Proven to protect muscle strength and function

DUVYZAT was studied in one of the largest Duchenne clinical trials to date

  • 18 months long
  • Participants received steroids along with either DUVYZAT or placebo
  • Included 179 participants* with a wide range of genetic variations of Duchenne

*The median age was 9.8 years, meaning that half the participants were younger than 9.8 years old, and half were older.

Ethan, taking DUVYZAT for 4+ years.
Ethan, taking DUVYZAT for 4+ years.
Ryan, taking DUVYZAT for 4+ years.

Ryan, taking DUVYZAT for 4+ years.

A clinical trial is a research study that tests new medicines to make sure they are safe and effective.

SEE WHAT THE RESEARCHERS LOOKED AT

4-stair climb

4-stair climb

How fast participants could climb 4 stairs before and after 18 months of using DUVYZAT and steroids, compared to steroids alone

NSAA

NSAA

Participants' mobility and muscle function across multiple muscle groups as measured by the North Star Ambulatory Assessment (NSAA)

Muscle Strength

Muscle strength

A muscle strength test that included how well participants could bend their elbow and straighten their knee

Fat tissue

Fat tissue

How much muscle was preserved compared to replaced by fat, a measurement of muscle loss

The NSAA is a test that helps healthcare providers understand how well people with Duchenne can move and do daily activities by watching them perform specific tasks.
Dive image
Ryan, taking DUVYZAT for 4+ years.

DUVYZAT protects from Duchenne progression

In the clinical trial, DUVYZAT and steroids were more effective at preserving muscle strength and function than steroids alone.

DUVYZAT helped maintain muscle strength and motor function about

40%

BETTER THAN USING STEROIDS ALONE

As measured by the mean change in time from baseline to 18 months of 4-stair climb in DUVYZAT vs steroids alone (placebo).

More measurements of protection§

After 18 months, participants taking DUVYZAT and steroids:

  • Maintained motor function abilities longer compared to those who took placebo, based on NSAA scores

  • Maintained more ability to bend their elbow or straighten their knee, a measure of muscle strength

DUVYZAT also reduced how much muscle was replaced by fat, a measurement of muscle preservation.

§After clinical trials, researchers use statistics to see if the results are really from the new medicine and not just by chance. In this trial, people taking DUVYZAT showed improvements in NSAA scores and muscle flexibility. However, the analysis wasn't able to prove that these results were due to the treatment.

Possible side effects of DUVYZAT

DUVYZAT was studied in multiple clinical trials involving 222 participants, many of whom were taking DUVYZAT for more than 2 years. Talk to your healthcare provider about how best to manage side effects if they do occur.

Most common side effects in the phase 3 clinical trial

  • Diarrhea (37%)
  • Abdominal pain (34%)
  • Low platelet levels (33%)
  • Nausea/vomiting (32%)
  • High triglyceride levels (23%)
  • Elevated temperature/fever (13%)

For patients who experienced diarrhea, it usually occurred in the first few weeks of treatment. Most of the time it was mild and manageable. Very few people had severe cases. Only one person needed to pause their treatment due to diarrhea, but no one stopped taking DUVYZAT because of it

Experienced by ≥10% of participants.

Your healthcare provider will monitor you or your loved one for changes in blood counts.

If side effects occur, your healthcare provider can adjust the dose to help reduce them.

Talk to your healthcare provider about trying DUVYZAT today.

Indication and Important Safety Information

What is DUVYZAT?

DUVYZAT is a prescription medicine that is used for the treatment of Duchenne muscular dystrophy (DMD) in people 6 years of age and older.

It is not known if DUVYZAT is safe and effective in children under 6 years of age.

Important Safety Information

What is the most important information I should know about DUVYZAT?

  • Low platelet counts in your blood (thrombocytopenia). Platelets are important for blood clotting, and a decrease in their numbers can lead to an increased risk of bleeding or bruising. Your healthcare provider will check your blood count before you start DUVYZAT and regularly during treatment for any signs of thrombocytopenia. Call your healthcare provider right away if you notice any unusual bleeding or small red or purple spots on the skin called petechiae. Your healthcare provider may change your dose of DUVYZAT if your blood platelet counts continue to be low or may stop your treatment with DUVYZAT.
  • Increased levels of fat (triglycerides) in your blood. You may not have any symptoms, so your healthcare provider will do blood tests before you start DUVYZAT and regularly during treatment to check your triglyceride levels. Your healthcare provider may change your dose of DUVYZAT if your triglyceride levels continue to be high or may stop your treatment with DUVYZAT.
  • Frequent watery loose stools (diarrhea) and vomiting. DUVYZAT can cause vomiting and moderate to severe diarrhea. If diarrhea occurs, you should keep track of the frequency and severity of your diarrhea symptoms, drink plenty of fluids, and contact your healthcare provider. Your healthcare provider may change your dose of DUVYZAT if the diarrhea cannot be managed or does not go away. Your healthcare provider may also stop your treatment with DUVYZAT.

