Evrysdi®

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

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About Evrysdi®

  • Evrysdi® (risdiplam), marketed by Genentech, a member of the Roche Group, is an FDA-approved in pediatric and adult patients for all ages and types of SMA
  • Evrysdi® is a daily oral non-invasive liquid medication. It can also be given through a g-tube
  • Evrysdi® dosing is based on safety and efficacy across SMA types studied
  • Many SMA healthcare providers across the U.S. prescribe Evrysdi®. You can search some of them on our map locator tool
  • To find insurance policies and payer information for Evrysdi®, please 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
  • 0.15 mg/kg/day up to two months of age
  • 0.2 mg/kg/day two months to < two years
  •  0.25 mg/kg >/= two years old and < 20 kg
  • 5 mg per day >/= two years old > 20 kg

Concentration: 0.75 mg/ml
How given Enteral liquid by mouth or feeding tube
How often Daily
Body distribution Throughout the body including the central nervous system and other organs
Warnings and precautions Animal toxicity studies raised concerns about mature male infertility and possible harm to fetus
Adverse reactions
  • Later-onset SMA: fever, diarrhea, and rash
  • Infantile-onset SMA: fever, diarrhea, rash, upper respiratory tract infection, pneumonia, constipation, vomiting, and cough
Monitoring
  • None required
  • Recommended: liver function testing, pregnancy testing, and multidrug and toxin extrusion (MATE) protein transporters medication interaction
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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