Is there a generic for Evrysdi (risdiplam)?

No. Evrysdi (risdiplam) is only available as a brand-name medication.

Is Evrysdi (risdiplam) a gene therapy?

No, Evrysdi (risdiplam) isn't a [gene therapy](https://www.goodrx.com/health-topic/gene-therapy). It's a type of medication called an [RNA splicing modifier](https://www.goodrx.com/classes/rna-splicing-modifiers). Gene therapy and RNA splicing modifiers both use genetic material to treat genetic conditions. But they work in different ways. Gene therapy gives your body a new working [gene](https://www.yourgenome.org/theme/what-is-a-gene/) to replace the one that's missing or not working. Evrysdi (risdiplam) doesn't do that. Instead, it [makes changes](https://pmc.ncbi.nlm.nih.gov/articles/PMC8201486/) to the faulty gene that people with [spinal muscular atrophy](https://www.ninds.nih.gov/health-information/disorders/spinal-muscular-atrophy) (SMA) have. These changes help you body make a protein called survival motor neuron (SMN) that works correctly. This can help muscles work better in people with SMA.

How well does Evrysdi (risdiplam) work?

In studies, people 2 to 25 years old with type 2 or 3 spinal muscular atrophy (SMA) [got better](https://pmc.ncbi.nlm.nih.gov/articles/PMC9897618/) after taking Evrysdi (risdiplam) for a year. They had better movement skills, motor function, and arm strength. In another study, Evrysdi (risdiplam) helped with muscle strength in babies with type 1 SMA. After a year of treatment, [about 30%](https://pmc.ncbi.nlm.nih.gov/articles/PMC8567805/) of babies could sit up without help for at least 5 seconds and [almost 90%](https://dailymed.nlm.nih.gov/dailymed/fda/fdaDrugXsl.cfm?setid=eceb9a99-7191-4be5-87c3-0102707cf98e&type=display#section-11) could breathe on their own. After another year of treatment, 60% could sit up without help for at least 5 seconds. If you have questions about how well Evrysdi (risdiplam) is working, ask your prescriber.

Evrysdi

risdiplam

Used for Musculoskeletal Conditions

Evrysdi (risdiplam) is used to treat a genetic condition called spinal muscular atrophy (SMA) in adults and children. It's a type of medication called an RNA splicing modifier. It works to help improve the motor function of people with SMA. Evrysdi (risdiplam) comes as a tablet and oral solution, and you take the medication by mouth once per day. But your specific dose depends on your age and weight. Side effects can include fever, diarrhea, and rash.

Reviewed by:

Robert Chad Hakim, PharmD, BCCCP

Cydnee Ng, PharmD

Last reviewed on August 19, 2025

What is Evrysdi (risdiplam)?

What is Evrysdi (risdiplam) used for?

Spinal muscular atrophy (SMA) in adults and children

How Evrysdi (risdiplam) works

People with spinal muscular atrophy (SMA) don't have enough of a protein called survival motor neuron (SMN) in their body. Without enough SMN, the nerves and muscles in the body can't function well.

Evrysdi (risdiplam) is a SMN2-directed RNA splicing modifier. It helps your body make more SMN protein that works as it should. This is thought to help improve motor skills in people with SMA.

When does Evrysdi (risdiplam) start working?Time passed since treatment started:

HOURS

DAYS

WEEKS

MONTHS

Full effect

Drug facts

Common BrandsEvrysdi

Drug ClassSMN2 splicing modifier

Controlled Substance ClassificationNot a controlled medication

Generic StatusNo lower-cost generic available

AvailabilityPrescription only

Side effects of Evrysdi (risdiplam)

The following side effects may get better over time as your body gets used to the medication. Let your healthcare provider know immediately if you continue to experience these symptoms or if they worsen over time.

