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

Structure/Sequence

Molecular FormulaC24H30ClN3O5

InChIKeyFKGKZBBDJSKCIS-UHFFFAOYSA-N

CAS Registry732302-99-7

Clinical Trials associated with Givinostat Hydrochloride

NCT06736223

/ CompletedPhase 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

242

Literatures (Medical) associated with Givinostat Hydrochloride

01 Jun 2026·CURRENT THERAPEUTIC RESEARCH-CLINICAL AND EXPERIMENTAL

Understanding Pharmacokinetic-Drug Interactions With Drugs Approved by the US Food and Drug Administration in 2024 to Better Manage the Risk of Drug Interactions With Concomitant Medications: A Review of Clinical Data From New Drug Applications

Review

Author: Owens, Katie ; Argon, Sophie ; Yu, Jingjing ; Ragueneau-Majlessi, Isabelle ; Wang, Yan

Objective:

This analysis aimed to provide a mechanistic understanding and an evaluation of the clinical relevance of pharmacokinetic drug-drug interactions (DDIs) associated with drugs approved by the Food and Drug Administration in 2024.

Methods:

Drug metabolism, transport, and absorption-based DDI data available in New Drug Applications (NDAs) of small molecular drugs approved (n = 34) was analyzed. The mechanism and clinical outcome of these interactions were characterized based on in vitro, in silico, and clinical data.

Results:

As objects, 11 drugs were identified as clinical substrates. Of these, 7 drugs were substrates of CYP3A, 3 of CYP2C9, one of CYP1A2, and one of CYP2C8, including the sensitive substrates vanzacaftor (CYP3A) and vorasidenib (CYP1A2). As precipitants, 6 drugs (acoramidis, cefepime/enmetazobactam, givinostat, lazertinib, mavorixafor, and resmetirom) were clinical inhibitors of CYP enzymes (2C8, 2C9, 2D6, 2E1, and 3A), with mavorixafor being a CYP2D6 strong inhibitor. Two drugs (elafibranor and tovorafenib) showed weak induction of CYP3A. Regarding transporter data, 3 drugs were substrates of transporters, including seladelpar (BCRP and OAT3), sulopenem (OAT3), and vadadustat (OAT1/3), and 8 drugs (arimoclomol, danicopan, givinostat, lazertinib, mavorixafor, resmetirom, vadadustat, and vazacaftor/tezacaftor/deutivacaftor) were inhibitors of transporters. All clinical DDIs with AUC changes ≥ 2-fold triggered labeling recommendations. Several DDIs with an AUC change <2 also had labeling recommendations, pertaining most often to the concomitant use of drugs with a narrow therapeutic index.

Conclusions:

Mechanistic DDI insights from newly approved therapies can be extrapolated to inform the management of commonly co-administered drugs, supporting a safer and more effective use of new drug products in the context of polypharmacy.

01 Dec 2025·ARCHIVES DE PEDIATRIE

Corticosteroid treatment in Duchenne muscular dystrophy

Review

Author: Fontaine-Carbonnel, Stéphanie

Duchenne muscular dystrophy (DMD) is a severe degenerative disease that remains incurable. Its prognosis has been transformed by multidisciplinary care, which has significantly improved the life expectancy of patients. Corticosteroids have been used for over 20 years and have modified the care of young boys with DMD: They delay the loss of ambulation, support the gradual introduction of technical mobility aids, and reduce the frequency of scoliosis surgery. Corticoids are started and maintained at the theoretical dose of 0.75 mg/kg/day of prednisone/prednisolone or 0.9 mg/kg/day of deflazacort, adjusting the dose to weight if the benefit-risk ratio favors treatment. If introduced early enough, when muscle degeneration is not too advanced, they prolong the ability to climb stairs, rise from the floor, walk, and bring the hands to the mouth. They have been suggested to contribute to delaying the onset of respiratory failure and cardiomyopathy. The well-known side effects of corticosteroids require specific management (to prevent obesity and osteoporosis) and/or adjustment of their dosage. The only reasons to discontinue treatment when the patient and family are adhering to it are the uncontrolled side effects. Thus, in many countries including France, as of late 2025, the only routinely prescribed symptomatic treatment for DMD remains conventional corticosteroids, which will be discussed in this article, followed by a description of vamorolone, a dissociative steroidal compound, and givinostat, a histone deacetylase inhibitor.

