Duvyzat (givinostat) / Italfarmaco, Medis, Multicare Pharmaceuticals - LARVOL DELTA

Adrenal insufficiency in individuals with Duchenne muscular dystrophy treated with glucocorticoids: Insights from the past, current challenges, and future directions. (PubMed, J Neuromuscul Dis) - "Despite a rapidly evolving therapeutic landscape that now includes multiple disease modifying treatments including exon skipping medications, microdystrophin gene therapy, and givinostat, high dose glucocorticoids (GCs) initiated at a young age remain central to the standard of care for DMD...The approach described in this document also applies to individuals with related dystrophinopathies including Becker muscular dystrophy and female manifesting carriers treated with GCs, recognizing that GCs are not standard of care for these conditions and are prescribed on an individualized basis at some centers to severely affected individuals. Key themes discussed include best practices for individual and caregiver education about adrenal suppression, creation and implementation of stress steroid plans, safe transition between GC treatment regimens, and discontinuation of GC therapy."

Journal • Review • Becker Muscular Dystrophy • Duchenne Muscular Dystrophy • Endocrine Disorders • Gene Therapies • Genetic Disorders • Muscular Dystrophy • Nephrology • Renal Disease • Respiratory Diseases

Givinostat attenuates sepsis-induced myocardial dysfunction by suppressing macrophage inflammation and restoring phagocytic activity. (PubMed, Int Immunopharmacol) - "Crucially, the cardioprotective effects of Givinostat were abolished by recombinant CCL7 administration, underscoring a CCL7-dependent mechanism. These findings reveal that Givinostat alleviates SIMD by attenuating macrophage inflammation through suppression of the CCL7/CCR1 signaling pathway and restoration of phagocytic function, highlighting its therapeutic potential for sepsis and SIMD."

Journal • Duchenne Muscular Dystrophy • Genetic Disorders • Infectious Disease • Inflammation • Muscular Dystrophy • Septic Shock • CCL7

Givinostat enhances antisense oligonucleotide efficacy in the mdx52 model of Duchenne muscular dystrophy. (PubMed, Mol Ther Nucleic Acids) - "Notably, treated mice showed improved muscle function, as evidenced by a significant reduction in force loss after eccentric contractions, a clinically meaningful outcome. Although the precise molecular mechanisms underlying these effects remain to be fully elucidated, these findings support the therapeutic potential of combining givinostat with ASOs to enhance dystrophin restoration and muscle preservation in DMD."

Journal • CNS Disorders • Duchenne Muscular Dystrophy • Fibrosis • Genetic Disorders • Immunology • Muscular Dystrophy

From design to clinic: Medicinal chemistry and pharmacology of approved epigenetic drugs. (PubMed, Eur J Med Chem) - "This review explores the ten epigenetic drugs that have attained worldwide regulatory approval for human therapy: the DNA methyltransferase inhibitors azacitidine (2004) and decitabine (2006), the histone deacetylase inhibitors vorinostat (2006), romidepsin (2009), belinostat (2014), panobinostat (2015) tucidinostat (2015) and givinostat (2024), and the histone methyltransferase inhibitors tazemetostat (2020) and valemetostat (2022). The history and strategy of their discovery and development, their biological targets and mechanisms of action and their therapeutic use. In addition, current advancements and efforts, as well as future perspectives in the design and clinical approval of new epigenetic drugs are discussed."

Journal • Review • Oncology

Real-world safety profile of givinostat: an early post-marketing pharmacovigilance study based on the FAERS database. (PubMed, Front Pharmacol) - "As hypothesis-generating signals derived from spontaneous reports, these initial findings require future prospective validation. They expand the current understanding of givinostat's safety profile and highlight the necessity for continuous pharmacovigilance."

