Evrysdi

Treatment with risdiplam should be initiated by a physician with experience in the management of SMA. Posology The recommended once daily dose of risdiplam is determined by age and body weight (see Table 1). Table 1. 25 mg/kg ≥ 2 years of age (≥ 20 kg) 5 mg * based on corrected age for preterm infants 3 There is an alternative film-coated tablet dosage form available for patients ≥ 2 years of age with ≥ 20 kg body weight.

Refer to the Evrysdi film-coated tablet summary of product characteristics (SmPC). The physician should prescribe the appropriate pharmaceutical form according to the dose required and the patient’s needs, including the patient’s ability to swallow.

For patients with difficulty swallowing a whole tablet or who require administration via a nasogastric or gastrostomy tube, the film-coated tablet can be dispersed in water, or the powder for oral solution can be prescribed. Treatment with a daily dose above 5 mg has not been studied.

Delayed or missed doses If a planned dose is missed, it should be administered as soon as possible if still within 6 hours of the scheduled dose. Otherwise, the missed dose should be skipped and the next dose should be administered at the regularly scheduled time the next day.

If a dose is not fully swallowed or vomiting occurs after taking a dose of risdiplam, another dose should not be administered to make up for the incomplete dose. The next dose should be administered at the regularly scheduled time. 2).

Renal impairment Risdiplam has not been studied in this population. 2). Hepatic impairment No dose adjustment is required in patients with mild or moderate hepatic impairment. 2). Paediatric population No data on risdiplam pharmacokinetics are available in patients less than 16 days of age.

Method of administration Oral use. g. pharmacist) prior to being dispensed. It is recommended that a healthcare professional (HCP) discuss with the patient or caregiver how to prepare the prescribed daily dose prior to administration of the first dose.

Evrysdi is taken orally once a day with or without food at approximately the same time each day, using the re-usable oral syringe provided. Evrysdi should not be mixed with milk or formula milk. Evrysdi should be taken immediately after it is drawn up into the oral syringe.

4%). 7%). The adverse reactions listed above occurred without an identifiable clinical or time pattern and generally resolved despite ongoing treatment in infantile‑onset and later‑onset SMA patients. 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), not known (cannot be estimated from the available data).

Adverse drug reactions from clinical studies (Table 2) are listed by MedDRA system organ class. 7 Table 2. Adverse drug reactions occurring in patients with infantile-onset and later-onset SMA based on risdiplam clinical studies and postmarketing experience System Organ Class Infantile-onset SMA (Type 1) Later-onset SMA (Type 2 and 3) Infections and infestations Urinary tract infection (including cystitis) Common Common Nervous system disorders Headache Not applicable Very common Gastrointestinal disorders Diarrhoea Very common Very common Nausea Not applicable Common Mouth ulcerations and aphthous ulcers Common Common Skin and subcutaneous tissue disorders Rash* Very common Very common Cutaneous vasculitis** Not known Musculoskeletal and connective tissue disorders Arthralgia Not applicable Common General disorders and administration site conditions Pyrexia (including hyperpyrexia) Very common Very common *Includes dermatitis, dermatitis acneiform, dermatitis allergic, erythema, folliculitis, rash, rash erythematous, rash maculo-papular, rash papular **Cutaneous vasculitis was reported during post‑marketing experience.

Symptoms recovered after permanent discontinuation of risdiplam. The frequency cannot be estimated based on available data. Safety profile in pre-symptomatic 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.

3). 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.

6). 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 risdiplam. 3). The effects of risdiplam on male fertility have not been investigated in humans.

97 mg per mL). Patients with rare hereditary problems of fructose intolerance should not take this medicine. 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).

e. is essentially ‘sodium-free’.

1.