Vyondys 53 (golodirsen)
An approved treatment for Duchenne Muscular Dystrophy.
The same compound appears under different names depending on the context. Here is how to identify Golodirsen wherever you encounter it, plus the key facts at a glance.
- Generic name
- Golodirsen
- Brand name
- Vyondys 53
- Development code
- SRP-4053
- Drug class
- Antisense oligonucleotide (exon skipping)
- Manufacturer
- Sarepta Therapeutics
- How it's taken
- Given as a weekly intravenous infusion over 35-60 minutes.
An exon 53 skipping therapy for DMD patients whose dystrophin gene mutation is amenable to skipping exon 53, representing about 8% of the DMD population.
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Where Golodirsen fits
Exon 53 skipping therapy for DMD, an alternative to viltolarsen for the same patient population. Applicable to roughly 8% of DMD patients with amenable mutations.
How Golodirsen works
Like other exon-skipping therapies, golodirsen tells cells to skip over exon 53 in the dystrophin gene instructions. This allows cells to produce a shorter but partially functional dystrophin protein instead of no dystrophin at all.
Mechanism: Antisense oligonucleotide that enables exon 53 skipping to produce shortened but functional dystrophin
Side effects and safety
Common side effects include headache, fever, fall, abdominal pain, sore nose and throat (nasopharyngitis), cough, vomiting, and nausea. Hypersensitivity reactions, including anaphylaxis, rash, fever, itching, hives and skin peeling, have occurred, and the drug should not be given to anyone who has had a serious allergic reaction to it. The label also warns about kidney toxicity: it was seen in animal studies but not in the clinical trials, and potentially fatal glomerulonephritis has occurred with some other antisense drugs, so kidney function is checked before and during treatment.
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 Golodirsen
Given as a weekly intravenous infusion over 35-60 minutes. Administered at infusion centers or potentially at home after initial doses.
Availability and cost
Only available as the brand-name product.
Antisense oligonucleotide for exon 53 skipping, applicable to a subset of DMD patients. Ultra-orphan pricing for a narrowly defined genetic subset.
Help paying for Vyondys 53
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.
- Insurance and case manager help
A dedicated SareptAssist Case Manager helps with insurance approvals and coordinating VYONDYS 53 delivery.
The official page does not say who qualifies. Ask the program. · source - Copay help
Co-pay Assistance Program may help commercially insured patients with co-pays, co-insurance and deductibles. Not for government insurance like Medicare.
For: private insurance · source - Free medicine program
Case Managers can share information on a Sarepta Patient Assistance Program. Eligibility is not described on the official pages.
The official page does not say who qualifies. Ask the program. · source
Good to know: Call 1-888-SAREPTA (Mon-Fri 8:30am-6:30pm ET). Copay terms are on Sarepta's corporate SareptAssist page, which covers 'a Sarepta product'. Who qualifies for the Patient Assistance Program is not stated on official pages; ask the Case Manager.
- From a charity · NORD RareCareDuchenne Muscular Dystrophy Medical Assistance fundOpen
Pays for: Medical and medication costs.
The foundation says: “Accepting new applications and re-enrollments for current year” - From a charity · NORD RareCareDuchenne Muscular Dystrophy Premium Copay Assistance fundOpen
Pays for: Insurance premiums and copays.
The foundation says: “Accepting new applications and re-enrollments for current year” - From a charity · The Assistance FundDuchenne Muscular Dystrophy fundOpen
Pays for: Copays, coinsurance, deductibles and other health-related expenses.
The foundation says: “OPEN — Accepting New Patients. TAF is currently accepting new patient enrollments for this program.” - 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”
Access and eligibility
Vyondys 53 is only for patients with a confirmed DMD gene mutation amenable to exon 53 skipping, which applies to a subset of DMD patients.
Source: Vyondys53.com (Sarepta Therapeutics)
Access program details are provided for informational purposes and may vary based on insurance coverage, geographic location, and individual circumstances. Confirm current eligibility directly with the manufacturer or your specialty pharmacy.
Clinical trial results
Approved December 2019 under accelerated approval. The 4053-101 study in 25 boys demonstrated increased dystrophin expression on muscle biopsy.
Development history
Developed by Sarepta Therapeutics as the second exon-skipping therapy for DMD, targeting a different patient subgroup than eteplirsen. Approved under accelerated approval. The confirmatory ESSENCE trial did not meet its main goal, though Sarepta reported trends favoring treatment. Sarepta has asked the FDA to convert Vyondys 53 to traditional approval, and the FDA set a decision date of February 28, 2027.
Explore Duchenne Muscular Dystrophy trials
Other Duchenne Muscular Dystrophy treatments
Common questions about Golodirsen
▸What is Golodirsen (Vyondys 53)?
An exon 53 skipping therapy for DMD patients whose dystrophin gene mutation is amenable to skipping exon 53, representing about 8% of the DMD population.
▸How does Golodirsen work?
Like other exon-skipping therapies, golodirsen tells cells to skip over exon 53 in the dystrophin gene instructions. This allows cells to produce a shorter but partially functional dystrophin protein instead of no dystrophin at all.
▸What are the side effects of Golodirsen?
Common side effects include headache, fever, fall, abdominal pain, sore nose and throat (nasopharyngitis), cough, vomiting, and nausea. Hypersensitivity reactions, including anaphylaxis, rash, fever, itching, hives and skin peeling, have occurred, and the drug should not be given to anyone who has had a serious allergic reaction to it. The label also warns about kidney toxicity: it was seen in animal studies but not in the clinical trials, and potentially fatal glomerulonephritis has occurred with some other antisense drugs, so kidney function is checked before and during treatment.
▸How is Golodirsen taken?
Given as a weekly intravenous infusion over 35-60 minutes. Administered at infusion centers or potentially at home after initial doses.
▸Is Golodirsen FDA approved?
Yes, Golodirsen (Vyondys 53) is FDA approved (2019) for the treatment of Duchenne Muscular Dystrophy.
▸What is the difference between golodirsen and viltolarsen?
Both skip exon 53 and are applicable to the same subset of DMD patients (about 8%). They are made by different companies (Sarepta vs. NS Pharma) and have slightly different chemical structures. In clinical trials, viltolarsen showed 100% of patients producing increased dystrophin, while golodirsen data showed similar results. Both require weekly IV infusions. The choice between them may depend on insurance coverage, physician preference, and individual tolerability.
▸What is the kidney toxicity warning?
Golodirsen carries a warning for kidney toxicity. Kidney damage was seen in animal studies but not in the clinical trials, and potentially fatal glomerulonephritis (inflammation of the kidney filtering units) has been seen with some other antisense oligonucleotides. The label recommends a monthly urine dipstick and checks of serum cystatin C and urine protein every 3 months. Regular monitoring of kidney function through blood tests and urine protein analysis is required. Any signs of kidney problems, such as changes in urine output, swelling, or blood in the urine, should be reported to the physician immediately.
▸What percentage of DMD patients can use golodirsen?
Approximately 8% of DMD patients have mutations amenable to exon 53 skipping. Genetic testing is required to confirm that a patient's specific deletion would benefit from skipping exon 53 to restore the dystrophin reading frame.
▸Is golodirsen given for life?
Yes. Like all exon-skipping therapies, golodirsen requires ongoing weekly IV infusions to maintain its effect. The drug modifies RNA processing but does not alter the DNA, so its effect is temporary and continuous treatment is necessary.