RegulationFast Track (United States), Orphan Drug (United States), Rare Pediatric Disease (United States), Orphan Drug (European Union), Conditional marketing approval (European Union), Priority Review (United States)

Structure/Sequence

Molecular FormulaC24H30ClN3O5

InChIKeyFKGKZBBDJSKCIS-UHFFFAOYSA-N

CAS Registry732302-99-7

Clinical Trials associated with Givinostat Hydrochloride

NCT06736223

/ RecruitingPhase 1

A Multicentric, Open-label, Non-randomized Study to Evaluate the Pharmacokinetic, Safety and Tolerability of ITF2357 Given as an Oral Single 50 mg Dose in Participants With Chronic Hepatic Impairment Relative to Matched Participants With Normal Hepatic Function

This is a multicentric, open-label, non-randomized study to evaluate the pharmacokinetic, safety and tolerability of ITF2357 in participants with chronic hepatic impairment relative to matched participants with normal hepatic function.

Start Date28 May 2025

Sponsor / Collaborator

Italfarmaco SpA

[+1]

NCT06769633

/ RecruitingPhase 2

A Phase 2 Open-label (Core Phase Plus Extension Phase) With 2 Cohorts Study to Assess the Pharmacokinetics and Safety of Givinostat in DMD Patients Ages From at Least 2 Years to Less Than 6 Years Old

This is a Phase 2 Open-label (Core Phase Plus Extension Phase) With 2 Cohorts Study to Assess the Pharmacokinetics and Safety of Givinostat in younger DMD Patients.
* Planned screening duration: approximately 4 weeks
* Planned Core Treatment duration: approximately 48 weeks
* Planned Extension Treatment duration: approximately 96 weeks
* Planned Follow Up duration: approximately 4 weeks (? 7 days)
* Total duration of study participation: up to 151 weeks (ie, 37-38 months)

Start Date02 Jan 2025

Sponsor / Collaborator

Italfarmaco SpA

[+1]

NCT06093672

/ RecruitingPhase 3

Randomized, Open-label, Multicenter Phase 3 Study to Assess the Efficacy and Safety of GIVinostat Versus Hydroxyurea IN JAK2V617F-positive High-risk Polycythemia Vera Patients: the GIV-IN PV TRIAL

The goal of this clinical trial is to compare the efficacy and safety of givinostat to hydroxyurea in Jak2V617F-positive high risk polycythemia vera patients.

Start Date26 Mar 2024

Sponsor / Collaborator

Italfarmaco SpA

[+1]

100 Clinical Results associated with Givinostat Hydrochloride

100 Translational Medicine associated with Givinostat Hydrochloride

100 Patents (Medical) associated with Givinostat Hydrochloride

255

Literatures (Medical) associated with Givinostat Hydrochloride

01 Oct 2025·BRAIN & DEVELOPMENT

Duchenne muscular dystrophy: Evolving therapeutic strategies and multidimensional evaluation approaches

Review

Author: Komaki, Hirofumi

Duchenne muscular dystrophy (DMD) is a severe X-linked muscular disorder caused by the absence of dystrophin. Standard therapies prolong survival; however, there is an increasing need for disease-modifying approaches and sensitive evaluation tools. This review presents a summary of the recent advances in DMD therapeutic strategies and multidimensional evaluation approaches, emphasizing their clinical applicability and future perspectives. In this study, a comprehensive review of the current literature was conducted focusing on mutation-specific therapies (exon skipping and gene therapy), non-mutation-specific pharmacological interventions (steroids, vamorolone, and histone deacetylase inhibitors), and emerging modalities such as cell-based therapy. The clinical outcome measures used across the disease stages were also examined, ranging from functional motor tests and imaging biomarkers to digital endpoints and quality of life (QOL) indices. Exon skipping and microdystrophin gene therapies have shown promising results in restoring partial dystrophin expression. Vamorolones and givinostat are alternative drugs with improved safety profiles. Time-based motor tests (such as time to sand, 10 m run) and digital biomarkers (Stride Velocity 95th Centile) enable the earlier detection of disease progression. Natural history data, including large registry-based datasets, enhance the interpretation of clinical trials. Patient-reported outcomes and disease-specific QOL measures, such as the DMD-QoL, are increasingly being incorporated to assess meaningful treatment benefits. The therapeutic landscape of DMD is evolving toward combination and personalized approaches. Multidimensional stage-specific evaluations are essential to capture functional changes and their therapeutic effects. Ongoing research on gene-, pharmacological-, and cell-based therapies, coupled with robust outcome assessments, may further improve patient care and QOL.

