CLINICAL TRIALS PROFILE FOR GIVINOSTAT HYDROCHLORIDE

All Clinical Trials for givinostat hydrochloride

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00606307 ↗	Phase IIA Study of the HDAC Inhibitor ITF2357 in Patients With JAK-2 V617F Positive Chronic Myeloproliferative Diseases	Completed	Italfarmaco	Phase 2	2007-12-01	Primary Objective: To evaluate efficacy and safety of ITF2357 in the treatment of patients with JAK2V617F positive myeloproliferative diseases [Polycythemia Vera (PV), Essential Thrombocytosis (ET), Myelofibrosis (MF)]. Efficacy was evaluated by ad hoc haematological and clinical criteria for PV and ET, and by internationally established response criteria (EUMNET criteria) for MF. Safety was evaluated by number of subjects experiencing an Adverse Event (AE), type, frequency, severity, timing and relatedness of AEs, including changes in vital signs and clinical laboratory results. Secondary Objective: To evaluate the JAK2 mutated allele burden by quantitative Real-Time Polymerase Chain Reaction (qRTPCR).
NCT00928707 ↗	Phase II Study of GIVINOSTAT (ITF2357) in Combination With Hydroxyurea in Polycythemia Vera	Completed	Italfarmaco	Phase 2	2009-06-01	The primary objective of the study was to evaluate the efficacy of Givinostat in combination with hydroxyurea in patients with JAK2V617F-positive Polycythemia Vera (PV) non-responders to the maximum tolerated dose of hydroxyurea monotherapy. The secondary objectives of this study were: - To evaluate the safety and tolerability of Givinostat in combination with hydroxyurea in patients with JAK2V617Fpositive PV non-responders to the maximum tolerated dose of hydroxyurea monotherapy; - To explore the impact in terms of efficacy and tolerability of Givinostat 50 mg dose escalation in patients not achieving at least a partial response at the time when the primary endpoint was assessed (week 12); - To evaluate the molecular response (JAK2 mutated allele burden) by quantitative Real Time-Polymerase Chain Reaction (RT-PCR); - To evaluate the reduction of the fraction of JAK2V617F positive clonogenic progenitors.
NCT01261624 ↗	Efficacy and Safety Dose Finding Study of Givinostat to Treat Polyarticular Course Juvenile Idiopathic Arthritis	Terminated	Italfarmaco	Phase 2	2010-10-01	The present study has been designed in order to evaluate the efficacy and safety of two doses of Givinostat in subjects with polyarticular course JIA Givinostat ready-to-use suspension especially intended for paediatric administration, will be administered orally at different daily doses. Patients with an established diagnosis of one of the following JIA forms (Polyarticular JIA rheumatoid factor positive or negative, Oligoarticular extended JIA, Systemic JIA without active systemic features) will be enrolled. The treatment regimen will remain unchanged for 12 weeks and the clinical response will by assessed by applying the ACR Pediatric response criteria. Patients achieving at least an ACR Pediatric 30 response will continue receiving the assigned dose for 12 further weeks. After the end of study (week 24) responder patients will be allowed to extend the treatment until they maintain a clinical benefit.
NCT01557452 ↗	Open-Label Extension of the Dose Finding Study (DSC/08/2357/36) in Patients With Poly Juvenile Idiopathic Arthritis	Terminated	Parexel	N/A	2011-12-28	Primary Objective of the study: the purpose of this extension study was to determine the safety of Givinostat in a long term treatment of patients who participated in DSC/08/2357/36 study with good results (clinical benefit at least pediACR30 response);
NCT01557452 ↗	Open-Label Extension of the Dose Finding Study (DSC/08/2357/36) in Patients With Poly Juvenile Idiopathic Arthritis	Terminated	Italfarmaco	N/A	2011-12-28	Primary Objective of the study: the purpose of this extension study was to determine the safety of Givinostat in a long term treatment of patients who participated in DSC/08/2357/36 study with good results (clinical benefit at least pediACR30 response);

Clinical Trial Conditions for givinostat hydrochloride

Condition Name

Condition Name for givinostat hydrochloride
Intervention	Trials
Duchenne Muscular Dystrophy	4
Polycythemia Vera	3
Drug Drug Interaction	2
Chronic Myeloproliferative Neoplasms	1