Common Side Effects

Fever (22%)
Diarrhea (17%)
Rash (17%)

Less Common Side Effects

Mouth sores
Joint pain
Urinary tract infection

Source: DailyMed

The following Evrysdi (risdiplam) side effects have also been reported

Side effects that you should report to your care team as soon as possible:

Allergic reactions—skin rash, itching, hives, swelling of the face, lips, tongue, or throat

Side effects that usually do not require medical attention (report these to your care team if they continue or are bothersome):

Constipation
Cough
Diarrhea
Fever
Skin rash
Vomiting

Pros and cons of Evrysdi (risdiplam)

Pros

Taken by mouth once a day
Okay for people of all ages with SMA to take
Available as a tablet and a liquid

Cons

Can cause diarrhea
Must follow a lot of instructions to take the liquid safely (e.g., refrigeration, no mixing with food)
Can take months to notice better muscle strength

Pharmacist tips for Evrysdi (risdiplam)

Take Evrysdi (risdiplam) once a day, with or without food. It's best to take the medication around the same time every day. Making it a part of your daily routine can help you remember when to take the medication. It also helps the medication work the best it can for you.
If you miss your dose and it's been less than 6 hours, take Evrysdi (risdiplam) as soon as you remember. Then, continue with your usual routine the next day. But if more than 6 hours have passed, skip the missed dose and then take your usual dose the next day.
If you don't fully swallow your dose of Evrysdi (risdiplam) or vomit after taking, don't take another dose to make up for it. Wait until the next day to take your usual dose at the usual time.
Be careful not to get Evrysdi (risdiplam) on your skin or in your eyes. If it gets on your skin, wash the area with soap and water. And if you get the medication in your eyes, rinse your eyes with water.

Tips for the Evrysdi (risdiplam) 0.75 mg/mL oral solution:

Each Evrysdi (risdiplam) box has a bottle of the liquid medication already prepared for you. There's also a cap for the bottle, bottle adapter, 1 or 2 reusable oral syringes, and the instructions for use. If the medication in the bottle is a powder, don't use it and call your pharmacist.
Review the instructions on how to store the medication, measure it out, and take it. Ask your prescriber or pharmacist if you have any questions.
When you're ready to take Evrysdi (risdiplam), draw up the liquid into the oral syringe provided. Use only the oral syringes from the Evrysdi (risdiplam) box. Don't use household spoons or other measuring devices. They might not be accurate and using them can cause you to take the wrong dose.
Within 5 minutes of drawing it up, take or give Evrysdi (risdiplam). If you don't, you'll need to throw that dose away and measure the medication out again.
Don't mix Evrysdi (risdiplam) with any foods or liquids. This includes formula or milk.
How to take or give Evrysdi (risdiplam): Place the oral syringe into the mouth so that the tip touches against the cheek. Slowly push the plunger all the way down to give all the medication in the syringe. Be careful not to give the medication too fast to prevent choking. Drink some water after taking the medication to make sure you swallowed your whole dose. Then, rinse the oral syringe according to the directions.
If you're giving the medication through a feeding tube (like a tube through the nose or stomach), follow the directions that came with the medication. After you give the medication, flush the tube with water.
Keep the Evrysdi (risdiplam) liquid in the refrigerator between 36°F and 46°F (or between 2°C and 8°C). Don't freeze the medication. Keep the liquid in the original bottle to protect the medication from light.
If needed, you can keep the liquid at room temperature up to 104°F (or 40°C) for a combined total of 5 days. This means it's okay to put it back in the fridge once it's at room temperature. But throw it away if it's been out for more than 5 days total.
Throw away any leftover liquid in the bottle 64 days (about 2 months) after the pharmacy prepared it. The pharmacist should've written this expiration date on the bottle for you.
If you don't have enough medication in the bottle for a full dose, throw away the bottle and the used oral syringes. Use a new bottle of Evrysdi (risdiplam) and the new oral syringes that come with it to get your full dose. Don't mix liquid from an old bottle and a new bottle together.

