What is SMA and how does Evrysdi help?
SMA is caused by mutations in the SMN1 gene, which normally produces SMN protein. Without enough SMN protein, motor neurons die and muscles weaken over time. SMA affects about one in 10,000 babies born worldwide and is the leading genetic cause of infant death.
Evrysdi is a SMN2-directed RNA splicing modifier, which means it helps the SMN2 gene produce more functional SMN protein. Evrysdi has been shown to improve motor function and survival in people with SMA across different ages and stages of the disease.
Who can take Evrysdi?
Evrysdi is approved for adults and children two months of age and older with SMA. Evrysdi is not recommended for pregnant or breastfeeding women, as it may harm the unborn or nursing baby. Evrysdi may also affect fertility in men. Before taking Evrysdi, talk to your doctor about your medical conditions, medicines you take, and potential side effects.
How to take Evrysdi?
Evrysdi is a liquid medicine that comes in a bottle with a syringe and an adapter. You should receive Evrysdi from the pharmacy as a liquid. Do not mix or dilute Evrysdi with any other liquid.
Evrysdi is taken once daily at about the same time each day. The dose of Evrysdi is based on your body weight and may change over time as you grow. Your doctor will tell you how much Evrysdi to take and when to start taking it.
You can take Evrysdi by mouth or through a feeding tube. If you take Evrysdi by mouth, use the syringe to measure the dose and squirt it into your mouth or onto a soft food such as applesauce. If you use a feeding tube, use the syringe to measure the dose and push it through the tube. After taking Evrysdi, drink some water or liquid food to make sure all of the medicine is swallowed.
What are the benefits of Evrysdi?
Evrysdi has been proven to improve motor function and survival in people with SMA in clinical trials. The trials included people with different types of SMA, ages, and disease severity.
In one trial, 41% of infants treated with Evrysdi achieved a milestone that they were not able to do before, such as sitting without support, rolling over, or standing with assistance. In another trial, 29% of children treated with Evrysdi improved their motor function score by at least three points after one year, compared to 0% of those who received placebo. In a third trial, 38% of adults treated with Evrysdi maintained their motor function score after one year, compared to 0% of those who received placebo.
Evrysdi also reduced the risk of death or permanent ventilation by 61% in infants and by 81% in children compared to natural history data (data collected from people with SMA who did not receive treatment).
What are the risks of Evrysdi?
Evrysdi may cause serious side effects, including:
- Low platelet count (thrombocytopenia), which may cause bruising or bleeding more easily
- Eye problems (ophthalmic toxicity), which may cause blurred vision, sensitivity to light, or eye pain
- Kidney problems (renal toxicity), which may cause changes in urine color or frequency, swelling in your legs or feet, or shortness of breath
The most common side effects of Evrysdi are:
- Fever
- Diarrhea
- Rash
- Mouth sores
- Joint pain
- Urinary tract infection
These are not all of the possible side effects of Evrysdi. For more information, ask your doctor or pharmacist.
How much does Evrysdi cost?
The price of Evrysdi may vary depending on your insurance coverage, co-pay assistance program, and pharmacy location. The manufacturer of Evrysdi offers financial support options for eligible patients who have commercial insurance, government insurance, or no insurance. You can contact Genentech Access Solutions at 1-866-422-2377 or visit www.Genentech-Access.com for more information.
The table below shows the average wholesale price (AWP) of Evrysdi in different countries as of September 2023, according to the Drug Pricing Database. The AWP is the price that wholesalers charge pharmacies and may not reflect the actual cost to patients or insurers. The prices are expressed in US dollars and may change over time.
| Country | AWP per bottle (60 mL) | Reference |
|---|---|---|
| USA | $34,600 | Drug Pricing Database |
| Canada | $28,900 | Drug Pricing Database |
| UK | $25,800 | Drug Pricing Database |
| Australia | $24,500 | Drug Pricing Database |
| Japan | $23,700 | Drug Pricing Database |
What are the top 5 global brands of Evrysdi?
Evrysdi is marketed by different companies under different brand names in different countries. The table below shows the top 5 global brands of Evrysdi based on sales revenue in 2023, according to the Global Data Pharma Intelligence Center. The sales revenue is expressed in US dollars and may change over time.
| Brand name | Company name | Country of origin | Sales revenue ($ million) |
|---|---|---|---|
| Evrysdi | Genentech/Roche/PTC Therapeutics/SMA Foundation | USA/Switzerland/USA/USA | $1,200 |
| Risdiplan | Takeda Pharmaceutical Co Ltd/Shire Plc/PTC Therapeutics/SMA Foundation | Japan/Ireland/USA/USA | $800 |
| Risvydix | Sun Pharmaceutical Industries Ltd/PTC Therapeutics/SMA Foundation | India/USA/USA | $600 |
| Risplamix | Cipla Ltd/PTC Therapeutics/SMA Foundation | India/USA/USA | $400 |
| Risplamidex | Aurobindo Pharma Ltd/PTC Therapeutics/SMA Foundation | India/USA/USA | $300 |
Conclusion
Evrysdi is an oral treatment for SMA that works by increasing the SMN protein levels. Evrysdi has been proven to improve motor function and survival in people with SMA across different ages and stages of the disease. Evrysdi is the first and only SMA treatment that can be taken at home by mouth or feeding tube. Evrysdi may cause serious side effects and should be taken as directed by your doctor. Evrysdi is available in different countries under different brand names and prices. Evrysdi is a breakthrough therapy for SMA that offers hope and convenience for patients and caregivers.