01 Oct 2025·BIOMEDICINE & PHARMACOTHERAPY

Givinostat inhibits in vitro differentiation of cardiac fibroadipogenic precursors from a mouse model of arrhythmogenic cardiomyopathy

Article

Author: Licandro, Simonetta Andrea ; Calore, Martina ; Fossati, Gianluca ; Forino, Monica ; Romualdi, Chiara ; Braghetta, Paola ; Vencato, Sara ; Romanato, Chiara ; Steinkuhler, Christian ; Rampazzo, Alessandra ; Velle, Angelo ; Facchinello, Nicola ; Vitiello, Libero

Arrhythmogenic cardiomyopathy (ACM) is a rare genetic cardiac disease and one of the leading causes of sudden cardiac death in young individuals and athletes. Current treatments focus on preventing arrhythmias and sudden cardiac death, but no effective therapy targeting disease progression is available. Myocardial replacement with fibro-fatty tissue, a hallmark of ACM, is primarily driven by the activation of cardiac fibroadipogenic precursors (cFAPs). Here, we evaluate the efficacy of a pan inhibitor of histone deacetylases (givinostat) in reducing cFAP proliferation and differentiation. These cells were found to be enriched in the hearts of a transgenic ACM murine model (Tg-hQ), which overexpresses the DSG2 p.Q558* mutation, compared to wild-type (WT) controls. We observed that givinostat reduced the proliferation of both Tg-hQ and WT isolated cFAPS. Additionally, cFAPS were induced to differentiate into adipocytes or fibroblasts and treated with givinostat. The drug treatment led to a reduction in the number of adipocytes under pro-adipogenic conditions. RNA sequencing analysis revealed a significant downregulation of key regulators of adipogenic differentiation and lipid metabolism (Pparg, Cebpa and Adipoq). In parallel, cells cultured under pro-fibrotic conditions showed decreased expression of genes encoding components of the extracellular matrix (Col1a1, Col6a1 and Postn) upon givinostat treatment. Overall, these results support the potential of givinostat in modulating cFAP proliferation and differentiation in vitro, warranting further in vivo studies to assess its impact on fibro-fatty tissue replacement in ACM.

01 Sep 2025·Molecular Therapy-Nucleic Acids

Epigenetic small molecule screening identifies a new HDACi compound for ameliorating Duchenne muscular dystrophy

Article

Author: Louie, Ke'ale W ; Farr, Gist H ; Maenza, Alyssa ; Paquette, Alison G ; Paguio, Alison ; Hasegawa, Eva H ; Henry, Clarissa A ; Ignacz, Amanda C ; Maves, Lisa

Duchenne muscular dystrophy (DMD) is the most common inherited muscle disease. There are currently few effective therapies to treat the disease, although many approaches are being pursued. Certain histone deacetylase inhibitors (HDACi) have been shown to ameliorate DMD phenotypes in mouse and zebrafish models, and the HDACi givinostat has recently gained FDA approval for DMD. Our goal was to identify additional HDACi, or other classes of epigenetic small molecules, that are beneficial for DMD. Using an established animal model for DMD, the zebrafish dmd mutant strain sapje, we screened a library of over 800 epigenetic small molecules. Our screening identified a new HDACi, SR-4370, that ameliorated dmd mutant zebrafish skeletal muscle degeneration, as well as additional HDACi that have previously been shown to improve dmd zebrafish. We find that a single early treatment of HDACi can ameliorate the muscle phenotype and increase lifespan in dmd zebrafish. Furthermore, we find that HDACi treatments that improve dmd muscle also cause increased histone acetylation in zebrafish larvae. Our results add to the growing evidence that HDACi are promising candidates for treating DMD. Our study also provides further support for the effectiveness of small molecule screening in dmd zebrafish.