Tips for the Evrysdi (risdiplam) 5 mg tablets:

Swallow the Evrysdi (risdiplam) tablet whole with water. Don't cut, chew, or crush them.
You can also place a tablet in a cup with 5 mL (a teaspoonful) of room-temperature filtered water. Gently swirl the cup for up to 3 minutes until it's mixed well. Then, drink the mixture within 10 minutes. If it's been more than 10 minutes, throw the mixture away and prepare a new dose.
Wash your hands well before and after taking the Evrysdi (risdiplam) tablet so it doesn't get on your skin or eyes.
Never put a dissolved tablet solution through a feeding tube.
Store Evrysdi (risdiplam) tablets at room temperature at 68°F to 77°F (or 15°C to 30°C). Keep the tablets in the original bottle it came in. Also keep the bottle closed tightly to protect it from moisture.

Common questions about Evrysdi

No. Evrysdi is only available as a brand-name medication.

No, Evrysdi isn't a gene therapy. It's a type of medication called an RNA splicing modifier. Gene therapy and RNA splicing modifiers both use genetic material to treat genetic conditions. But they work in different ways. Gene therapy gives your body a new working gene to replace the one that's missing or not working. Evrysdi doesn't do that. Instead, it makes changes to the faulty gene that people with spinal muscular atrophy (SMA) have. These changes help you body make a protein called survival motor neuron (SMN) that works correctly. This can help muscles work better in people with SMA.

In studies, people 2 to 25 years old with type 2 or 3 spinal muscular atrophy (SMA) got better after taking Evrysdi for a year. They had better movement skills, motor function, and arm strength. In another study, Evrysdi helped with muscle strength in babies with type 1 SMA. After a year of treatment, about 30% of babies could sit up without help for at least 5 seconds and almost 90% could breathe on their own. After another year of treatment, 60% could sit up without help for at least 5 seconds. If you have questions about how well Evrysdi is working, ask your prescriber.

In studies, the levels of working SMN protein needed for nerve and muscle function went up within 4 weeks of taking Evrysdi. But how fast your muscles get stronger can be different for everyone. In general, it can depend on your age, the type of SMA you have, and your symptoms. In studies, people 2 to 25 years with Type 2 or 3 SMA had better motor strength within 4 months of treatment. But it took 8 to 12 months to get the most help from the medication. Follow up with your prescriber about your motor skills and muscle strength. If you have questions about how long Evrysdi is taking to help, ask your prescriber.

Both Evrysdi and Spinraza (nusinersen) can help treat all types of spinal muscular atrophy (SMA) in adults and children. They work in different ways to get the levels of SMN protein in the body go up. , but they do so in different ways. The medications also differ in how you take them. Evrysdi is taken by mouth once a day. But Spinraza is as an injection into the spine given by a healthcare professional every 4 months (after some starter doses). The side effects slightly differ as well. Evrysdi can cause diarrhea and rash. But Spinraza has a risk of more serious side effects like kidney and bleeding. Talk to your prescriber to learn about how these medications compare and which is right for you.

Evrysdi and Zolgensma (onasemnogene abeparvovec) work in different ways to treat spinal muscular atrophy (SMA). They're also approved for people of different ages. Evrysdi is for people of all ages. But Zolgensma is only for children younger than 2. You also take the medications in different ways. Evrysdi is taken by mouth once a day. Zolgensma is given as an infusion into your vein as a single dose. And the side effects can be different. Evrysdi can cause diarrhea and rash. On the other hand, Zolgensma has risks like infusion reactions and liver damage. Talk to your prescriber to learn more about comparing the two medications and see which is right for you.

If you're pregnant or thinking of getting pregnant, talk to your prescriber. They can explain the risks of taking Evrysdi. It's not clear if it's safe to take while pregnant. But in animal studies, doses that were 2- to 4-times higher than the typical dose harmed babies. If you get pregnant while taking Evrysdi, tell your prescriber right away. They can talk to you about joining the Evrysdi Pregnancy Registry. This registry helps researchers learn more about how safe the medication is during pregnancy. You can visit the registry's website or call 1-833-760-1098 for more information.

