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HDAC may play a key role in regulating muscle homeostasis1-6

The potential role of HDAC in normal muscle repair1-3

HDACs are thought to be important for regulating muscle homeostasis. Through nuclear and cytoplasmic enzymatic activity, HDACs modify both histone and nonhistone proteins. They may play a key role in maintaining and repairing muscle tissue by modifying proteins that regulate muscle fiber repair pathways.

HDAC overactivity in DMD2,4,5

In DMD, HDAC is overactive.

Increased HDAC activity, in combination with the structural instability of dystrophin-deficient muscle cells, may disrupt the process of normal muscle repair and accelerate the deterioration of tissue.

HDAC upregulation may drive2,4,5:

  • Activation of chronic inflammatory pathways
  • Impairment of muscle repair
  • Fibrogenesis and adipogenesis
  • Muscular atrophy

While the exact mechanism is unknown,

DUVYZAT targets HDAC overactivity—thought to be a key pathologic process in DMD1-6

How DUVYZAT may work5,6

DUVYZAT is a pan-HDAC inhibitor.

While its exact mechanism is unknown, DUVYZAT is designed to target HDAC, reduce its enzymatic activity, and address the key pathologic process of HDAC overactivity.

How DUVYZAT may work5,6

DUVYZAT is a pan-HDAC inhibitor.

While its exact mechanism is unknown, DUVYZAT is designed to target HDAC, reduce its enzymatic activity, and address the key pathologic process of HDAC overactivity.

Explore the efficacy and safety results from the pivotal clinical trial.
HDAC, histone deacetylase.

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References: 1. Yang C, Croteau S, Hardy P. Histone deacetylase (HDAC) 9: versatile biological functions and emerging roles in human cancer. Cell Oncol (Dordr). 2021;44(5):997-1017. 2. Sandonà M, Cavioli G, Renzini A, et al. Histone deacetylases: molecular mechanisms and therapeutic implications for muscular dystrophies. Int J Mol Sci. 2023;24(5):4306. 3. Seto E, Yoshida M. Erasers of histone acetylation: the histone deacetylase enzymes. Cold Spring Harb Perspect Biol. 2014;6(4):a018713. 4. Mercuri E, Vilchez JJ, Boespflug-Tanguy O, et al; EPIDYS Study Group. Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial. Lancet Neurol. 2024;23(4):393-403. 5. Consalvi S, Saccone V, Giordani L, Minetti G, Mozzetta C, Puri PL. Histone deacetylase inhibitors in the treatment of muscular dystrophies: epigenetic drugs for genetic diseases. Mol Med. 2011;17(5-6):457-465. 6. DUVYZAT. Prescribing information. ITF Therapeutics; 2024.

IMPORTANT SAFETY INFORMATION

Warnings and Precautions

  • Hematological Changes: DUVYZAT can cause dose-related thrombocytopenia and other signs of myelosuppression. Monitor blood count every 2 weeks for the first 2 months, at month 3, and every 3 months thereafter. Modify the dosage for confirmed thrombocytopenia. Discontinuation may be needed if abnormalities worsen.
  • Increased Triglycerides: DUVYZAT can cause elevations in triglycerides. Monitor triglycerides at 1 month, 3 months, 6 months, and then every 6 months thereafter. Modify the dosage if fasting triglycerides are verified >300 mg/dL. Treatment with DUVYZAT should be discontinued if triglycerides remain elevated despite adequate dietary intervention and dosage adjustment.
  • Gastrointestinal Disturbances: Gastrointestinal disturbances, including diarrhea, nausea/vomiting, and abdominal pain were common adverse reactions in DUVYZAT clinical trials. Antiemetics or antidiarrheal medications may be considered during treatment with DUVYZAT. Modify the dosage of DUVYZAT in patients with moderate or severe diarrhea and discontinue treatment if significant symptoms persist.
  • QTc Prolongation: DUVYZAT can cause prolongation of the QTc interval. Avoid use of DUVYZAT in patients who are at an increased risk for ventricular arrhythmias (including torsades de pointes), such as those with congenital long QT syndrome, coronary artery disease, electrolyte disturbance or in patients taking concomitant medicinal products known to cause QT prolongation. Obtain ECGs prior to initiating treatment with DUVYZAT in patients with underlying cardiac disease or in patients who are taking concomitant medications that cause QT prolongation.

