As key team members in the management of spinal muscular atrophy (SMA),1physical therapists need to understand how disease-modifying therapies may impact patients with SMA and how to best assess these patients over time. Evrysdi (risdiplam) is a new disease-modifying treatment for infants (2 months and older), children, and adults with SMA.2Approved in August of 2020, Evrysdi is the first and only at-home treatment for SMA and presents a new option for patients living with this progressive neuromuscular disease.2,3This video provides a high-level review of both infantile- and later-onset SMA, with a discussion of motor scales that physical therapists might use to assess and monitor their patients with SMA. It also reviews the efficacy, safety, and administration information for Evrysdi in infants, children, and adults across SMA Types 1, 2, and 3. Presented by Vamshi K. Rao, MD, Assistant Professor of Pediatrics (Neurology and Epilepsy) at Northwestern University Feinberg School of Medicine, and Tina Duong, PT, PhD, Clinical Research Manager and Research Physical Therapist at Stanford University.
Evrysdi 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, Evrysdi may increase plasma concentrations of drugs eliminated via MATE1 or MATE2-K, such as metformin
- Avoid coadministration of Evrysdi 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
- In animal studies, administration of Evrysdi 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 Evrysdi. Advise female patients of reproductive potential to use effective contraception during treatment with Evrysdi and for at least 1 month after the last dose
- There is no data on the presence of Evrysdi 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 Evrysdi and any potential adverse effects on the breastfed infant
Potential Effects on Male Fertility
- Male fertility may be compromised by treatment with Evrysdi. Counsel male patients on the potential effects on fertility. Male patients may consider sperm preservation prior to treatment
- The safety and efficacy of Evrysdi in patients with hepatic impairment have not been studied
- Because Evrysdi is predominantly metabolized in the liver, hepatic impairment may potentially increase the exposures to Evrysdi. Avoid use of Evrysdi 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 Evrysdi 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.
- Mercuri E, Finkel RS, Muntoni F et al. Diagnosis and management of spinal muscular atrophy: Part 1: Recommendations for diagnosis, rehabilitation, orthopedic and nutritional care. Neuromuscul Disord. 2018;28:103-115.
- Evrysdi [package insert]. South San Francisco, CA: Genentech, Inc.; 2020.
- FDA approves Roche’s Evrysdi (risdiplam) for treatment of spinal muscular atrophy (SMA) in adults and children 2 months and older. News release. Roche; August 10, 2020.
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