Condition MeSH

Condition MeSH for givinostat hydrochloride
Intervention	Trials
Muscular Dystrophy, Duchenne	7
Muscular Dystrophies	5
Polycythemia Vera	3
Polycythemia	3

Clinical Trial Locations for givinostat hydrochloride

Trials by Country

Trials by Country for givinostat hydrochloride
Location	Trials
Italy	43
United States	25
Canada	9
United Kingdom	8
Spain	5

Trials by US State

Trials by US State for givinostat hydrochloride
Location	Trials
California	3
Virginia	2
Pennsylvania	2
Oregon	2
Missouri	2

Clinical Trial Progress for givinostat hydrochloride

Clinical Trial Phase

Clinical Trial Phase for givinostat hydrochloride
Clinical Trial Phase	Trials
PHASE3	1
PHASE2	1
Phase 3	2

Clinical Trial Status

Clinical Trial Status for givinostat hydrochloride
Clinical Trial Phase	Trials
Completed	8
Recruiting	3
Active, not recruiting	3

Clinical Trial Sponsors for givinostat hydrochloride

Sponsor Name

Sponsor Name for givinostat hydrochloride
Sponsor	Trials
Italfarmaco	17
Fortrea	2
Parexel	1

Sponsor Type

Sponsor Type for givinostat hydrochloride
Sponsor	Trials
Industry	21
Other	2

Clinical Trials Update, Market Analysis, and Projection for Givinostat Hydrochloride

Last updated: October 26, 2025

Introduction

Givinostat hydrochloride is an oral histone deacetylase (HDAC) inhibitor primarily under investigation for its potential in treating rare hematologic and oncologic diseases. Its mechanism involves modulating gene expression, leading to anti-proliferative and pro-apoptotic effects in malignant cells. As the pharmaceutical landscape evolves, understanding the trajectory of Givinostat hydrochloride—encompassing clinical trial developments, market dynamics, and future projections—is vital for stakeholders. This analysis offers a comprehensive review of current data and strategic outlooks tailored for industry professionals.

Clinical Trials Update

Current Status and Ongoing Studies

Givinostat hydrochloride has progressed through pivotal clinical stages, with notable research focusing on myeloproliferative neoplasms (MPNs), histiocytic disorders, and rare inherited diseases.

• Myelofibrosis and Polycythemia Vera (PV): The drug has undergone Phase II trials assessing its efficacy as a monotherapy or in combination with JAK inhibitors like ruxolitinib. Preliminary results demonstrate manageable safety profiles and promising reductions in spleen size and symptom improvement [1].

• Histiocytic Disorders: Trials in Erdheim-Chester disease and Langerhans cell histiocytosis (LCH) have entered Phase II stages, highlighting Givinostat's potential to modulate inflammatory pathways.

• Other Hematologic Malignancies: Investigations extend to multiple myeloma, leukemia, and essential thrombocythemia, emphasizing its versatile mechanism but remaining in early exploratory phases.

Recent Developments

As of 2023, the most recent clinical trials database entries indicate ongoing Phase II studies, with some extensions and protocol amendments to optimize dosing and combination strategies [2]. While no late-stage (Phase III) data has been published publicly, companies and academic consortia remain committed to exploring Givinostat's full therapeutic potential, especially in rare diseases.

Regulatory Considerations and Approvals

Givinostat has obtained orphan drug designation for certain indications, such as myelofibrosis and histiocytic disorders, in regions including the U.S. and Europe. This status can expedite review processes and incentivize further research, though large-scale approval remains pending [3].

Market Analysis

Market Landscape and Competitive Position

The global HDAC inhibitors market is projected to expand at a CAGR of approximately 12% over the next five years, driven by advancements in epigenetic therapies and unmet medical needs in rare diseases [4].

Givinostat's primary competitors include:

• Vorinostat (Zolinza) and romidepsin (Istodax), approved for certain lymphomas.

• Panobinostat (Farydak), approved for multiple myeloma.

However, Givinostat occupies a unique niche due to its potential efficacy in rare hematological disorders and favorable safety profile, offering a differentiated value proposition.

