Evrysdi®

Evrysdi® is an FDA- approved therapy indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients with SMA. It has been studied in a population that included patients that were pre-symptomatic, Type 1, 2, and 3 from newborn to 60 years of age. Evrysdi is a survival motor neuron 2 (SMN2) splicing modifier.

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About Evrysdi®

  • Evrysdi® (risdiplam), marketed by Genentech, a member of the Roche Group, is FDA-approved for the treatment of SMA in pediatric and adult patients of all ages and types of SMA
  • Evrysdi® is a daily oral medication available as a tablet that may be taken whole with water or dispersed in one teaspoon (5 mL) of room temperature non-chlorinated drinking water; Evrysdi® is also available as a daily oral liquid medication that may be given through a feeding tube; Evrysdi® may be given with or without food
  • Evrysdi® dosing is based on age and body weight
  • Many SMA healthcare providers across the U.S. prescribe Evrysdi®. You can search some of them with our Find a Treatment Center tool
  • To find insurance policies and payer information for Evrysdi®, please click here
  • For Cure SMA’s outreach to public and private insurers regarding Evrysdi® coverage, click here
  • Check out the Evrysdi® patient brochure for adults, children, and infants with SMA
  • For the most up-to-date prescribing information, see here

Overview:

Description Small molecule
Mechanism SMN2 mRNA splicing modifier
Approved age All ages and all types
Dose Liquid (Concentration 0.75 mg/ml)

  • For less than 2 months of age, give 0.15 mg/kg
  • For 2 months of age to less than 2 years give 0.2 mg/kg
  • For 2 years of age and older and less than 20kg, give 0.25 mg/kg
  • 5 mg per day >/= two years old > 20 kg

Liquid (5 mg/6.6 ml) or Tablet (5 mg)

  • For 2 years of age and older weighing 20 kg or more, give 5 mg per day
How given Enteral liquid by mouth or feeding tube

Tablet by mouth (oral whole or dispersed in one teaspoon (5 mL) of room temperature non-chlorinated drinking water)
How often Daily
Body distribution Throughout the body including the central nervous system and other organs
Adverse reactions
  • Later-onset SMA: fever, diarrhea, and rash
  • Infantile-onset SMA: fever, diarrhea, rash, upper respiratory tract infection, lung infection or pneumonia, constipation, vomiting, and cough
Monitoring
  • None required
Drug Interactions
  • EVRYSDI may increase the concentration of drugs in the blood that are eliminated through multidrug and toxin extrusion (MATE) protein transporters, such as metformin.
Use in Specific Populations
  • Pregnancy: no adequate data in pregnant women. In non-clinical animal studies, risdiplam during pregnancy resulted in adverse effects on fetus development. 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 https://www.evrysdipregnancyregistry.com.
  • If you are a woman who can become pregnant, before starting treatment with EVRYSDI, your healthcare provider may test you for pregnancy. Because EVRYSDI may harm your unborn baby, you and your healthcare provider will decide if taking EVRYSDI is right for you. Talk to your healthcare provider about birth control methods and use birth control while on treatment and for at least 1 month after stopping EVRYSDI.
  • If you are an adult male planning to have children: EVRYSDI may affect a man’s ability to have children (fertility) based on animal toxicity studies. If this is of concern, please ask your healthcare provider for advice. Also, consider sperm preservation prior to treatment.
  • Breastfeeding or planning to breastfeed: It is not known if EVRYSDI passes into breast milk and may harm your baby. If you plan to breastfeed, discuss with your healthcare provider about the best way to feed your baby while on treatment with EVRYSDI.
Prescribing information See here

History of Evrysdi®

Roche and Genentech led the clinical development of Evrysdi® in collaboration with the SMA Foundation and PTC Therapeutics. The approval of Evrysdi® was based on three multicenter clinical trials in people with SMA. These initial clinical trials studied symptomatic infantile onset SMA (FIREFISH), symptomatic later onset SMA (SUNFISH), and pre-symptomatic infants with SMA (RAINBOWFISH). These trials support the effectiveness of Evrysdi® in pediatric and adult patients and support the early initiation of treatment with Evrysdi®.

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