News (Medical) associated with Givinostat Hydrochloride

24 Dec 2025

·www.businesswire.com

Italfarmaco and JCR Pharmaceuticals Announce Commercialisation Agreement for Givinostat in Japan and Strategic Collaboration in Rare Disease

MILAN & HYOGO, Japan--(BUSINESS WIRE)--Italfarmaco S.p.A. and JCR Pharmaceuticals Co., Ltd. (TSE 4552; “JCR”), today announced an exclusive licensing agreement for the development and commercialisation of givinostat in Japan. Under the terms of the agreement, JCR receives exclusive rights to commercialise givinostat for the treatment of Duchenne muscular dystrophy (DMD) in Japan and will be responsible for the local execution of clinical development activities, as well as regulatory submissions. Givinostat (marketed as Duvyzat® in the US, UK and EU) is an orally administered histone deacetylase inhibitor developed by Italfarmaco to treat DMD, regardless of the underlying dystrophin gene mutation. It has obtained regulatory authorisation across several major markets, including the US, EU and the UK; it is currently not approved in Japan. The agreement also establishes a broader strategic collaboration between the two companies to explore joint opportunities for the treatment of rare diseases. “This partnership is a key milestone in our global strategy to expand access to givinostat and deepen our impact in rare diseases,” said Antonio Nardi, Vice President and Head of Business & Portfolio Development of Italfarmaco. “JCR’s commitment to innovation, strong local expertise, and focus on patient-centred science make them an ideal partner for the expansion of our rare disease portfolios, not only in Japan, but also globally.” “Partnering with Italfarmaco is an important step for JCR as we enter the next phase of our growth and may be the first step in a long-lasting relationship between both companies that may extend into future research and development partnerships and cross-licensing opportunities,” said Shin Ashida, Chairman, President and CEO of JCR Pharmaceuticals. “We remain dedicated to developing therapies for the rare disease community, and givinostat extends this commitment to an even broader group of patients with rare diseases in Japan.” Francesco De Santis, Chairman of Italfarmaco Group, added: “By joining forces with JCR, we are expanding access to an important DMD therapy for the Japanese Duchenne community and also laying the groundwork for future innovations in rare diseases. Together, we are committed to delivering meaningful solutions where they are needed most.” About Duchenne Muscular Dystrophy (DMD) Duchenne muscular dystrophy (DMD) is a rare, progressive neuromuscular disorder caused by mutations in the dystrophin gene. These mutations prevent the production of functional dystrophin, causing the dystrophin-associated protein complex (DAPC) to break down. This makes muscle fibres more vulnerable to damage and increases histone deacetylase (HDAC) levels in the muscle cells, blocking the activation of important genes needed for muscle maintenance and repair. As a result, muscle fibres experience ongoing damage, leading to chronic inflammation and poor regeneration. Over time, muscle cells die and are replaced by scar tissue and fat.1-4 DMD primarily affects males, with symptoms typically appearing between the ages of two and five years. As the condition progresses, muscle weakness worsens, leading to difficulty walking and eventually loss of ambulation. Over time, the heart and respiratory muscles are also affected, which are the leading causes of premature death.5 DMD is one of the most severe and common forms of childhood muscular dystrophy, with a global birth incidence of approximately 1 in 5,050 boys.6 DMD affects an estimated 3,500 patients in Japan.7 About Givinostat Givinostat (Duvyzat®) was discovered through Italfarmaco’s research and development efforts in collaboration with Telethon and Duchenne Parent Project (Italy). Givinostat is the first nonsteroidal drug approved to treat patients with all genetic variants of DMD. It is a histone deacetylase (HDAC) inhibitor that