Before taking DUVYZAT, tell your healthcare provider about all of your medical conditions, including if you:

  • have any heart problems or if you take any medicines that could increase your chance for irregular heart rhythms.
  • have any bleeding problems.

Tell your healthcare provider about all of the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

Taking DUVYZAT with certain other medicines may affect each other. Taking DUVYZAT with other medicines can cause serious side effects. Do not start or stop other medicines without talking to your healthcare provider.

DUVYZAT can cause serious side effects, including:

  • See “What is the most important information I should know about DUVYZAT?”
  • changes in the electrical activity of your heart called QT Prolongation. QT Prolongation can increase the risk of developing a type of irregular heart rhythm known as Torsades de Pointes. Call your healthcare provider right away if you feel faint, have an irregular heartbeat, feel dizzy, or lose consciousness.

The most common side effects of DUVYZAT included diarrhea, nausea, vomiting, stomach pain, low platelet counts in the blood, increased fat level in the blood, and fever.

These are not all of the possible side effects of DUVYZAT. For more information, ask your healthcare provider or pharmacist.

Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Please see full Prescribing Information and Medication Guide.

Indication and Important Safety Information

What is DUVYZAT?

DUVYZAT is a prescription medicine that is used for the treatment of Duchenne muscular dystrophy (DMD) in people 6 years of age and older.

It is not known if DUVYZAT is safe and effective in children under 6 years of age.

Important Safety Information

What is the most important information I should know about DUVYZAT?

  • Low platelet counts in your blood (thrombocytopenia). Platelets are important for blood clotting, and a decrease in their numbers can lead to an increased risk of bleeding or bruising. Your healthcare provider will check your blood count before you start DUVYZAT and regularly during treatment for any signs of thrombocytopenia. Call your healthcare provider right away if you notice any unusual bleeding or small red or purple spots on the skin called petechiae. Your healthcare provider may change your dose of DUVYZAT if your blood platelet counts continue to be low or may stop your treatment with DUVYZAT.
  • Increased levels of fat (triglycerides) in your blood. You may not have any symptoms, so your healthcare provider will do blood tests before you start DUVYZAT and regularly during treatment to check your triglyceride levels. Your healthcare provider may change your dose of DUVYZAT if your triglyceride levels continue to be high or may stop your treatment with DUVYZAT.
  • Frequent watery loose stools (diarrhea) and vomiting. DUVYZAT can cause vomiting and moderate to severe diarrhea. If diarrhea occurs, you should keep track of the frequency and severity of your diarrhea symptoms, drink plenty of fluids, and contact your healthcare provider. Your healthcare provider may change your dose of DUVYZAT if the diarrhea cannot be managed or does not go away. Your healthcare provider may also stop your treatment with DUVYZAT.

Before taking DUVYZAT, tell your healthcare provider about all of your medical conditions, including if you:

  • have any heart problems or if you take any medicines that could increase your chance for irregular heart rhythms.
  • have any bleeding problems.

Tell your healthcare provider about all of the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

Taking DUVYZAT with certain other medicines may affect each other. Taking DUVYZAT with other medicines can cause serious side effects. Do not start or stop other medicines without talking to your healthcare provider.

DUVYZAT can cause serious side effects, including:

  • See “What is the most important information I should know about DUVYZAT?”
  • changes in the electrical activity of your heart called QT Prolongation. QT Prolongation can increase the risk of developing a type of irregular heart rhythm known as Torsades de Pointes. Call your healthcare provider right away if you feel faint, have an irregular heartbeat, feel dizzy, or lose consciousness.

The most common side effects of DUVYZAT included diarrhea, nausea, vomiting, stomach pain, low platelet counts in the blood, increased fat level in the blood, and fever.

These are not all of the possible side effects of DUVYZAT. For more information, ask your healthcare provider or pharmacist.

Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Please see full Prescribing Information and Medication Guide.