ITF Therapeutics

Hello! My name is Caroline Allen, and I am the Head of Patient Advocacy and Communications at ITF Therapeutics. It is my pleasure to welcome you to the ITF Therapeutics Team Blog. We wanted to connect with people in the DMD community in a way that feels more personal than the usual corporate modes of communication. We will take turns writing about what we think may be helpful or interesting to you as you make your way through life with Duchenne. We will share what we do all day at work, explain new developments and what they may mean in the context of real life, and break down topics that may seem heavily scientific or otherwise difficult to sort through and make sense of.

As much as we want to introduce ourselves and keep you updated about key developments here at ITF Therapeutics, we also want to get to know you better. Your insights have played a central role in advancing our research and our effort to bring a new treatment to the community, and with your help we can make sure that we are always improving our ability to support you and meet your needs. In our blog posts we will often ask you for your opinions and questions – and we want to hear from you!

So please consider this blog as a two-way street, a place where we can learn more about each other and keep learning from each other. The more we share insights, the better we will get at making this world a better place for all people affected by DMD.

Caroline

A Good Day

By Caroline Allen

Head, US Patient Advocacy & Communications

7/25/24

In the long path to new treatments for Duchenne, today is a good day. DUVYZAT™ (givinostat), a new medicine that is designed to interfere with Duchenne’s relentless progression, is now officially available in the United States. While I am feeling positive and hopeful because of this news, I am also mindful that we must commit ourselves to faster and better progress going forward for all people with DMD. If there is one thing I have learned from this incredibly brave, resilient, and determined community, “urgent” just barely hints at how critical the need is for more treatments to slow or stop the decline caused by DMD.

Our main clinical study, EPIDYS, included 179 patients – one of the largest trials in DMD to date – so a good deal of data exists to inform treatment decisions. In the study, 179 ambulant boys six years of age or older received glucocorticosteroid treatment, and either DUVYZAT twice daily or placebo. DUVYZAT is the eighth FDA-approved therapy for Duchenne. While recent advancements bring us closer to a day when every patient will have access to safe and effective treatments, a busy treatment landscape can also make some decisions more complicated. I thought you might feel bombarded with information overload, so I compiled a “Top Ten” list of important things to know about today’s news.

Here are my Top Ten things you need to know about DUVYZAT:

First of all, it’s pronounced DOO-vee-zat.
The treatment is approved in the United States for people with DMD who are at least six years old. There is no limitation based on ambulatory status or mutation.
It is also referred to as givinostat, which you may have heard about when it was in clinical trials.
The medicine is only available by doctor’s prescription, so talk to your doctor if you want to learn more about the treatment and consider whether it may be right for you or your loved one.
It is available in liquid form and is administered twice a day.
ITF Therapeutics has set up a special support program to help patients and families make informed decisions about treatment with DUVYZAT. The program is called ITF ARC (for Access, Resources and Care), and it helps with navigating insurance coverage, working with the specialty pharmacist, and accessing educational resources. For more information about ITF ARC, click here.
We have “field reps” who are working in all areas of the U.S. to help ensure that physicians who treat patients with DMD, like the ones you see, have the information they need about DUVYZAT so they can answer your questions and determine if the treatment is right for you.
DUVYZAT is not yet available in any other countries, but we are working on that.
You are part of an incredibly dedicated community. Many DMD families sacrificed precious time and energy to participate in the clinical trials that led to this momentous day. We are forever grateful to every family member who contributed to this progress.
Your advocacy leaders have blown me away! As we have prepared to launch DUVYZAT, we have worked closely with many pioneers who are working extremely hard and making a huge difference. They helped us design ITF ARC (see #6) to make sure we are providing the customized support you may need throughout your treatment experience.

When considering treatment, ask your doctor how patients treated with DUVYZAT performed on tests such as the four-stair climb compared to those who got the placebo. It is also important to review the safety information, as there are potential side effects for you to understand. Every individual is unique and every decision is personal, so be sure to discuss these topics with your physician and understand what they could mean for you or your loved one.

With so much information flying around the news and social media, I hope my Top Ten list is helpful. I plan to post new blog entries in the coming weeks and months, so drop me a line if there’s a topic you would like me to cover!

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-0012 07/2024