Evrysdi (risdiplam)
An approved treatment for Spinal Muscular Atrophy.
The same compound appears under different names depending on the context. Here is how to identify Risdiplam wherever you encounter it, plus the key facts at a glance.
- Generic name
- Risdiplam
- Brand name
- Evrysdi
- Development codes
- RG7916, RO7034067
- Drug class
- SMN2 splicing modifier
- Manufacturer
- Roche / Genentech
- How it's taken
- Taken once daily by mouth, with or without food, as a strawberry-flavored liquid.
The first oral treatment for spinal muscular atrophy. Taken daily at home as a liquid or, for some patients, a tablet, Evrysdi works throughout the entire body (not just the nervous system) to increase SMN protein levels.
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Where Risdiplam fits
First and only oral SMA therapy, taken daily at home as a flavored liquid. Provides a convenient non-invasive alternative to intrathecal nusinersen or one-time gene therapy.
How Risdiplam works
Like nusinersen, risdiplam targets the SMN2 gene's splicing to produce more functional SMN protein. But unlike nusinersen (which only works in the spinal fluid), risdiplam is a small molecule taken by mouth that distributes throughout the entire body, reaching muscles, nerves, and other organs. This systemic distribution may provide benefits beyond the central nervous system.
Mechanism: Small molecule SMN2 splicing modifier that increases full-length SMN protein production throughout the body
Side effects and safety
Common side effects include fever, diarrhea, rash, upper respiratory infections, and mouth sores. In infants, constipation and vomiting are frequent. Risdiplam may affect male fertility, and animal studies suggest it could harm an unborn baby, so talk to your doctor about pregnancy and birth control.
This is not a complete list of side effects. Talk to your doctor or pharmacist about what to expect and when to seek medical attention.
Taking Risdiplam
Taken once daily by mouth, with or without food, as a strawberry-flavored liquid. People on the 5 mg dose can instead take a 5 mg tablet, swallowed whole or dispersed in water. The liquid or dispersed tablet can also be given through a feeding tube. Dose is weight-based. No injections or hospital visits required for administration, making it the most convenient SMA treatment option.
Availability and cost
Only available as the brand-name product.
First oral therapy for SMA, a small molecule that modifies SMN2 pre-mRNA splicing. Daily oral liquid taken at home, avoiding the need for hospital-based infusions or injections.
Help paying for Evrysdi
Pick your insurance to see which help fits. Drugmaker copay cards can't be used with Medicare, Medicaid or TRICARE; charity funds are the usual route there.
- Copay help
Evrysdi Co-pay Program: eligible commercially insured patients could pay as little as $0 per 30-day supply, up to $25,000 per calendar year. Excludes Medicare, Medicaid, TRICARE.
For: private insurance · source - Free medicine program
Genentech Patient Foundation gives free Evrysdi to eligible uninsured patients or insured patients without coverage, with incomes under $150,000; other income-based criteria apply.
For: no insurance, underinsured · source - Other support
Referrals to independent co-pay assistance foundations, which may help commercially or publicly insured patients.
For: private insurance, Medicare, Medicaid · source
Good to know: Free drug comes from the separate Genentech Patient Foundation (888-941-3331); both a patient consent form and a prescriber form are required. The MySMA Support Program and Partnership and Access Liaisons (PALs) offer additional support.
- From a charity · TotalAssist (formerly PAN Foundation)Spinal Muscular Atrophy fundOpen
Pays for: Out-of-pocket costs for approved medications, up to $6,500 per year. Requires health insurance (any kind).
- From a charity · Cure SMAEquipment Pool and Travel Support Package fundApply directly
Pays for: Medical equipment loans (wheelchairs, car beds) and travel equipment.
The foundation says: “Status not shown on page” - From a charity · Muscular Dystrophy AssociationMDA Durable Medical Equipment (DME) Grant Program fundApply directly
Pays for: Medical equipment (wheelchairs, lifts, canes and other DME), up to $1,000 per year.
The foundation says: “Status not shown on page”
Clinical trial results
FIREFISH trial (SMA Type 1): 29% of infants could sit independently at 12 months, versus 0% expected naturally. SUNFISH trial (SMA Types 2/3): significant improvement in motor function versus placebo. RAINBOWFISH (presymptomatic): 25 of 26 treated babies (96%) could sit without support for at least 5 seconds at 12 months.
Development history
Developed by Roche/Genentech in collaboration with the SMA Foundation and PTC Therapeutics. Approved August 2020 for patients 2 months and older. Later expanded to newborns. The oral formulation dramatically improved treatment accessibility for SMA patients worldwide.
Explore Spinal Muscular Atrophy trials
Other Spinal Muscular Atrophy treatments
Risdiplam in Trial Friend News and Guides
Common questions about Risdiplam
▸What is Risdiplam (Evrysdi)?
The first oral treatment for spinal muscular atrophy. Taken daily at home as a liquid or, for some patients, a tablet, Evrysdi works throughout the entire body (not just the nervous system) to increase SMN protein levels.
▸How does Risdiplam work?
Like nusinersen, risdiplam targets the SMN2 gene's splicing to produce more functional SMN protein. But unlike nusinersen (which only works in the spinal fluid), risdiplam is a small molecule taken by mouth that distributes throughout the entire body, reaching muscles, nerves, and other organs. This systemic distribution may provide benefits beyond the central nervous system.
▸What are the side effects of Risdiplam?
Common side effects include fever, diarrhea, rash, upper respiratory infections, and mouth sores. In infants, constipation and vomiting are frequent. Risdiplam may affect male fertility, and animal studies suggest it could harm an unborn baby, so talk to your doctor about pregnancy and birth control.
▸How is Risdiplam taken?
Taken once daily by mouth, with or without food, as a strawberry-flavored liquid. People on the 5 mg dose can instead take a 5 mg tablet, swallowed whole or dispersed in water. The liquid or dispersed tablet can also be given through a feeding tube. Dose is weight-based. No injections or hospital visits required for administration, making it the most convenient SMA treatment option.
▸Is Risdiplam FDA approved?
Yes, Risdiplam (Evrysdi) is FDA approved (2020) for the treatment of Spinal Muscular Atrophy.
▸What makes risdiplam unique among SMA treatments?
Risdiplam (Evrysdi) is the first and only oral therapy for SMA, taken daily as a flavored liquid at home. This provides a major convenience advantage over nusinersen (intrathecal injections) and gene therapy (hospital-based infusion).
▸How does risdiplam work?
Risdiplam is a small molecule that modifies SMN2 pre-mRNA splicing to increase production of functional SMN protein. Unlike nusinersen which works only in the central nervous system, risdiplam distributes throughout the body, potentially benefiting peripheral organs as well.
▸Who is eligible for risdiplam?
Risdiplam is approved for SMA in children and adults of all ages, including newborns, regardless of SMA type. The broad age range and lack of procedural requirements make it accessible to the widest patient population.
▸How is risdiplam administered?
Risdiplam is taken once daily as a flavored oral liquid (strawberry-flavored solution). It can be administered at home by patients, caregivers, or through a feeding tube for patients who cannot swallow. No hospital visits are required.
▸How does risdiplam compare to nusinersen?
Both increase SMN protein by modifying SMN2 splicing, but risdiplam is oral (daily at home) while nusinersen requires intrathecal injections every 4 months. Risdiplam distributes systemically, while nusinersen primarily targets the central nervous system.
▸What patient support is available for Evrysdi?
Genentech provides patient support through their access solutions program, including insurance navigation, copay assistance, and treatment coordination for Evrysdi patients.