Adverse Reactions

The most common adverse reactions reported in >5% of patients treated with DUVYZAT are diarrhea (37%), abdominal pain (34%), thrombocytopenia (33%), nausea/vomiting (32%), hypertriglyceridemia (23%), pyrexia (13%), myalgia (9%), rash (9%), arthralgia (8%), fatigue (8%), constipation (7%), and decreased appetite (7%).

Drug Interactions

Closely monitor when DUVYZAT is used in combination with an oral CYP3A4 sensitive substrate or a sensitive substrate of the OCT2 transporter, for which a small change in substrate plasma concentrations may lead to serious toxicities.

Avoid concomitant use with other drugs that prolong the QTc interval; monitor ECG if concomitant use cannot be avoided. If concomitant use cannot be avoided, obtain ECGs when initiating, during concomitant use, and as clinically indicated. Withhold DUVYZAT if the QTc interval is >500 ms or the change from baseline is >60 ms.

INDICATION

DUVYZAT is a histone deacetylase inhibitor indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients 6 years of age and older.

To report SUSPECTED ADVERSE REACTIONS, contact ITF Therapeutics LLC at 1-833-582-4312 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Please see full Prescribing Information for additional safety information.

IMPORTANT SAFETY INFORMATION

Warnings and Precautions

  • Hematological Changes: DUVYZAT can cause dose-related thrombocytopenia and other signs of myelosuppression. Monitor blood count every 2 weeks for the first 2 months, at month 3, and every 3 months thereafter. Modify the dosage for confirmed thrombocytopenia. Discontinuation may be needed if abnormalities worsen.
  • Increased Triglycerides: DUVYZAT can cause elevations in triglycerides. Monitor triglycerides at 1 month, 3 months, 6 months, and then every 6 months thereafter. Modify the dosage if fasting triglycerides are verified >300 mg/dL. Treatment with DUVYZAT should be discontinued if triglycerides remain elevated despite adequate dietary intervention and dosage adjustment.
  • Gastrointestinal Disturbances: Gastrointestinal disturbances, including diarrhea, nausea/vomiting, and abdominal pain were common adverse reactions in DUVYZAT clinical trials. Antiemetics or antidiarrheal medications may be considered during treatment with DUVYZAT. Modify the dosage of DUVYZAT in patients with moderate or severe diarrhea and discontinue treatment if significant symptoms persist.
  • QTc Prolongation: DUVYZAT can cause prolongation of the QTc interval. Avoid use of DUVYZAT in patients who are at an increased risk for ventricular arrhythmias (including torsades de pointes), such as those with congenital long QT syndrome, coronary artery disease, electrolyte disturbance or in patients taking concomitant medicinal products known to cause QT prolongation. Obtain ECGs prior to initiating treatment with DUVYZAT in patients with underlying cardiac disease or in patients who are taking concomitant medications that cause QT prolongation.

Adverse Reactions

The most common adverse reactions reported in >5% of patients treated with DUVYZAT are diarrhea (37%), abdominal pain (34%), thrombocytopenia (33%), nausea/vomiting (32%), hypertriglyceridemia (23%), pyrexia (13%), myalgia (9%), rash (9%), arthralgia (8%), fatigue (8%), constipation (7%), and decreased appetite (7%).

Drug Interactions

Closely monitor when DUVYZAT is used in combination with an oral CYP3A4 sensitive substrate or a sensitive substrate of the OCT2 transporter, for which a small change in substrate plasma concentrations may lead to serious toxicities.

Avoid concomitant use with other drugs that prolong the QTc interval; monitor ECG if concomitant use cannot be avoided. If concomitant use cannot be avoided, obtain ECGs when initiating, during concomitant use, and as clinically indicated. Withhold DUVYZAT if the QTc interval is >500 ms or the change from baseline is >60 ms.

INDICATION

DUVYZAT is a histone deacetylase inhibitor indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients 6 years of age and older.

To report SUSPECTED ADVERSE REACTIONS, contact ITF Therapeutics LLC at 1-833-582-4312 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Please see full Prescribing Information for additional safety information.