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Dan O'Connell, VP, Head of US Marketing, ITF Therapeutics

Fraught with Treatment Decisions: How Can We Help?

By Dan O'Connell

VP, Head of US Marketing

11/15/24

I can only imagine what it must feel like to be responsible for treatment decisions on behalf of someone who lives with Duchenne muscular dystrophy (DMD). Whether that person is yourself or someone you love, the stakes are unspeakably high. And it may feel like you don't know how to make the "right" decision.

"The more we hear from you, the better we will understand how the data and numbers may come to life in the context of your lived experience."

As the Head of Marketing at ITF Therapeutics, it is my fervent hope that my team and I can help with this intense situation. We believe there is no uniform, "right" answer for everyone. Instead, we support and foster individualized, shared decision making among doctors, patients, and caregivers. We aim to raise awareness of DUVYZAT™ (givinostat) as a treatment option and to equip physicians and patients with information. Our ultimate goal is to provide each and every individual with the resources they need to make the best possible treatment choices.

To that end, I try to make sure that the information we disseminate is grounded in the context of authentic lived experiences. Of course, I expect our Key Account Managers – the representatives who visit physician's offices to educate medical professionals about DUVYZAT – to be well versed in the data. But it's also critical for them to understand how the data relates to the real life of a person living with DMD. For example, clinical trial results are often based on functional measures like the 4-stair climb, 6 Minute Walk Test, and the Performance of Upper Limb test. But what relevance do these measures have in real life? The more people I meet who live with DMD, the more I understand that everyday experiences like going fishing, riding a bike, or walking around town after school on a half-day can be seminal activities for individuals with DMD. When we educate physicians about DUVYZAT, we want them to see not just scientific data points but also what this treatment might mean for a city-dwelling kid who desperately wants to keep up with his friends on a walk to the ice cream shop.

I am also intent on communicating how DUVYZAT works in a way that makes sense to everyone, especially patients and their families. How a medication works is known in drug development circles as its mechanism of action, or MOA for short. DUVYZAT's MOA is thought to inhibit (AKA reduce) histone deacetylase. That doesn't exactly roll off the tongue, so we often refer to this type of medication as an "HDAC inhibitor." That makes pronouncing it easier, but explaining it is another whole ball of wax. I am committed to coming up with new and better ways to explain, describe, and illustrate the concept until every single person who needs the information can access it in a digestible, easy-to-understand format. I have adopted this famous Da Vinci quote as a personal mantra: "Simplicity is the ultimate sophistication."

While we have a lot of valuable information to share with you, we firmly believe that often patients and physicians learn the most from each other. So beyond delivering information to the DMD community, my team strives to facilitate conversations and incorporate the patient voice into what we do. The more we hear from you, the better we will understand how the data and numbers may come to life in the context of your lived experience. I am deeply grateful to those who have shared personal stories, even sensitive ones like a detailed explanation of a gastrointestinal situation. I know that stuff is not always easy to talk about. But the more we understand about your day-to-day lives, the better we will be able to partner with you and support you.

We are just getting started, so we have a lot of work to do. We always want our work to be informed by a strong connection with the community we serve. So please, let us know if you feel something that could support your treatment decisions is missing from the conversation.

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.

C-DUV-US-0140 11/2024