Vyondys 53
Vyondys 53 (golodirsen) is an injectable medication that's used for a rare, genetic condition called Duchenne Muscular Dystrophy (DMD). It was approved by the FDA through its Accelerated Approval Program. Vyondys 53 (golodirsen) is given as an intravenous (IV) infusion by a healthcare provider. Your child will need routine blood and urine test to check for kidney problems when receiving this medication.
What is Vyondys 53 (golodirsen)?
What is Vyondys 53 (golodirsen) used for?
- Treatment of Duchenne Muscular Dystrophy (DMD) in people with a specific genetic mutation
How Vyondys 53 (golodirsen) works
Vyondys 53 (golodirsen) is an antisense oligonucleotide. It attaches to exon 53, a part of the gene that makes dystrophin. Dystrophin is a protein that keeps muscle cells intact and strong. In people with DMD, this protein is missing because of a mutation in exon 53. Vyondys 53 (golodirsen) works by exon skipping, meaning it cuts out exon 53 and its mutation from the finished protein. This helps to make a shorter but more functional dystrophin, which potentially provides strength to muscles and relief from symptoms of DMD.
Drug facts
Side effects of Vyondys 53 (golodirsen)
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
- Headache (41%)
- Fever (41%)
- Fall (29%)
- Belly pain (27%)
- Common cold (e.g., sneezing, runny nose, sore throat; 27%)
- Cough (27%)
- Vomiting (27%)
- Nausea (20%)
Less Common Side Effects
- Pain around the injection site
- Back pain
- Bruising
- Diarrhea
- Dizziness
- Flu-like symptoms (body aches, tiredness)
- Sore throat
- Stuffy nose
- Ear infection (ear pain, hearing loss)
- Seasonal allergy (itching eyes, sneezing)
- Fast heartbeat
- Constipation
- Fracture
- Skin tear
Vyondys 53 (golodirsen) serious side effects
Contact your healthcare provider immediately if you experience any of the following.
- Serious allergic reactions: fever, rash, itching, hives, skin peeling, skin irritation
The following Vyondys 53 (golodirsen) 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
- Kidney injury—decrease in the amount of urine, swelling of the ankles, hands, or feet
Side effects that usually do not require medical attention (report these to your care team if they continue or are bothersome):
- Cough
- Fever
- Headache
- Nausea
- Runny or stuffy nose
- Stomach pain
- Vomiting
Pros and cons of Vyondys 53 (golodirsen)
Pros
- Can help some people with DMD who never had a treatment option before
- Only needs to be injected once a week so it's convenient for parents with a busy schedule
- Doesn't interact with other medications or supplements
Cons
- Only available as a brand medication, so it might be expensive
- More likely to cause severe kidney problems
- Requires IV access, so it's not as convenient as taking a pill
Pharmacist tips for Vyondys 53 (golodirsen)
- It takes about 35 minutes to 1 hour to give the full dose of Vyondys 53 (golodirsen). Most commonly, your child will receive this medication at an infusion center or a provider's office. In some cases, it can be given at home by a home health nurse.
- It's best to get your Vyondys 53 (golodirsen) injection around the same day every week. If you miss an infusion, talk with your provider right away about rescheduling your appointment.
- If you have pain around the injection site after receiving Vyondys 53 (golodirsen), let your provider know. They might suggest using a topical cream on the area before injection to ease the pain and discomfort from the needle.
- Make sure your child gets routine blood and urine tests done on time while they are receiving Vyondys 53 (golodirsen). This is because this medication can cause kidney problems which can possibly be detected in these tests. By getting regular tests done, you and your child’s provider can continue to make sure this medication is safe for them.
- Tell your provider if your child experiences any changes in weight while receiving Vyondys 53 (golodirsen) because the dose might need to be changed.
Common questions about Vyondys 53 (golodirsen)
DMD is a rare, genetic condition that causes muscle weakness and wasting in young people. Your body needs a specific protein called dystrophin to keep muscle cells intact. In people with DMD, this protein is either missing or present at lower levels. Over time, this can cause movement problems (e.g., repeated falls, difficulty walking), heart problems (e.g., irregular heartbeat, heart failure), and stomach problems (e.g., constipation, diarrhea). Vyondys 53 (golodirsen) helps to raise the levels of dystrophin in certain people, providing relief from symptoms of DMD.