Adverse events • Journal • P4 data • Real-world evidence • Duchenne Muscular Dystrophy • Dyslipidemia • Genetic Disorders • Hematological Disorders • Hypertriglyceridemia • Muscular Dystrophy • Musculoskeletal Diseases • Orthopedics • Psychiatry

JCR Pharmaceuticals Sets 2026–2027 Timeline to Launch Givinostat for Duchenne Muscular Dystrophy in Japan (TipRanks) - "The company intends to file for marketing authorization in 2026 using existing overseas clinical data, seek approval, and launch the drug in 2027. "

Launch Japan • Duchenne Muscular Dystrophy

Italfarmaco S.p.A. today announced the presentation of new data from its clinical development programme for givinostat in patients with Duchenne muscular dystrophy (DMD) at the 19th International Congress on Neuromuscular Diseases (ICNMD) 2026 (GlobeNewswire) - "Quantitative MRI measures of DMD disease progression from the Phase 3 EPIDYS trial were associated with statistically significant differences between treatment group vs. placebo in measures of muscle composition, including contractile cross-sectional area (cCSA) and fat fraction, across selected lower limb muscles"

P3 data • Duchenne Muscular Dystrophy

ISS07: Givinostat: Bridging Clinical Evidence to Real-World Practice in Duchenne Muscular Dystrophy (ICNMD 2026) - "It will cover the scientific foundation of histone deacetylase (HDAC) inhibition, Phase 3 EPIDYS efficacy and safety data, long-term functional outcomes from the open-label extension (OLE) study, and practical management insights from the Italian Early Access Program (EAP) and emerging US clinical experience. Review the mechanism of action of HDAC inhibition and its role in targeting DMD pathology Analyze safety and efficacy results from the Phase 3 EPIDYS trial and long-term OLE data Discuss real-world safety and dose management strategies from the Italian EAP cohort Evaluate practical considerations for implementing weight-based dosing and monitoring (platelets, triglycerides, and cardiac parameters) in accordance with the summary of product characteristics (SmPC) guidance"

Clinical • Real-world • Real-world evidence • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

An HDAC Inhibitor Reduces Decline ofContractile Cross-Sectional Area andDecreases Fat Infiltration in Duchenne Muscular Dystrophy (ICNMD 2026) - P3 | "Givinostat treatment appeared to be associated with a reduced decline in muscle contractile area and reduced fat infiltration compared with placebo, indicating a consistent pattern suggestive of attenuation of muscle loss in patients with DMD."

Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

US Clinical Practice Experience With Givinostat (Dr. Perry Shieh) (ICNMD 2026) - "Sponsored By Italfarmaco"

Clinical • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy

Interim Safety Results From an Open-Label Extension Study of Givinostat Treatment For Duchenne Muscular Dystrophy (ICNMD 2026) - P2/3, P3 | "These results align with the known safety profile of givinostat. Early decreases in platelets and increases in triglycerides, consistent with treatment initiation, stabilized over time. One patient in the givinostat group had a single ECG over the safety threshold for QTc monitoring that subsequently resolved without intervention."

Clinical • Duchenne Muscular Dystrophy • Genetic Disorders • Hematological Disorders • Muscular Dystrophy • Thrombocytopenia

Open-Label Extension Analysis Suggests Givinostat Delays Age at Loss of Ambulation in Patients With DMD (ICNMD 2026) - P1/2, P2/3, P3 | "This updated interim analysis evaluated LoA in patients with DMD with 2 additional years of data from the OLE. These updated OLE data suggest that givinostat consistently delays the onset of LoA in patients with DMD. Relative to published estimates from a meta-analysis reporting a median age at LoA of 11.0-13.4 years in patients treated with corticosteroids alone, the median age at persistent LoA with givinostat was 17.3 years, suggesting a delay in the loss of ambulation in patients treated with givinostat."

Clinical • Duchenne Muscular Dystrophy

Meta-analysis of clinical trials assessing the safety of pharmacological treatments for muscular degeneration in Duchenne muscular dystrophy. (PubMed, BMC Pharmacol Toxicol) - "Precise ADR estimates can support decision-making in DMD care, helping clinicians balance safety and efficacy, address family concerns, and promote adherence by contextualizing treatment risks."

