Managing SMA Across a Range of Patient Types: An Expert Forum


Evrysdi is indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.


Introduction: Understanding SMA

Discussion: Conveying the Importance and Urgency of SMA Treatment

Evrysdi® (risdiplam) Important Safety Information

Overview: Evrysdi Clinical Trial Program

Discussion: Setting Treatment Expectations and Goals

Data and Discussion: Evrysdi for Patients with Later-Onset SMA

Data and Discussion: Evrysdi for Presymptomatic Newborns with SMA

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
Pregnancy & Breastfeeding
  • Evrysdi may cause embryofetal harm when administered to a pregnant woman.In animal studies, administration of Evrysdi during pregnancy and/or lactation resulted in adverse effects on development. Advise pregnant women of the potential risk to the fetus
  • Pregnancy testing is recommended prior to initiating Evrysdi. Advise female patients to use contraception during treatment with Evrysdi and for at least 1 month after the last dose
  • There is a pregnancy exposure registry that monitors pregnancy and fetal/neonatal/infant outcomes in women exposed to Evrysdi during pregnancy. Physicians are encouraged to register patients and pregnant women are encouraged to register themselves by calling 1-833-760-1098 or visiting
  • 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
  • Counsel male patients that fertility may be compromised by treatment with Evrysdi. Male patients may consider sperm preservation prior to treatment
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 (including nasopharyngitis, rhinitis), lower respiratory tract infection (including pneumonia, bronchitis), constipation, vomiting, and cough
  • The safety profile for presymptomatic patients is consistent with the safety profile for symptomatic SMA patients treated with Evrysdi in clinical trials

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

Please see full Prescribing Information for additional Important Safety Information.

Clinical Perspectives Presented By:

Person 1

John Brandesma, MD

Assistant Professor of Neurology
Perelman School of Medicine
University of Pennsylvania
Neuromuscular Education Director
Neuromuscular Section Head
Attending Physician
Children’s Hospital of Philadelphia
Philadelphia, PA

Person 3

Tina Duong, MPT, PhD

Senior Research Scientist
Basic Life, Neurology
Stanford Medicine
Stanford, CA

Person 4

Shafeeq Ladha, MD

Gregory W. Fulton ALS and
Neuromuscular Disease Center
Barrow Neurological Institute
Phoenix, AZ

The objectives of this iPub® are to:

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Understand the importance of early intervention in SMA across patient types

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Discuss treatment goals and expectations across patient types

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Deepen the understanding of the efficacy and safety of Evrysdi across a range of patient types