Vyondys 53 (golodirsen) is used in certain people with DMD that have a mutated exon 53, a specific part of a gene. It skips (cuts out) exon 53 to help the body make a shortened dystrophin protein that somewhat works. About 8% of young people with DMD are affected by this mutation. Typically, a genetic test that looks at changes in DNA can help identify your child's specific mutation. There are also other "exon skipping" medications available to treat different mutations in exons, such as Exondys 51 (eteplirsen) and Amondys 45 (casimersen). If you have any questions about whether Vyondys 53 (golodirsen) is right for your child, follow up with your child's provider for more information.
The FDA's Accelerated Approval Program allows a new medication to be approved faster than usual if it fulfills an unmet need or if it treats serious or life-threatening conditions. These approvals are based on lab measurements that are thought to predict health outcomes before the medication shows clear benefits. For Vyondys 53 (golodirsen), the medication raised the amount of dystrophin in skeletal muscles, which might predict better muscle control in certain people with DMD. After accelerated approval, the manufacturer is required to complete clinical studies to confirm beneficial health outcomes (in this case, if Vyondys 53 (golodirsen) improves muscle control and other symptoms in people with DMD). Depending on the results of these studies, the FDA can either give the medication full approval or pull it off the market.
It's not currently clear whether Vyondys 53 (golodirsen) can improve muscle control in certain people with DMD. As mentioned in the above FAQ, it's only known that the medication raises the amount of dystrophin. More clinical studies are still required to make sure this medication can help with muscle movement. Typically, physical therapy and gentle exercises (e.g., swimming, stretching) are recommended along with Vyondys 53 (golodirsen) to build muscle strength and prevent joint stiffening. Talk with your provider to see what exercises can benefit your child while receiving this medication.
Vyondys 53 (golodirsen) and Viltepso (viltolarsen) are both medications that cut out exon 53 in people with DMD. One main difference between the two medications is how fast you receive the medication. Vyondys 53 (golodirsen) is infused over 35 to 60 minutes, while Viltepso (viltolarsen) is infused over 60 minutes. Another main difference is the side effects. While Vyondys 53 (golodirsen) and Viltepso (viltolarsen) have many similar side effects, Vyondys 53 (golodirsen) is more likely to cause headache and fever. On the other hand, Viltepso (viltolarsen) is more likely to cause upper respiratory tract infections (e.g., runny nose, coughing, sneezing) and injection site reaction (e.g., bruising, redness, swelling). Talk with your child's provider to determine which medication works best for your child.
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Risks and warnings for Vyondys 53 (golodirsen)
Vyondys 53 (golodirsen) 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.
Allergic reaction
Vyondys 53 (golodirsen) can cause allergic reactions, which can be life-threatening. Watch out for signs and symptoms of allergic reactions, such as fever, rash, itching, hives, skin peeling, and skin irritation. Please let your provider know right away if allergic reactions occur. Your provider might slow the infusion or stop the treatment temporarily to help with your symptoms.
Kidney problems
- Risk factor: History of kidney problems
Studies showed that some animals that received Vyondys 53 (golodirsen) developed kidney problems, some serious enough to lead to kidney failure. While animal studies aren’t always representative of what happens in humans, people who received medications similar to Vyondys 53 (golodirsen) have also reported to have kidney problems that can be life-threatening.
You’ll need to get blood and urine tests done before you start treatment and while you receive Vyondys 53 (golodirsen) so your provider can check your kidney health. Please also talk with your provider if you or your child has face swelling, is urinating less than usual, or has brown urine while receiving Vyondys 53 (golodirsen) injections. These can be signs and symptoms of severe kidney problems.
Vyondys 53 (golodirsen) dosage
Typical dosage for Vyondys 53 (golodirsen)
Vyondys 53 (golodirsen) is an intravenous (IV) infusion that is administered by a healthcare provider. The dose depends on your weight and your provider will determine the appropriate dose for you each time.
In general, the typical dose is 30 mg/kg once a week injected into a vein.