works by targeting pathogenic processes to reduce inflammation and loss of muscle. Givinostat’s mechanism of action inhibits HDAC pathological overactivity in an effort to address the cascade of events leading to muscle damage, thereby counteracting the disease pathology and slowing down muscle degeneration. About Italfarmaco S.p.A. Founded in 1938 in Milan, Italy, Italfarmaco is a private global pharmaceutical company that has led the successful development and approval of many pharmaceutical products around the world. The Italfarmaco group has operations in more than 90 countries through directly controlled or affiliated companies. The company is a leader in pharmaceutical research, product development, production and commercialisation with proven success in many therapeutic areas including immuno-oncology, gynaecology, neurology, cardiovascular disease and rare diseases. Italfarmaco's rare disease unit includes programmes in Duchenne muscular dystrophy, Becker muscular dystrophy, amyotrophic lateral sclerosis and polycythaemia vera. About JCR Pharmaceuticals Co., Ltd. JCR Pharmaceuticals Co., Ltd. (TSE 4552) is a global specialty pharmaceutical company that develops treatments that go beyond rare diseases to solve the world’s most complex healthcare challenges. We continue to build upon our 50-year legacy in Japan while expanding our global footprint into the U.S., Europe, and Latin America. We improve patients’ lives by applying our scientific expertise and unique technologies to research, develop, and deliver next-generation therapies. Our approved products in Japan include therapies for the treatment of growth disorder, MPS II (Hunter syndrome), Fabry disease, acute graft-versus host disease, and renal anemia. Our investigational products in development worldwide are aimed at treating rare diseases including MPS I (Hurler, Hurler-Scheie and Scheie syndrome), MPS II, MPS IIIA and B (Sanfilippo syndrome type A and B), and more. Our core values – Putting people first, Forging our own path, Always advancing, and Committed to excellence – mean that the work we do benefits all our stakeholders, including partners, patients and employees. We strive to expand the possibilities for patients while accelerating medical advancement at a global level. For more information, please visit JCR’s global website: https://jcrpharm.com/. Cautionary Statement Regarding Forward-Looking Statements This document contains forward-looking statements that are subject to known and unknown risks and uncertainties, many of which are outside our control. Forward-looking statements often contain words such as “believe,” “estimate,” “anticipate,” “intend,” “plan,” “will,” “would,” “target” and similar references to future periods. All forward-looking statements regarding our plans, outlook, strategy and future business, financial performance and financial condition are based on judgments derived from the information available to us at this time. Factors or events that could cause our actual results to be materially different from those expressed in our forward-looking statements include, but are not limited to, a deterioration of economic conditions, a change in the legal or governmental system, a delay in launching a new product, impact on competitors’ pricing and product strategies, a decline in marketing capabilities relating to our products, manufacturing difficulties or delays, an infringement of our intellectual property rights, an adverse court decision in a significant lawsuit and regulatory actions. This document involves information on pharmaceutical products (including those under development). However, it is not intended for advertising or providing medical advice. Furthermore, it is intended to provide information on our company and businesses and not to solicit investment in securities we issue. Except as required by law, we assume no obligation to update these forward-looking statements publicly or to update the factors that could cause actual results to differ materially, even if new information becomes available in the future. References:

