fertility preservation strategies should be discussed with male patients of reproductive potential (see Use in Pregnancy & Lactation and Pharmacology: Toxicology: Preclinical safety data under Actions). The effects of Evrysdi on male fertility have not been investigated in humans.
Retinal toxicity: The effects of Evrysdi on retinal structure observed in the non-clinical safety studies have not been observed in clinical studies with SMA patients. However, long-term data are still limited. The clinical relevance of these nonclinical findings in the long-term has therefore not been established (see Pharmacology: Toxicology: Preclinical safety data under Actions).
Use with SMA gene therapy: Efficacy data of Evrysdi treatment when used in patients that previously received SMN1 gene therapy is not available.
Excipients: Isomalt: Evrysdi contains isomalt (2.97 mg per mL). Patients with rare hereditary problems of fructose intolerance should not take this medicine.
Sodium: Evrysdi contains 0.375 mg of sodium benzoate per mL. Sodium benzoate may increase jaundice (yellowing of the skin and eyes) in newborn babies (up to 4 weeks old).
Evrysdi contains less than 1 mmol sodium (23 mg) per 5 mg dose, i.e. is essentially 'sodium-free'.
Effects on ability to drive and use machines: Evrysdi has no or negligible influence on the ability to drive and use machines.