Journal • Retrospective data • Dermatology • Duchenne Muscular Dystrophy • Genetic Disorders • Hematological Disorders • Muscular Dystrophy

Study of Class I HDAC-1, -2, and -3 Inhibitors Designed by Bioisosteric Replacement of Zinc Binding Groups and Caps of Traditional Pan Inhibitors: An In Silico Approach. (PubMed, Curr HIV Res) - "This study identified two promising novel HDAC inhibitors, Hdi2 and Hdi10, for further experimental investigation and optimization as potential LRAs for HIV latency reversal. These findings support the rational design of selective HDACis using computational approaches as efficient and cost-effective methods for the identification of future LRAs."

Journal • Human Immunodeficiency Virus • Infectious Disease • HDAC1 • HDAC2 • HDAC3

Italfarmaco…announced that Health Canada has accepted for review the New Drug Submission (NDS) for givinostat (Duvyzat) for the treatment of Duchenne muscular dystrophy (DMD) (GlobeNewswire) - "Health Canada has granted givinostat Priority Review status, a designation reserved for drugs that may provide a significant improvement in the benefit-risk profile over existing therapies....The submission...is supported by data from the EPIDYS Phase 3 multicentre, randomized, double-blind, placebo-controlled trial (NCT02851797) evaluating givinostat in ambulant boys with DMD aged six and older."

Canada filing • Priority review • Duchenne Muscular Dystrophy

The National Institute for Health and Care Excellence (NICE) has approved givinostat (also named Duvyzat and manufactured by ITF Pharma) for the treatment of Duchenne muscular dystrophy (DMD), a chronic, progressive and a fatal genetic disorder which mainly affects boys. (Pharmacy Business) - "Givinostat was suggested after successful clinical trials done on populations, which proved that it increased the possibility of DMD patients to move freely by 5 years on an average unlike other treatments such as corticosteroids and supportive care."

NICE • Duchenne Muscular Dystrophy

EXPERT CONSENSUS ON TREATMENT GUIDANCE FOR FDA-APPROVED AND SECOND-GENERATION EXON-SKIPPING THERAPIES IN DUCHENE MUSCULAR DYSTROPHY (DMD): A RAND/UCLA MODIFIED DELPHI PANEL (ISPOR 2026) - "We aimed to characterize the current therapeutic landscape for DMD, including exon-skipping therapies, gene therapy, and givinostat, as well as emerging second-generation exon-skipping agents. Using the RAND/UCLA modified Delphi panel method, nine US experts (seven pediatric neurologists, two physical therapists) rated the likelihood of recommending FDA-approved therapies (eteplirsen, golodirsen, viltolarsen, casimersen, GT, givinostat) and the anticipated clinical value of investigational therapies with Phase 1/2 data (delpacibart zotadirsen, DYNE-251, WVE-N531, and NS-089/NCNP-02)... The panel reached consensus that approved exon-skipping therapies provide modest benefit, particularly in earlier stages, while early data suggest that second-generation exon-skippers may have the potential to offer greater functional improvement. However, trials remain in early stages, and the full risks and benefits of these therapies are not yet known. The findings highlight the rapidly..."

Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy

Structure evaluation coupled with all-atom molecular dynamics and advanced quantum mechanical DFT revealed kaempferol as a potent binding flavonol for the epigenetic-target HDAC9. (PubMed, J Biosci) - "Similar to givinostat, kaempferol exhibited tenable stability in bound form with this acetylation-eraser enzymatic protein. Most importantly, this flavonol demonstrated higher chemical reactivity and, as such, lower kinetic stability than givinostat which is quite important from a pharmacological perspective."

