Dear DMD Community,

We are pleased to share important new data on the longer-term safety and efficacy of DUVYZAT, which was published this week in the Annals of Clinical and Translational Neurology. The publication describes results from an ongoing open-label extension (no randomized comparison group) that follows 194 participants from earlier DUVYZAT studies, with some individuals being treated for up to 8 years. These findings are descriptive only and cannot predict individual results. Prior studies enrolled ambulant boys on background corticosteroids.

Overall, the safety and tolerability of long-term administration of givinostat in patients with DMD was consistent with previous studies. Comparisons with natural history data suggest that, in many patients, givinostat delays the occurrence of major disease progression milestones.

Please see Important Safety Information below and the full Prescribing Information

We know from countless conversations with families and advocacy partners that maintaining motor function for as long as possible is among the highest priorities. These new data reinforce our belief in DUVYZAT’s potential to help achieve that goal.

To learn more about today’s data announcement, please read our press release. For those interested in reading more about our findings, the full study can be accessed here.

Thank you for your continued partnership. Together, we are working toward achieving even more for the Duchenne community in the years ahead.

What is DUVYZAT?

DUVYZAT is a prescription medicine 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 age 6.

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, so having fewer can increase your risk of bleeding or bruising. Your doctor will check your blood count before you start DUVYZAT and regularly during treatment for signs of thrombocytopenia. Call your doctor right away if you notice unusual bleeding or small red or purple spots on the skin.
• Increased levels of fat (triglycerides) in your blood. You may not have any symptoms, so your doctor will do blood tests before you start DUVYZAT and regularly during treatment to check your triglyceride levels.
• 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 doctor.
• If thrombocytopenia, increased triglycerides, or diarrhea cannot be managed, your doctor may change your dose or stop your treatment with DUVYZAT, if needed.

Before taking DUVYZAT, tell your doctor about all of your medical conditions, including:

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

Tell your doctor 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 doctor.

What are the possible side effects of DUVYZAT?

• DUVYZAT can cause serious side effects, including 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 doctor right away if you feel faint, have an irregular heartbeat, feel dizzy, or lose consciousness. See the section titled “What is the most important information I should know about DUVYZAT?” for more information about side effects.

The most common side effects (occurring in >5% of DUVYZAT-treated patients) included diarrhea, abdominal pain, low platelet levels, nausea/vomiting, high triglyceride levels, elevated temperature/fever, muscle aches, rash, joint pain, fatigue, constipation and decreased appetite.

These are not all of the possible side effects of DUVYZAT. For more information, ask your doctor 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.

DUVYZAT is a registered trademark of Italfarmaco S.p.A.

Warmly,
Caroline Allen
Head of Patient Advocacy, ITF Therapeutics