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Risks and warnings for Evrysdi (risdiplam)

Evrysdi (risdiplam) can cause some serious health issues. This risk may be even higher for certain groups. If this worries you, talk to your doctor or pharmacist about other options.

Possible interactions with other medications

Evrysdi (risdiplam) might interact with some medications. Examples include metformin and cephalexin. These drug interactions might raise the risk of side effects.

Before starting Evrysdi (risdiplam), tell your care team about all the medications you take or plan to take. They can check for drug interactions. If needed, they can recommend dose adjustments or treatment changes to keep you safe.

Possible harm to an unborn baby

Risk factors: Pregnant or planning to become pregnant

There's not enough research to know for sure if it's safe to take Evrysdi (risdiplam) while pregnant. But animal studies show that it can cause harm to an unborn baby. This can include miscarriage (pregnancy loss), birth defects, and low birth weight.

If you can become pregnant, talk to your prescriber. They can help you understand the risks and benefits of taking Evrysdi (risdiplam). If you decide to take it, you might need take a pregnancy test to make sure you're not pregnant before starting. Then, use reliable birth control during treatment and for at least 1 month after you stop taking Evrysdi (risdiplam).

If you become pregnant while taking Evrysdi (risdiplam), tell your prescriber right away. It's recommended that you join the Evrysdi Pregnancy Registry. It helps researchers learn more about the risks of taking this medication when pregnant. Ask your prescriber for more information on how to enroll.

Possible fertility problems in males

It isn't clear if Evrysdi (risdiplam) can cause problems for males who want to have children. Animal studies show that it's possible. But in a recent case series, three males who took the medication were still able to have children.

If the risk of fertility problems worries you, speak to your prescriber. They can talk to you about options like sperm freezing.

Evrysdi (risdiplam) dosage

Typical dosage for Evrysdi (risdiplam)

Your prescriber will find the dose for you. It's based on your age and body weight (in kilograms, or kg).

Younger than 2 months of age: The typical dose is 0.15 mg/kg by mouth once a day. Use the Evrysdi oral solution.
Age 2 months to under 2 years: The typical dose is 0.2 mg/kg by mouth once a day. Use the Evrysdi oral solution.
Age 2 years and older who weigh less than 20 kg (44 lbs): The typical dose is 0.25 mg/kg by mouth once a day. Use the Evrysdi oral solution.
Age 2 years and older who weigh 20 kg (44 lbs) or more: The typical dose is 5 mg by mouth once a day. Use either the Evrysdi oral solution or tablet.

What are alternatives to Evrysdi (risdiplam)?

There are a number of medications that your doctor can prescribe in place of Evrysdi (risdiplam). Compare a few possible alternatives below.

Evrysdi (risdiplam)

Used for:

Spinal muscular atrophy (SMA) in adults and children

View prices

Spinraza (nusinersen)

Used for:

Treatment of spinal muscular atrophy (SMA) in children and adults

View prices

Zolgensma

Used for:

Certain type of spinal muscular atropy (SMA) in children under 2 years old

View prices

References

Best studies we found

American Optometric Association. (n.d.). Protecting your eyes at work.

Borate, G. M., et al. (2022). Cryopreservation of sperm: A review. Cureus.

Canadian Agency for Drugs and Technologies in Health. (2022). Nusinersen (Spinraza): CADTH reimbursement review: Therapeutic area: Spinal muscular atrophy.

GoodRx Health has strict sourcing policies and relies on primary sources such as medical organizations, governmental agencies, academic institutions, and peer-reviewed scientific journals. Learn more about how we ensure our content is accurate, thorough, and unbiased by reading our editorial guidelines.

Common questions about Evrysdi

No. Evrysdi is only available as a brand-name medication.

Evrysdi