Drug ApprovalLicense out/in

07 Oct 2025

·www.globenewswire.com

New Data Presented on Givinostat for Treatment of Duchenne Muscular Dystrophy at 2025 World Muscle Society Congress

Long-term treatment with givinostat in boys with Duchenne muscular dystrophy continues to show a favourable safety and tolerability profile Consistent efficacy demonstrated across age groups MILAN, Italy, October 07, 2025 – Italfarmaco S.p.A. announced today that additional data on givinostat for the treatment of Duchenne muscular dystrophy (DMD) are being presented at the 30th annual International Congress of the World Muscle Society (WMS) held October 7-11 in Vienna, Austria. Poster presentations highlight the long-term safety of givinostat in patients with DMD, explore efficacy by age in a post hoc analysis of the pivotal EPIDYS study, and provide a detailed characterisation of gastrointestinal adverse events observed in this Phase 3 study. “These presentations provide new insights into the long-term safety, efficacy by age, and tolerability of givinostat in DMD,” said Scott Baver, PhD, VP, Head of Global Medical Affairs, Rare Diseases. “We are proud to share this data with global leaders advancing research, treatment, and patient care in DMD.” These data from studies sponsored by Italfarmaco presented at the 2025 WMS Congress include: #651P Long-Term Safety of Givinostat in Patients With Duchenne Muscular Dystrophy: Results From an Open-Label Extension StudyThe ongoing long-term study of givinostat in boys with DMD continues to provide insights into the safety and tolerability of the treatment, with results consistent with previous shorter-duration studies. Some patients have now been taking givinostat for more than 8 years in total, across several studies and extension phases. #684VP Efficacy of Givinostat by Age (6–7 and >7 Years): A Post Hoc Analysis of EPIDYSBaseline assessments in the study provided key insights into physical function in boys with DMD across different age groups. #688VP Characterizing Gastrointestinal Adverse Events of Interest From a Phase 3 Study of Givinostat in Patients With Duchenne Muscular DystrophyThe study continues to monitor gastrointestinal events in participants, with most reported events being mild to moderate in severity and resolving without requiring discontinuation of treatment. These findings provide important context for understanding the overall safety and tolerability of long-term therapy. Abstracts have been published in the Sept 2025 supplement of Neuromuscular Disorders here. About Givinostat Givinostat was discovered through Italfarmaco’s research and development efforts in collaboration with Telethon and Duchenne Parent Project (Italy). Givinostat is an orally administered histone deacetylase (HDAC) inhibitor that regulates the excessive HDAC activity characteristic of DMD muscles. By doing so, it helps restore the expression of key genes and biological processes essential for muscle maintenance and repair. Its mechanism of action is independent of the specific dystrophin gene mutation causing the disease.1,2 Givinostat is approved as a treatment of DMD in multiple regions, including the US, EU and UK. About ITALFARMACO Founded in 1938 in Milan, Italy, Italfarmaco is a private global pharmaceutical company that has led the successful development and approval of many pharmaceutical products around the world. The Italfarmaco group has operations in more than 90 countries through directly controlled or affiliated companies. The company is a leader in pharmaceutical research, product development, production and commercialisation with proven success in many therapeutic areas including immuno-oncology, gynaecology, neurology, cardiovascular disease and rare diseases. Italfarmaco's rare disease unit includes programmes in Duchenne muscular dystrophy, Becker muscular dystrophy, amyotrophic lateral sclerosis and polycythaemia vera. Media enquiries:Anja Heuer / Adolfo Luna | +49 (0) 151 106 199 05 | italfarmaco@trophic.euOther enquiries:Samantha Parker | Patient Advocacy and Communications Lead | RDEnquiries@italfarmacogroup.com References: Comi G, Bertini E, Vita G, et al. S22.008: Development of the histone deacetylases inhibitor Givinostat in Duchenne Muscular Dystrophy. Poster. Neurology. 2018;90(15 (Supplement)).Licandro SA, Crippa L, Pomarico R, et al. The pan HDAC inhibitor Givinostat improves muscle function and histological parameters in two Duchenne muscular dystrophy murine models expressing different haplotypes of the LTBP4 gene. Skelet Muscle. 2021;11(1):19. https://doi-org.libproxy1.nus.edu.sg/10.1186/s13395-021-00273-6. Attachment New Data Presented on Givinostat for Treatment of Duchenne Muscular Dystrophy at 2025 World Muscle Society Congress

Phase 3Drug ApprovalClinical Result

26 Sep 2025

·www.prnewswire.com

New Data Presented on Givinostat for Treatment of Duchenne Muscular Dystrophy at 2025 Neuromuscular Study Group Annual Scientific Meeting

