Indication

Risdiplam is indicated for the treatment of spinal muscular atrophy (SMA) in patients 2 months of age and older.

Important Safety Information

Interactions with Substrates of MATE Transporters

• Based on in vitro data, risdiplam may increase plasma concentrations of drugs eliminated via MATE1 or MATE2-K, such as metformin
• Avoid coadministration of risdiplam with MATE (multidrug and toxin extrusion) substrates. If coadministration cannot be avoided, monitor for drug-related toxicities and consider dosage reduction of the coadministered drug if needed

Pregnancy

• In animal studies, administration of risdiplam during pregnancy or throughout pregnancy and lactation resulted in adverse effects on development
• Based on animal data, advise pregnant women of the potential risk to the fetus. Pregnancy testing is recommended for females of reproductive potential prior to initiating risdiplam. Advise female patients of reproductive potential to use effective contraception during treatment with risdiplam and for at least 1 month after the last dose

Breastfeeding

• There is no data on the presence of risdiplam in human milk, the effects on the breastfed infant, or the effects on milk production. The developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for risdiplami and any potential adverse effects on the breastfed infant

Potential Effects on Male Fertility

• Male fertility may be compromised by treatment with risdiplam. Counsel male patients on the potential effects on fertility. Male patients may consider sperm preservation prior to treatment

Hepatic Impairment

• The safety and efficacy of risdiplam in patients with hepatic impairment have not been studied
• Because risdiplam is predominantly metabolized in the liver, hepatic impairment may potentially increase the exposures to risdiplam. Avoid use of risdiplam in patients with impaired hepatic function

Most Common Adverse Reactions

• The most common adverse reactions in later-onset SMA (incidence in at least 10% of patients treated with risdiplam and more frequent than control) were fever, diarrhea, and rash
• The most common adverse reactions in infantile-onset SMA were similar to those observed in later-onset SMA patients. Additionally, adverse reactions with an incidence of at least 10% were upper respiratory tract infection, pneumonia, constipation, and vomiting

You may report side effects to the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. You may also report side effects to Genentech at 1-888-835-2555.

Please see full Prescribing Information for additional Important Safety Information.