Market Size and Revenue Potential

• Rare Disease Segment: The orphan drug market for rare hematological conditions is expected to reach $40 billion by 2027 globally [5], with significant contributions from targeted therapies like Givinostat.

• Pricing and Reimbursement: Given its orphan status, Givinostat could command premium pricing, estimated between $100,000 to $200,000 per patient annually, contingent on indication, efficacy, and regulatory status.

• Adoption Factors: Pivotal factors influencing market penetration include clinical efficacy, safety, regulatory approvals, and clinician acceptance, particularly in niche markets that lack effective alternatives.

Market Entry Challenges

Key hurdles encompass:

• The necessity for robust Phase III data to secure regulatory approvals.
• Competition from existing HDAC inhibitors and emerging epigenetic therapies.
• Limited patient populations due to the rarity of target diseases, constraining revenue scalability.

Market Projection and Future Outlook

Short-term Outlook (Next 2 Years)

• Expectation of data readouts from ongoing Phase II trials.
• Potential regulatory designations, including orphan status or breakthrough therapy, bolstering development prospects.
• Initiation of combination studies with JAK inhibitors or other targeted agents.

Mid-to-long-term Outlook (3-5 Years)

• Potential for FDA and EMA approval if clinical trial endpoints demonstrate significant clinical benefit.
• Market penetration in rare hematological disorders, with initial revenues likely in the $50-100 million range annually, assuming successful approval.
• Growth driven by increased understanding of epigenetic mechanisms and expanding indications.

Risks and Opportunities

• Risks: Delays in clinical validation, unmet endpoints, or unfavorable safety profiles could impede commercialization.
• Opportunities: Novel combination regimens, expanding indications, and strategic partnerships could accelerate adoption.

Key Takeaways

• Givinostat hydrochloride has shown promising activity in early-stage clinical trials for rare hematological and histiocytic disorders, with ongoing studies aimed at substantively extending its therapeutic landscape.
• The drug's positioning as an orphan drug provides both strategic advantages and market exclusivity benefits, although substantial clinical data are needed to achieve regulatory approval.
• The rapidly expanding epigenetic therapy market presents significant opportunities; however, success hinges on demonstration of clear clinical benefit and effective stakeholder engagement.
• Stakeholders should monitor trial developments closely, evaluate partnership prospects, and prepare for commercialization strategies aligned with regulatory milestones.
• Emphasizing its unique mechanism and targeted niche can enable Givinostat to carve a distinctive market space in rare disease therapeutics.

FAQs

1. What are the main indications currently being evaluated for Givinostat hydrochloride?
   Givinostat is in clinical trials for myeloproliferative neoplasms (including myelofibrosis and PV), histiocytic disorders (like Erdheim-Chester disease and LCH), and exploratory studies in other hematologic malignancies.

2. Has Givinostat received regulatory approval for any indication?
   As of 2023, Givinostat has not gained formal regulatory approval but holds orphan drug status in the U.S. and Europe for specific rare diseases, which may facilitate future approvals.

3. What are the competitive advantages of Givinostat over other HDAC inhibitors?
   Its oral administration, tolerable safety profile, potential efficacy in rare hematological disorders, and orphan drug status distinguish it from some existing HDAC inhibitors primarily approved for cancers like lymphomas.

4. What are the major challenges facing Givinostat’s commercialization?
   Key obstacles include the need for conclusive Phase III trial data, regulatory hurdles, limited patient populations due to rarity of targeted diseases, and competition from other epigenetic therapies.

5. What is the market potential for Givinostat in the next five years?
   If approved, Givinostat could generate revenues in the range of $50-100 million annually within niche markets, with growth driven by expanded indications and increased clinical validation.

References

[1] ClinicalTrials.gov. "Givinostat in Treating Patients with Myelofibrosis or Polycythemia Vera." Accessed 2023.
[2] EU Clinical Trials Register. "Givinostat studies: update 2023."
[3] OrphanDrugDesignations.com. "Givinostat entries."
[4] MarketsandMarkets. "Epigenetic Therapy Market by Type, Application, and Region." 2022.
[5] GlobalData. "Orphan Drugs Market Analysis and Forecast." 2022.