Additional analyses from the givinostat clinical development program review observations on weight-based flexible dosing, characterization of thrombocytopenia, correlations between pharmacodynamic markers and functional outcomes, and respiratory function CONCORD, Mass., Sept. 26, 2025 /PRNewswire/ -- ITF Therapeutics LLC, the U.S. affiliate of Italfarmaco, today announced that results on givinostat for the treatment of Duchenne muscular dystrophy (DMD) are being presented at the 2025 Neuromuscular Study Group (NMSG) Annual Scientific Meeting held September 26-28 in Stresa, Italy. Poster presentations include analyses of functional outcomes based on weight-based flexible dosing in the pivotal Phase 3 EPIDYS trial, further characterization of thrombocytopenia, the link between vastus lateralis fat fraction (VLFF) measurement and efficacy outcomes, and observations on respiratory function. "These presentations reflect our focus on generating data that support clinicians in assessing the benefit-risk profile and full potential of givinostat, including the opportunity to consider flexible dosing strategies that may help to optimize tolerability and efficacy for some patients," said Scott Baver, Ph.D., VP, Head of Medical Affairs. "We are pleased that new insights on givinostat are being shared with global leaders in DMD research, treatment, and patient advocacy at the 2025 NMSG Annual Scientific Meeting." These data from studies sponsored by Italfarmaco presented at the 2025 NMSG Annual Scientific Meeting include: #1213 Givinostat Weight-based Flexible Dosing: Rationale and Efficacy at the Different Doses This poster includes an analysis of data on weight-based flexible dosing from the multicenter, randomized, double-blind, placebo-controlled, Phase 3 EPIDYS clinical trial. In the EPIDYS study, patients with DMD received weight-based givinostat dosing plus standard of care (i.e., corticosteroids) at two levels, dose A or B, depending on study protocol. Doses were adjusted (A to B or B to C) based on predefined rules related to tolerability to achieve the highest tolerated dose for each patient. This analysis assesses mean change from baseline in 4-stair climb (4SC) time and total North Star Ambulatory Assessment (NSAA) scores to evaluate efficacy based on the final administered dose at week 72. #1207 Characterizing Thrombocytopenia in Patients with Duchenne Muscular Dystrophy Treated with Givinostat: Results from the Phase 3 EPIDYS Trial Thrombocytopenia is a known adverse event of givinostat. This analysis further characterizes the risk and severity of thrombocytopenia or decreased platelet count based on an assessment of data from patients treated with givinostat in the Phase 3 EPIDYS study. #1211 Vastus Lateralis Fat Fraction is Associated with Functional Efficacy Endpoints in Patients with Duchenne Muscular Dystrophy Treated with Givinostat This poster reports findings from a post-hoc analysis assessing whether changes from baseline in VLFF correlate with measures of disease progression in patients with DMD. The analysis examines VLFF alongside efficacy endpoints observed at the end of the study (month 18), including 4SC, time to rise, 6-minute walking test, and NSAA. #1208 Givinostat Effect on Respiratory Function in Duchenne Muscular Dystrophy Before and After Ambulation Loss: Results from EPIDYS, LTSE, and PRO-DMD-01 Studies This poster, which includes findings previously presented at the 2025 MDA Clinical and Scientific Conference, reports observations on respiratory function in patients who experienced loss of ambulation during follow-up. The analysis indirectly compares forced vital capacity (FVC) %-predicted trajectories from patients treated with givinostat and corticosteroids in the Phase 3 EPIDYS study and its ongoing open-label extension with FVC %-predicted trajectories from patients in a natural history study of DMD disease progression who received corticosteroids only. For additional poster details, see abstract #1207, #1208, #1211, and #1213 published here. "The data presented at NMSG highlight important insights into the patient experience, ranging from the impact of flexible dosing to how thrombocytopenia was observed and characterized in the clinical trial setting," said Aravindhan Veerapandiyan, M.D., Associate Professor of Pediatrics, Division of Pediatric Neurology at the University of Arkansas for Medical Sciences and Arkansas Children's Hospital. "Together, these findings contribute to a deeper understanding of givinostat's use in the treatment of DMD and may help inform clinical decision-making." ITF Therapeutics is not a sponsor of the 2025 NMSG Annual Scientific Meeting. About DUVYZAT® (givinostat) DUVYZAT was discovered through Italfarmaco's research and development efforts in collaboration with Telethon and Duchenne Parent Project (Italy). DUVYZAT is an orally administered histone deacetylase (HDAC) inhibitor that regulates the excessive HDAC activity characteristic of DMD muscles. By doing so, it helps restore the expression of key genes and biological processes essential for muscle maintenance and repair. Its mechanism of action is independent of the specific dystrophin gene mutation causing the disease. For more information visit . About ITF Therapeutics LLC ITF Therapeutics was launched in January 2024 as the U.S. affiliate of Italfarmaco focused on the development and commercialization of products to treat rare diseases. Building on a legacy grounded in collaboration and innovation, ITF Therapeutics strives to partner with leaders from the patient advocacy and treatment communities to ensure that our programs reflect and support their unique needs and goals. The establishment of ITF Therapeutics reflects Italfarmaco's goal to build a world-class team of experts who share a passion to make a positive impact for rare disease communities. For more information visit . About Italfarmaco Founded in 1938 in Milan, Italy, Italfarmaco is a private global pharmaceutical company that has led the successful development and approval of many pharmaceutical products around the world. The Italfarmaco group has operations in more than 90 countries through directly controlled or affiliated companies. The company is a leader in pharmaceutical research, product development, production, and commercialization with proven success in many therapeutic areas including immuno-oncology, gynecology, neurology, cardiovascular disease, and rare diseases. Italfarmaco's rare disease unit includes programs in Duchenne muscular dystrophy, Becker muscular dystrophy, amyotrophic lateral sclerosis, and polycythemia vera. For more information visit . INDICATION DUVYZAT is a histone deacetylase inhibitor indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients 6 years of age and older. 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. To report SUSPECTED ADVERSE REACTIONS, contact ITF Therapeutics LLC at 1-833-582-4312 or FDA at 1-800-FDA-1088 or . Please see full Prescribing Information for additional safety information. DUVYZAT is a registered trademark of Italfarmaco S.p.A. U.S.A. Media inquiries: Kaitlin Gallagher | +1 212 253 8881 | [email protected] Other inquiries: Martha McKennitt | Communications Lead U.S.A. | [email protected] Global Media inquiries: Anja Heuer / Adolfo Luna | +49 (0)151 106 199 05 | [email protected] Other inquiries: Samantha Parker | Patient Advocacy and Communications Lead | [email protected] C-DUV-US-0583 09/2025 SOURCE ITF Therapeutics LLC WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Phase 3

