On April 25, 2025, ITF Therapeutics announced that the Committee for Medicinal Products (CHMP) of the European Medicines Agency (EMA) recommended granting a conditional marketing authorization for DUVYZAT® (givinostat) for the treatment of ambulant patients six years of age and older with Duchenne muscular dystrophy (DMD), and alongside concomitant corticosteroid treatment. This is encouraging news for individuals living with DMD in the European Union (EU) and their families as it brings them one step closer to accessing another treatment option, pending confirmation by the European Commission.
Please continue reading to view their full update.
Earlier today (April 25, 2025) Italfarmaco announced that the Committee for Medicinal Products (CHMP) of the European Medicines Agency (EMA) recommended granting a conditional marketing authorization for DUVYZAT® (givinostat) for the treatment of ambulant patients six years of age and older with Duchenne muscular dystrophy (DMD), and alongside concomitant corticosteroid treatment. This is encouraging news for individuals living with DMD in the European Union (EU) and their families as it brings them one step closer to accessing another treatment option, pending confirmation by the European Commission.
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. For more information about side effects associated with DUVYZAT, please see the Full Prescribing Information at www.DUVYZAT.com.
While the CHMP recommendation differs slightly from the FDA approval received in 2024, it is important to note that regulatory decisions in the EU will not affect the availability of DUVYZAT in the U.S. Regulatory agencies operate independently and make decisions based on their own assessments of the available evidence.
As part of its post-marketing commitments in the EU, Italfarmaco plans to collect observational real-world data and conduct a complementary clinical trial. Together, these efforts will further our understanding of the long-term impact of treatment with DUVYZAT across a broader range of patients and support its continued availability in the EU.
This progress in the EU also builds on similar developments in other parts of the world. In December 2024, the UK Medicines and Healthcare products Regulatory Agency approved DUVYZAT for patients six years of age and older who are ambulatory and granted conditional marketing approval for non-ambulatory patients.
Our team at ITF Therapeutics looks forward to our continued collaboration with Jett Foundation to help meet the needs of individuals living with Duchenne in the U.S. If you have any questions related to today’s news from Italfarmaco, please do not hesitate to reach out.
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.