28 Nov 2025·BIOLOGICAL & PHARMACEUTICAL BULLETIN

Comparative Study of Histone Deacetylase Inhibitors for Radiation Protection Using Survival Outcomes in a Mouse Model

Article

Author: Watanabe, Shinichi ; Tanaka, Mamoru ; Yano, Takaaki ; Yamaguchi, Takumi ; Hidaka, Noriaki ; Kurokawa, Yukiro

Histone deacetylases (HDACs) regulate chromatin structure and gene expression, and their inhibition has been proposed as a radioprotective strategy. However, few studies have compared multiple HDAC inhibitors (HDACis) under identical conditions. This study evaluated the efficacy of seven HDACis in a mouse total body irradiation (TBI) model. Male ICR mice received 7.5 Gy TBI followed by a single administration of valproic acid (VPA; 300 or 600 mg/kg), sodium butyrate (NaB; 500 or 1000 mg/kg), trichostatin A (TSA; 0.5 or 1.0 mg/kg), vorinostat (10 or 50 mg/kg), panobinostat (25 or 50 mg/kg), givinostat (5 or 10 mg/kg), or entinostat (25 or 50 mg/kg). Survival was monitored for 20 d. Only VPA at 600 mg/kg significantly improved survival compared with vehicle (overall p = 0.00241; p = 0.0039 vs. vehicle), while all other HDACis showed no significant benefit. VPA's efficacy may reflect a combination of effects on DNA repair, inflammation, and redox regulation rather than HDAC inhibition alone. These findings suggest VPA to be a promising candidate for radioprotection and emphasize the need for further studies to optimize dosing and explore underlying mechanisms.

News (Medical) associated with Givinostat Hydrochloride

23 Feb 2026

·www.prnewswire.com

ITF Therapeutics LLC Announces Multiple Data Presentations at 2026 MDA Clinical and Scientific Conference

