Duchenne muscular dystrophy (DMD) is a genetic disorder that mainly affects boys. DMD is usually diagnosed in early childhood and tends to worsen over time. DMD happens when a change (mutation) in the DMD gene prevents the body from making enough of an important muscle protein called dystrophin.
DMD treatment options are available to help manage symptoms and slow muscle damage. Steroids (corticosteroids) are a standard DMD treatment used to help people stay strong and active for as long as possible. However, long-term steroid use leads to many unwanted side effects.
Agamree (vamorolone) is a once-daily oral medicine that is FDA-approved to treat DMD. It’s a newer type of steroid that may be easier to take over the long term. This article covers frequently asked questions about Agamree for DMD.
Is Agamree Right for Me?
A health care provider can help decide if this medicine is right for you based on your age, health history, and current treatments. Agamree is FDA-approved to treat DMD in children aged 2 years or older.
Before you start Agamree, any routine vaccinations should be up to date. Some types of vaccines (live vaccines) should be given at least four to six weeks before starting Agamree. Your health care team can review your immunization records and let you know if any vaccines are due.
If you are already taking a steroid, such as prednisone, you may be able to switch to Agamree if your health care provider recommends it.
How Does Agamree Work for DMD?
Dystrophin is a protein that normally helps protect muscle cells. When dystrophin is missing or does not work properly, muscle cells become fragile. Muscles are more easily damaged and do not repair well. Over time, this leads to ongoing inflammation and muscle loss.
Agamree is a type of steroid medicine. It works by attaching to steroid receptors in the body that help control inflammation and immune system activity. This calms inflammation, which is thought to be the main way Agamree works for treating DMD.
How Do I Take Agamree?
Agamree is usually taken once daily. It is best to take it with a meal. Your health care provider will calculate the exact dose for you based on your body weight. Your dose may be adjusted if you take certain other medicines or have liver problems.
Your health care provider will monitor body weight and overall response to treatment. Let them know if you are having side effects. They may lower the dose if the medicine is not well tolerated.
It is important not to stop Agamree suddenly without medical guidance. If you need to stop Agamree, your health care provider may tell you to slowly lower your dose before stopping.
Agamree comes as an orange-flavored oral liquid in a glass bottle. Shake the bottle well for about 30 seconds before each use. The medicine comes with two oral dosing syringes. Use an oral syringe to measure each dose.
For detailed steps on how to use Agamree, see the Instructions for Use guide that comes with your medicine. If you have questions about how to measure the proper dose, your health care provider or pharmacist can help.
How Was Agamree Studied for DMD?
Agamree was studied in a 24-week clinical study done at multiple sites across several countries. The study included 121 boys aged 4-7 years with DMD. The average age was around 5 years old when the study began, and all participants could walk and had not used steroid treatment before.
Most participants were White or Caucasian (83%). About 10% were Asian, 2% were Black or African American, 1% were American Indian/Alaska Native, and the rest identified as multiple races or unknown races. About 4% of participants were of Hispanic or Latino ethnicity, and 96% were not.
The boys were randomly placed into one of four groups. Each group received either a lower dose of Agamree, a higher dose of Agamree, prednisone, or a placebo (which contains no active medicine). During the study, neither the families nor their doctors knew which treatment each child was receiving.
The main goal of the study was to measure the change in muscle strength from the start of the study to week 24 by comparing the children taking Agamree to those taking a placebo. Muscle strength was measured using a test that timed how long it took participants to go from lying on the floor to standing upright. Standing up faster shows better muscle strength.
Researchers also compared other changes in ability from the start to the end of the study, such as how far the boys could walk in six minutes and how fast they could run (or walk) a short distance.
What Benefits of Agamree Were Seen in Studies?
At the start of the study, all participants had similar results on standing, walking, and speed tests. By the end of the study, children taking Agamree improved their time on the stand-up test, while those taking a placebo did not.
The table below summarizes some of the main results at week 24, on average. Positive numbers (+) show improvement by the end of the study; negative numbers (–) show a decline.