100 Deals associated with Givinostat Hydrochloride

External Link

KEGG	Wiki	ATC	Drug Bank
-	Givinostat Hydrochloride	-	DB12645

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Muscular Dystrophy, Duchenne	United States	Italfarmaco SpA	21 Mar 2024

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Polycythemia Vera	Phase 3	United States	Italfarmaco SpA	26 Mar 2024
Polycythemia Vera	Phase 3	Austria	Italfarmaco SpA	26 Mar 2024
Polycythemia Vera	Phase 3	Bulgaria	Italfarmaco SpA	26 Mar 2024
Polycythemia Vera	Phase 3	Croatia	Italfarmaco SpA	26 Mar 2024
Polycythemia Vera	Phase 3	France	Italfarmaco SpA	26 Mar 2024
Polycythemia Vera	Phase 3	Germany	Italfarmaco SpA	26 Mar 2024
Polycythemia Vera	Phase 3	Hungary	Italfarmaco SpA	26 Mar 2024
Polycythemia Vera	Phase 3	Ireland	Italfarmaco SpA	26 Mar 2024
Polycythemia Vera	Phase 3	Israel	Italfarmaco SpA	26 Mar 2024
Polycythemia Vera	Phase 3	Italy	Italfarmaco SpA	26 Mar 2024