Presenting ten abstracts including observations on long-term safety, disease progression, and decline in muscle contractile area with givinostat Encore oral presentation also highlights analyses from the givinostat clinical development program on weight-based flexible dosing CONCORD, Mass., Feb. 23, 2026 /PRNewswire/ -- ITF Therapeutics LLC, the U.S. rare disease affiliate of Italfarmaco, today announced that one oral and nine poster presentations have been accepted at the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference to be held March 8-11, 2026, in Orlando, Florida. Poster presentations will include long-term safety observations, as well as new findings on disease progression and decline in muscle contractile area in patients with Duchenne muscular dystrophy (DMD) treated with givinostat. In addition, an encore oral presentation delivered by Aravindhan Veerapandiyan, M.D., Associate Professor of Pediatrics, University of Arkansas for Medical Sciences and Arkansas Children's Hospital, includes analyses of functional outcomes with weight-based flexible dosing in the pivotal Phase 3 EPIDYS trial. Functional outcomes were evaluated based on time to complete the 4-stair climb test and North Star Ambulatory Assessment scores. Encore poster presentations will also cover findings related to cardiac safety data in ambulant patients from the EPIDYS study, additional insights on the simplified givinostat dosing regimen, and the characterization of thrombocytopenia and gastrointestinal adverse events. Please see the Indication and Important Safety Information for DUVYZAT® (givinostat) below. "Since the launch of ITF Therapeutics just over two years ago, our progress has been shaped by the voices and experiences of people living with Duchenne and their families whom we are privileged to serve," said Matt Trudeau, President, ITF Therapeutics. "The MDA Clinical and Scientific Conference offers an essential opportunity for us to hear directly from community members to better understand their evolving needs and priorities. At this year's meeting, we look forward to presenting new data — including a range of perspectives on the safety and efficacy of DUVYZAT — that reflect our ongoing dialogue with the Duchenne community." Encore Oral Presentation Title: Givinostat Weight-Based Flexible Dosing: Rationale and Efficacy at the Different Doses Presenter: Aravindhan Veerapandiyan, M.D., Associate Professor of Pediatrics, University of Arkansas for Medical Sciences and Arkansas Children's Hospital Date and time: Wednesday, March 11, 2026, at 1:30 p.m. ET De Novo P oster Presentations Title: Long-Term Safety of Givinostat in Patients with Duchenne Muscular Dystrophy: Interim Results from an Open-Label Extension Study Presenter: John F. Brandsema, M.D., Pediatric Neurologist, Division of Neurology, Children's Hospital of Philadelphia Poster number: 51S Title: Givinostat Reduces the Decline of Contractile Cross-Sectional Area and Decreases Fat Infiltration in Patients with Duchenne Muscular Dystrophy Presenter: Sara Cazzaniga, R&D Senior Manager, Neuromuscular Area, Italfarmaco Poster number: 37S Title: Open Label Extension Analysis Shows Potential Delay in Age at Loss of Ambulation in Patients with Duchenne Muscular Dystrophy Treated with Givinostat Presenter: Paolo Bettica, M.D., Ph.D., Chief Medical Officer, Italfarmaco Poster number: 64S Title: Givinostat Reprograms TGFβ-Induced Fibrotic Network in DMD Stromal Cells Presenter: Monica Forino, M.D., Molecular Biologist, Italfarmaco Poster number: 276T Encore Poster Presentations Title: A Simplified Givinostat Dosing Regimen Minimizes Exposure Differences and Dose Reductions in Higher-Weight Patients Presenter: Paolo Bettica, M.D., Ph.D., Chief Medical Officer, Italfarmaco Poster number: 36S Title: Cardiac Safety Data for Givinostat in Ambulant Patients with Duchenne Muscular Dystrophy: Results from the EPIDYS Study Presenter: Han C. Phan, M.D., Principal Investigator and Head of Research, Rare Disease Research Poster number: 42S Title: Characterizing Thrombocytopenia in Patients with Duchenne Muscular Dystrophy Treated with Givinostat: Results from the Phase 3 EPIDYS Trial Presenter: John F. Brandsema, M.D., Pediatric Neurologist, Division of Neurology, Children's Hospital of Philadelphia Poster number: 50S Title: Characterizing Platelet Count Reductions Below 75 × 109/L in Patients with Duchenne Muscular Dystrophy Treated with Givinostat from the Phase 3 EPIDYS Trial and LTSE Study Presenter: Paolo Bettica, M.D., Ph.D., Chief Medical Officer, Italfarmaco Poster number: 67S Title: Characterizing Gastrointestinal Adverse Events of Interest from a Phase 3 Study of Givinostat in Patients with Duchenne Muscular Dystrophy Presenter: John F. Brandsema, M.D., Pediatric Neurologist, Division of Neurology, Children's Hospital of Philadelphia Poster number: 49S ITF Therapeutics is also sponsoring the following educational program taking place during the conference: Industry Forum: Clinical Perspectives on HDAC Inhibition in Duchenne Muscular Dystrophy Presenters: Omer A. Abdul Hamid, M.D., Neuromuscular Specialist, Pediatric Neurology, Nemours Children's Hospital, joined by a caregiver from the DMD community Date and time: Tuesday, March 10, 2026, at 12:00-1:30 p.m. ET Room: Florida 5-7 During the forum, Dr. Hamid will share perspectives on unmet needs in the DMD treatment landscape, the potential role of histone deacetylase (HDAC) inhibition in the treatment of DMD, and expanded findings from the Phase 3 clinical trial evaluating the safety and efficacy of givinostat including the latest open-label extension data. A caregiver will also share their personal experiences with DUVYZAT. This is not an official event of the 2026 MDA Clinical and Scientific Conference. This event is not sponsored, endorsed, or accredited by MDA. About DUVYZAT® (givinostat) DUVYZAT is a U.S. FDA-approved histone deacetylase (HDAC) inhibitor indicated for the treatment of patients six years of age and older with Duchenne muscular dystrophy (DMD). The therapy was discovered through the research and development efforts of Italfarmaco in collaboration with Telethon and Duchenne Parent Project (Italy). HDACs are enzymes located in the body's cells that play a key role in maintaining and repairing muscles. In DMD, the HDAC enzymes become overactive, leading to chronic muscle inflammation, decreased muscle repair, and replacement of muscle with fat and scar tissue. DUVYZAT inhibits HDAC overactivity and is thought to help reduce inflammation, increase the body's ability to repair muscles, and slow muscle loss. For more information visit . About ITF Therapeutics LLC ITF Therapeutics was established in January 2024 as the United States affiliate of Italfarmaco Group to develop and commercialize treatments for rare diseases. The company combines end-to-end execution with close listening to learn directly from advocates, researchers, clinicians, and families, ensuring their perspectives help shape the design of innovative and potentially life-changing solutions. With deep expertise across regulatory, commercial operations, access, and community partnerships, ITF Therapeutics is focused on delivering proven therapies with urgency, ambition, and compassion to each rare disease community it serves. For more information visit . 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 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 and Important Safety Information for DUVYZAT® (givinostat) 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: Emily Steinhauer | [email protected] Other inquiries: Martha McKennitt | Communications Lead U.S.A.| [email protected] Global Media inquiries: [email protected] | +49 (0) 151 106 199 05 Other inquiries: Samantha Parker | Patient Advocacy and Communications Lead| [email protected] C-ITF-US-0022 02/2026 SOURCE ITF Therapeutics LLC 21% more press release views with Request a Demo

Phase 3

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/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

100 Deals associated with Givinostat Hydrochloride

External Link

KEGG	Wiki	ATC	Drug Bank
-	Givinostat Hydrochloride	-	DB12645

R&D Status

Approved

10 top approved records.

