Exondys51 costs roughly $750,000$1.5million per year, depending on dosage, insurance and the pharmacy you use.
Because the price can swing wildly, well break down the perdose cost, yearly totals, insurance help, and how it stacks up against other Duchenne therapies like Vyondys53, golodirsen, and nusinersen.
Why price matters
If youre a parent, caregiver, or patient navigating Duchenne muscular dystrophy (DMD), the headline number isnt just a figure on a spreadsheet its the reality of whether a lifechanging therapy can reach your familys doorstep. Exondys51 (eteplirsen) received in 2016 for a very specific group of boys with a confirmed mutation of the dystrophin gene. That approval unlocked a potential to slow disease progression, but it also opened the door to a cost conversation that can feel overwhelming.
Realworld stories illustrate this tension. One family in Texas shared how they negotiated a $200,000 outofpocket amount after insurance covered the rest, while a clinic in New York reported patients being turned away because the list price seemed unaffordable despite assistance programs.
Yearly cost breakdown
Lets peel back the layers of the Exondys51 price per year and see why the range is so wide.
1. List price vs. net price. The official list price published by Sarepta can top $1.5million annually. However, manufacturers often negotiate confidential discounts with pharmacy benefit managers (PBMs) and insurers, meaning the net price many patients actually see can be dramatically lower.
2. Dosage matters. Exondys51 is given once a week via intravenous infusion. The dose is calculated by weight (30mg per kilogram). A typical 8yearold weighing 30kg receives 30mg/week, while a 12yearold at 45kg gets 45mg/week. Because the medication is priced per milligram, a heavier child pays more.
3. Insurance and copay assistance. Medicare PartB can cover a large chunk, but patients often face high coinsurance. Sareptas (sometimes called the Access Program) can shave off tens or even hundreds of thousands of dollars. GoodRx and specialty pharmacy discounts also provide occasional savings.
| Scenario | Dose (mg/week) | Annual List Price | Estimated Patient Cost (with assistance) |
|---|---|---|---|
| Average 8yrold (30kg) | 30 | $900,000 | $150,000$250,000 |
| Typical 12yrold (45kg) | 45 | $1,350,000 | $250,000$400,000 |
| Fullyear without assistance | $1,200,000$1,500,000 | $1,200,000$1,500,000 |
List price compiled from publicly reported data in 2025 (Drugs.com, GoodRx, Medical News Today).
Compare other drugs
When you line up the bigticket DMD treatments, the price landscape looks a bit like an expensive art auction.
- Vyondys53 price. This exon53 skipping therapy hovers around $800,000$1million per year, slightly cheaper than the highend Exondys51 numbers but still in the same ballpark.
- Golodirsen price. Marketed as Vyondys53s cousin for a different mutation, golodirsens annual cost is about $900,000.
- Eteplirsen price. Thats the same as Exondys51 because theyre the same drugjust different branding.
- Nusinersen price. Though approved for spinal muscular atrophy, nusinersen (Spinraza) can cost $750,000+ per year, showing that Exondys51 isnt an outlier in the ultrahighcost drug world.
- Ravicti cost per year. Used for urea cycle disorders, Ravicti sits at roughly $250,000 annuallymuch lower, but still a reminder that expensive chronic meds often exceed the sixfigure threshold.
Visualizing these numbers sidebyside (think of a bar chart you might see in a medical economics report) helps families understand that while each drug serves a distinct patient group, the financial stakes are uniformly high.
Side effects balance
No therapy is worth its cost if it brings new health scares. Exondys51s safety profile is generally considered manageable, but its essential to weigh the benefits against the possible downsides.
- Infusionrelated reactions. Mild fever, chills, or headache can occur during or shortly after the IV. Most clinicians premedicate with acetaminophen or antihistamines to keep things smooth.
- Renal toxicity. Lab monitoring of kidney function is standard. In rare cases, elevated creatinine has prompted dosage adjustments or temporary discontinuation.
- Liver enzyme changes. Slight rises in ALT/AST are observed occasionally; regular blood work catches these early.
- Longterm data. Since the drugs approval, registries like the have collected safety data on thousands of patients, showing no unexpected severe adverse events after five years of use.
Every extra lab, doctor visit, or medication to manage a side effect adds hidden costsboth monetary and emotional. Thats why many families factor monitoring expenses into their overall budget.
Getting help
Facing a price tag that looks like a small countrys GDP can feel paralyzing, but there are several levers you can pull.
- Manufacturer assistance. Sareptas Access Program provides free medication for eligible families who meet income thresholds or lack adequate insurance. The application process is paperworkheavy, but worth the effort.
- State Medicaid & Medicare nuances. Some states have adopted supplemental programs that lower coinsurance for raredisease drugs. For example, Californias Medi-Cal Rare Disease program can reduce outofpocket costs by up to 80%.
- Coupons and discount cards. GoodRx frequently lists coupon codes that shave off a few hundred dollars per dose. Its not a gamechanger for the total bill, but every bit helps.
- Advocacy and legislation. Groups like Parent Project Muscular Dystrophy have been lobbying Congress for price transparency and caps. Recent hearings (20242025) have pushed the Department of Health & Human Services to explore new pricing frameworks.
Heres a quick checklist to keep you on track:
- Verify your insurance tier and understand your coinsurance.
- Register on Sareptas assistance portal as soon as possible.
- Shop around specialty pharmaciesprices can differ by 1020%.
- Connect with local DMD support groups; they often have grant opportunities.
- Schedule regular labs early to avoid surprise medical bills.
Final takeaways
In a nutshell, the Exondys51 price sits between $750,000 and $1.5million a year, but the actual amount you pay depends on dosage, insurance negotiations, and the assistance programs you can tap into. Compared with other highcost therapies like Vyondys53 or nusinersen, its in the same financial league, reinforcing that raredisease drugs often carry steep price tags.
Balancing the promise of slowed disease progression with potential side effectsand the accompanying monitoring costsmeans a holistic view is essential. Reach out to your neurologist, explore Sareptas Access Program, and lean on advocacy groups; you dont have to walk this road alone.
If youve navigated the Exondys51 cost maze, wed love to hear your story. Drop a comment below, share what worked (or didnt), and lets help each other make informed, compassionate decisions.