Test | Placebo | Agamree (lower dose) | Agamree (higher dose) |
|---|---|---|---|
| Time to stand up from the floor | –0.012 rises/sec | +0.033 rises/sec | +0.048 rises/sec |
| Distance walked in 6 minutes | –14 meters (m) | +27 m | +29 m |
| Running/walking speed | +0.014 m/sec | +0.141 m/sec | +0.258 m/sec |
As the table shows, children taking the higher dose of Agamree had better results across all three tests (stand up, distance, speed) compared to those taking a placebo. These differences were statistically significant, meaning the improvements were unlikely to be due to chance. Children taking the lower dose of Agamree also had significantly better results than placebo for the time-to-stand and six-minute walk tests, but the difference in walking speed was not large enough to be certain it wasn’t due to chance.
Overall, the study found that Agamree improved the ability to stand up, walk, and run in children with DMD. This suggests that Agamree may help improve muscle strength and function in some children with DMD. Your results may be different from clinical studies.
How Fast Does Agamree Start to Work?
Agamree starts working in the body right away, but it may take several weeks to notice it working. In the 24-week clinical study, children taking the higher dose of Agamree saw improvement as early as week 6 and continued through week 24. Children taking the lower dose of Agamree took closer to 12 weeks to start to see improvement.
What Can I Do to Prevent or Manage Side Effects of Agamree?
Like other medicines, Agamree can cause side effects. Not everyone will have the same effects, and many are manageable. A health care provider can help monitor for side effects and suggest ways to reduce them.
The most common Agamree side effects include facial puffiness (cushingoid appearance), psychiatric disorders, vomiting, weight gain, and vitamin D deficiency. Most of these side effects are more likely to happen if you take a higher dose. Your health care provider may lower your dose if necessary to help manage side effects.
Changes in appearance and weight. The most common side effect of Agamree is changes in your appearance (cushingoid features), such as facial puffiness, weight gain, acne, and fat buildup on the back of the neck. Appetite may increase soon after starting treatment. Extra weight can make movement harder, so regular weight checks are important. Also, it is advised to limit high-calorie foods and sugary snacks and focus on a balanced diet. Staying as active as possible can also help. Limiting salty foods may also help with puffiness. Acne can often be managed by consistently washing your face twice daily with a gentle cleanser and using over-the-counter topical treatments.
Mood and behavior changes. Agamree may affect mood or behavior. Changes can include irritability, trouble sleeping, mood swings, or feeling anxious or sad. Let a health care provider know if these changes affect daily life. They may suggest mental health support or adjust treatment if needed.
Bone health and vitamin levels. Long-term use can affect bones and vitamin D levels, and children with DMD already have a higher risk of low bone density. Health care providers often monitor bone health and vitamin D levels. They may recommend calcium and vitamin D supplements.
Eye health. Agamree can raise the risk of cataracts or eye pressure changes. Regular eye exams help catch problems early.
Infection risk. Agamree can make it harder for the body to fight infections. Washing hands often, avoiding close contact with people when they are ill, and regularly disinfecting high-touch surfaces can help lower the risk. Always report signs of infection to a health care provider.
This is not a full list of possible side effects. Talk with your health care provider if you are having symptoms that bother you.
What Do We Know About Long-Term Effects of Agamree?
After the 24-week study ended, some participants who had taken prednisone or a placebo switched to Agamree and continued in the next, longer-term phase of the study.
The study found that improvements in muscle function seen with the higher dose of Agamree after 24 weeks of treatment continued through 48 weeks. The higher dose helped maintain benefits better than the lower dose for most of the measures of muscle function.
Children who switched from a placebo to Agamree also improved quickly. Growth and bone health improved after switching from prednisone to Agamree. Overall, the study showed that Agamree helped maintain muscle function and had a better safety profile over longer treatment.
Is There Any Cost Assistance Available for Agamree?
Financial support programs are available that may help lower the cost of Agamree. Your health care provider can help you enroll to see if you qualify for patient assistance or copay savings. You can also visit this website or call 833-422-8259 for more information.