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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

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT06093672 (ASH2025)	Phase 3	Polycythemia Vera	904	Givinostat	jvgivconre(pfznufksur) = yiwcepukrt hpvesicnvk (zrsmcfnlej )	Positive	06 Dec 2025
NCT06093672 (GIV-IN-PV, ASH2025)	Phase 3	Polycythemia Vera JAK2V617F mutation	220	Givinostat	gcvvucbqyf(yibnkpwoxi) = csstgmcths jssdzawugo (acxphfdedo ) View more	Positive	06 Dec 2025
				Hydroxyurea	bbmvyuyhun(eqdppepovb) = ixpedktcoe hmsfftulbf (aeueqgwjle ) View more
NCT03373968 (EPIDYS, Pubmed \| Ann Clin Transl Neurol)	Phase 3	Muscular Dystrophy, Duchenne	194	Givinostat oral suspension + Givinostat oral suspension	lwxjuykjbg(rxvdvuehcu) = bqjmkzchqc bocixuugyh (llggaxudhj )	Positive	01 Nov 2025
NCT02851797 (EPIDYS, GlobeNewswire) Manual	Phase 3	Muscular Dystrophy, Duchenne	179	Givinostat	krpqmryvtg(bhfetyiuwz) = 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. yxpwbwcalx (fzgkdclqyo ) View more	Positive	11 Jun 2025
NCT02851797 (EPIDYS, MDA2025)	Phase 3	Muscular Dystrophy, Duchenne	179	Givinostat (off-target group)	hvppjbvhab(qjieocpjfi) = vmqqhhakan beonsjoqhi (emnmlokryf, 7.9)	Positive	16 Mar 2025
				Placebo (off-target group)	hvppjbvhab(qjieocpjfi) = timswdieij beonsjoqhi (emnmlokryf, 13.3)
NCT03373968 (MDA2025)	Phase 2/3	Muscular Dystrophy, Duchenne	194	Givinostat	bzjyesquqw(wbxljtuhyc) = pktyuqeswp wpzlfogjcl (iqyzkmrgjr ) View more	Positive	16 Mar 2025
				Givinostat (Prior Placebo)	bzjyesquqw(wbxljtuhyc) = zssiajqeio wpzlfogjcl (iqyzkmrgjr ) View more
NCT02851797 (EPIDYS, MDA2025)	Phase 3	Muscular Dystrophy, Duchenne	56	Givinostat in addition to steroids	ajfpgfuwvl(djznjsilcv) = frglzyvjxc heqfghyjug (nnhabdqcqy, 2.2) View more	Positive	16 Mar 2025
				Steroids only (PRO-DMD-01)	ajfpgfuwvl(kccdeuwabg) = yttxbvezwa adjuuzriea (ecpsvxxdho, 2.2) View more
NCT02851797 (EPIDYS, MDA2025)	Phase 3	Muscular Dystrophy, Duchenne	284	Givinostat	cctuophrbb(vkqzhpmonh) = ixpldtqqay oqxncqgldr (fbqkxhkldb ) View more	Positive	16 Mar 2025
				Steroids	cctuophrbb(vkqzhpmonh) = ywkxfrxtrg oqxncqgldr (fbqkxhkldb ) View more
NCT05860114 (CTgov)	Phase 1	Medication interactions	8	Givinostat (Givinostat)	mkdywzfiuo(wsuyvivjty) = barkdhoqii bxtlqvuwyq (bfyzbibcmn, 25.8) View more	-	24 Jan 2025
				Givinostat (ITF2374)	mkdywzfiuo(wsuyvivjty) = yjqgrqweaa bxtlqvuwyq (bfyzbibcmn, 45.7) View more
NCT03238235 (CTgov)	Phase 2	Becker Muscular Dystrophy	51	Givinostat (Givinostat)	jopuylkecr(pnwhikcilj) = jkleuhzisx smlslxschg (kwdgzsfqqm, alqkoqnfbs - oiipmdxypz) View more	-	18 Nov 2024
				Placebo (Placebo)	jopuylkecr(pnwhikcilj) = arnhhnqeko smlslxschg (kwdgzsfqqm, essatyxmje - uvsjbihwip) View more

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