Journal • Gastric Cancer • Hepatocellular Cancer • Lung Cancer • Non Small Cell Lung Cancer • Oncology • Solid Tumor • HDAC9

Histone deacetylase inhibitor givinostat attenuates metabolic dysfunction-associated steatotic hepatitis and liver fibrosis (APASL 2026) - No abstract available

Epigenetic controller • Fibrosis • Hepatology • Immunology • Inflammation • Liver Cirrhosis • Metabolic Disorders

Safety and Tolerability of Givinostat: Evidence From Real-World and Clinical Practice. (PubMed, Ann Clin Transl Neurol) - "Our results confirm the safety profile observed in the pivotal study providing further evidence of the management of the drug in a real-world setting. In our experience, treatment with givinostat was well managed by maintaining a strict monitoring. This was facilitated by allowing families to perform blood tests in local labs, reducing the stress and burden of frequent visits to the hospital."

Journal • Real-world evidence • Duchenne Muscular Dystrophy • Dyslipidemia • Genetic Disorders • Hematological Disorders • Hypertriglyceridemia • Muscular Dystrophy • Thrombocytopenia

The Italian Medicines Agency (AIFA) has approved the reimbursement of givinostat for the treatment of Duchenne muscular dystrophy (DMD) in walking patients aged 6 years and older, receiving concomitant corticosteroid therapy, regardless of the underlying genetic mutation. (La Milano) - "Italfarmaco announced the decision in a statement, emphasizing that AIFA's decision follows the European Commission's conditional approval in 2025 and is based on the results of clinical trials demonstrating the drug's efficacy and favorable benefit-risk balance."

Reimbursement • Duchenne Muscular Dystrophy

European Commission Approves Duvyzat for the Treatment of Duchenne Muscular Dystrophy (GlobeNewswire) - "Italfarmaco S.p.A. announced today that the European Commission (EC) has granted conditional marketing authorisation for Duvyzat (givinostat)....It is approved for the treatment of Duchenne muscular dystrophy (DMD) in ambulant patients 6 years and older, regardless of the underlying genetic mutation, when taken together with corticosteroids. The EC decision follows the positive opinion of the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) on the 25th of April, 2025. The approval applies to all 27 EU member states, as well as Iceland, Liechtenstein, and Norway....The approval is based on the positive results of the EPIDYS Phase 3 multicentre, randomised, double-blind, placebo-controlled trial."

EMA approval • Duchenne Muscular Dystrophy

Single cell-based elucidation of molecularly distinct glioblastoma states and drug sensitivity (AACR 2025) - "The top six drugs predicted to target MES and PN subpopulations were tested in acute slices from GBM resections allowing direct characterization of the drug-mediated depletion of subpopulations predicted to be sensitive in adjacent drug vs. vehicle control treated slices [Zhao et al., 2021].Confirming our predictions, we observed statistically significant depletion of the MES and PN compartments by 3 of 3 (Dasatinib, Bexarotene and Prednisone) and 2 of 3 (Givinostat and Ixazomib) OncoTreat-predicted drugs, respectively. Given its high validation rate, this study paves the road and provides significant insight into the future development of combination trials where these drugs may be administered either concomitantly or sequentially. Moreover, the computationally inferred differential activity of subtype-specific MR proteins (CD44, OLIG2, KDM2B, EPHB3, TOP2B and others) was experimentally validated by FACS in cultured patients-derived GBM neurospheres."

Brain Cancer • CNS Tumor • Glioblastoma • Glioma • Oncology • Solid Tumor • EPHB3 • OLIG2

Treatment advances for Duchenne muscular dystrophy. (PubMed, Curr Opin Pediatr) - "This review summarizes the mechanism of action, key safety considerations and available evidence on motor function impact that these novel medications have demonstrated in DMD."

Journal • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy

Advances in the pharmacotherapeutic management of duchenne muscular dystrophy: an update. (PubMed, Expert Opin Pharmacother) - "Corticosteroids remain the standard of care, with newer agents, such as vamorolone offering alternative safety profiles...The recent approval of the histone deacetylase inhibitor givinostat is also discussed...While these advances provide important access, long-term efficacy, and safety data remain limited. Future progress will likely require rigorous clinical outcomes and rational combination of multimodal treatment strategies."

Journal • Review • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy • Respiratory Diseases