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT06093672 (ASH2025)	Phase 3	Polycythemia Vera	904	Givinostat	vsakfdglgp(ygvayquear) = vxbyivcegs fboiihtrpz (umrnsgrzbz )	Positive	06 Dec 2025
NCT06093672 (GIV-IN-PV, ASH2025)	Phase 3	Polycythemia Vera JAK2V617F mutation	220	Givinostat	uwdhihupmw(ygqbbpszqf) = zanzbzbdij mhzeqrtspv (jefswbsuxy ) View more	Positive	06 Dec 2025
				Hydroxyurea	zalyarxvga(hlwabhqfgt) = ziinufeezt hzivclamsx (zdqriithdv ) View more
NCT03373968 (EPIDYS, Pubmed \| Ann Clin Transl Neurol)	Phase 3	Muscular Dystrophy, Duchenne	194	Givinostat oral suspension + Givinostat oral suspension	bkvnblvcke(mnbwlyqlib) = ghfnjpcvns spfvsytcsx (bremfddnkx )	Positive	01 Nov 2025
NCT02851797 (EPIDYS, GlobeNewswire) Manual	Phase 3	Muscular Dystrophy, Duchenne	179	Givinostat	ikmrihwfgt(irxdtchfwc) = Post hoc analysis from the completed Phase 3 EPIDYS trial showed less decline in cardiac function throughout the study, compared to those who received placebo. jwewoioxgp (lyjyodjgcj ) View more	Positive	11 Jun 2025
NCT02851797 (EPIDYS, MDA2025)	Phase 3	Muscular Dystrophy, Duchenne	284	Givinostat	dxlutpmixp(vokvudjbcm) = mpidgpmwtz ztsvfcknoz (jzjqopfbvy ) View more	Positive	16 Mar 2025
				Steroids	dxlutpmixp(vokvudjbcm) = lalxvyurfd ztsvfcknoz (jzjqopfbvy ) View more
NCT02851797 (EPIDYS, MDA2025)	Phase 3	Muscular Dystrophy, Duchenne	56	Givinostat in addition to steroids	royarmvenl(qxkxmrfuxt) = kavbejgmsp kilqespplg (ulpesaymge, 2.2) View more	Positive	16 Mar 2025
				Steroids only (PRO-DMD-01)	royarmvenl(kxroaaqzji) = wixiyxnvtm jdymmrvvug (tokmkebfdk, 2.2) View more
NCT02851797 (EPIDYS, MDA2025)	Phase 3	Muscular Dystrophy, Duchenne	179	Givinostat (off-target group)	juizvuaahk(nwdxuikckr) = jrzdexahhc hskfhpmosa (qkypwhywjx, 7.9)	Positive	16 Mar 2025
				Placebo (off-target group)	juizvuaahk(nwdxuikckr) = vwemsqlptv hskfhpmosa (qkypwhywjx, 13.3)
NCT03373968 (MDA2025)	Phase 2/3	Muscular Dystrophy, Duchenne	194	Givinostat	wvlugpwlhi(qxefaxjnpl) = amwsrqkuwd ymiwttluth (swnzapwsqk ) View more	Positive	16 Mar 2025
				Givinostat (Prior Placebo)	wvlugpwlhi(qxefaxjnpl) = fntbcgekyv ymiwttluth (swnzapwsqk ) View more
NCT05860114 (CTgov)	Phase 1	Medication interactions	8	Givinostat (Givinostat)	ugpogvajqp(qrapydhxay) = ysetmyonlo ckhcalfnky (rxufwbsjfg, 25.8) View more	-	24 Jan 2025
				Givinostat (ITF2374)	ugpogvajqp(qrapydhxay) = kkacfrfnze ckhcalfnky (rxufwbsjfg, 45.7) View more
NCT03238235 (CTgov)	Phase 2	Becker Muscular Dystrophy	51	Givinostat (Givinostat)	vmxrzvwjid(lrtksgchii) = tlgjrfsnrg mvzcxvgrew (yxrxlumbhm, ekeurmcizj - tgbxrakvpr) View more	-	18 Nov 2024
				Placebo (Placebo)	vmxrzvwjid(lrtksgchii) = ggxqwmxtti mvzcxvgrew (yxrxlumbhm, baplpqibtq - ewsatompgv) View more

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