Molecular mass: 401.474 g/mol
Risdiplam interacts in the following cases:
The effect of co-administration of risdiplam on the pharmacokinetics of MATE1 and MATE2-K substrates in humans is unknown. Based on in vitro data, risdiplam may increase plasma concentrations of medicinal products eliminated via MATE1 or MATE2-K, such as metformin. If coadministration cannot be avoided, drug-related toxicities should be monitored and dosage reduction of the co-administered medicinal product should be considered if needed.
Patients with severe hepatic impairment have not been studied and may have increased risdiplam exposure.
Male fertility may be compromised while on treatment, based on nonclinical findings. In rat and monkey reproductive organs, sperm degeneration and reduced sperm numbers were observed. Based on observations from animal studies, the effects on sperm cells are expected to be reversible upon discontinuation of risdiplam.
Male patients may consider sperm preservation prior to treatment initiation or after a treatment-free period of at least 4 months. Male patients who wish to father a child should stop treatment for a minimum of 4 months. Treatment may be re-started after conception.
There are no data from the use of risdiplam in pregnant women. Studies in animals have shown reproductive toxicity. Risdiplam is not recommended during pregnancy and in women of childbearing potential not using contraception.
It is not known whether risdiplam is excreted in human breast milk. Studies in rats show that risdiplam is excreted into milk. As the potential for harm to the breastfed infant is unknown, it is recommended not to breastfeed during treatment.
Male and female patients of reproductive potential should adhere to the following contraception requirements:
The pregnancy status of female patients of reproductive potential should be verified prior to initiating risdiplam therapy. Pregnant women should be clearly advised of the potential risk to the foetus.
Male fertility may be compromised while on treatment, based on nonclinical findings. In rat and monkey reproductive organs, sperm degeneration and reduced sperm numbers were observed. Based on observations from animal studies, the effects on sperm cells are expected to be reversible upon discontinuation of risdiplam.
Male patients may consider sperm preservation prior to treatment initiation or after a treatment-free period of at least 4 months. Male patients who wish to father a child should stop treatment for a minimum of 4 months. Treatment may be re-started after conception.
Based on nonclinical data, an impact of risdiplam on female fertility is not expected.
Risdiplam has no or negligible influence on the ability to drive and use machines.
In infantile-onset SMA patients, the most common adverse reactions observed in risdiplam 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 risdiplam clinical studies were pyrexia (21.7%), headache (20.0%), diarrhoea (16.7%), and rash (16.7%).
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.
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 are listed by MedDRA system organ class.
Adverse drug reactions occurring in patients with infantile-onset and later-onset SMA based on risdiplam clinical studies:
| System Organ Class | Infantile-onset SMA (Type 1) | Later-onset SMA (Type 2 and 3) |
|---|---|---|
| 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 |
| Nervous system disorders | ||
| Headache | Not applicable | Very common |
| General disorders and administration site conditions | ||
| Pyrexia (including hyperpyrexia) | Very common | Very common |
| Infections and infestations | ||
| Urinary tract infection (including cystitis) | Common | Common |
| Musculoskeletal and connective tissue disorders | ||
| Arthralgia | Not applicable | Common |
* Includes dermatitis, dermatitis acneiform, dermatitis allergic, erythema, folliculitis, rash, rash erythematous, rash maculo-papular, rash papular
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