Givinostat Hydrochloride

Now approved in the EU, Duvyzat offers an important treatment option for delaying Duchenne muscular dystrophy (DMD) disease progression Duvyzat is an orally administered treatment for DMD in patients 6 years and older who are able to walk The approval is based on Phase 3 EPIDYS trial data that demonstrated meaningful treatment benefits in ambulant patients MILAN, Italy, June 06, 2025 – Italfarmaco S.p.A. announced today that the European Commission (EC) has granted conditional marketing authorisation for Duvyzat ® (givinostat), a novel histone deacetylase (HDAC) inhibitor. 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. Italfarmaco is now working closely with national authorities and distribution partners to facilitate timely access to Duvyzat across the EU. “People living with DMD in Europe have long awaited new therapeutic options that can alter the course of this devastating disease. Until now, there have been limited approved treatments that address the underlying pathology of DMD across the broad patient population. That changes with the approval of Duvyzat, which slows disease progression and preserves muscle function — regardless of the gene mutation — by targeting disease mechanisms,” said Paolo Bettica, MD, PhD, Chief Medical Officer at Italfarmaco Group . “We are committed to working closely with health authorities and the DMD community to ensure timely access to this important new treatment across Europe.” “The EC’s approval of Duvyzat is a recognition of its therapeutic potential and a testament to Italfarmaco’s scientific excellence and commitment to innovation in rare diseases,” said Dr Francesco De Santis, President of Italfarmaco Holding and Chairman of Italfarmaco Group . “This milestone means that a broad range of patients with DMD have access to a new treatment. At Italfarmaco, this achievement reaffirms our focus on advancing therapies that can make a meaningful difference in people’s lives.” The approval is based on the positive results of the EPIDYS Phase 3 multicentre, randomised, double-blind, placebo-controlled trial (NCT02851797). In the EPIDYS study, a total of 179 ambulant boys six years of age or older received either Duvyzat twice daily or placebo, in addition to corticosteroid treatment. The EPIDYS study met its primary endpoint demonstrating a statistically significant and clinically meaningful difference in time to complete the four-stair climb assessment. Duvyzat also showed favourable results on key secondary endpoints including North Star Ambulatory Assessment (NSAA) and fat infiltration evaluation by magnetic resonance imaging. Specifically, Duvyzat treatment was associated with 40% less decline in cumulative loss of NSAA items, indicating Duvyzat’s potential to delay disease progression in affected individuals. Most adverse effects observed with Duvyzat were mild to moderate in severity. Results from this study were published in The Lancet Neurology in March 2024. 1 Long-term data from the ongoing EPIDYS extension study was compared to natural history cohorts using propensity score matching showing that the median age at loss of ambulation was 18.1 years in the Duvyzat group versus 15.2 years in controls. 2 The EC has granted Duvyzat conditional marketing authorisation in the EU. The approval makes Duvyzat available to ambulant DMD patients 6 years and older while Italfarmaco conducts additional clinical studies designed to further confirm and characterise its therapeutic benefit. Outside of the EU, Duvyzat was granted approval by the US Food and Drug Administration (FDA) in March 2024 for the treatment of DMD patients 6 years and older. In the UK, Duvyzat received approval by the Medicines and Healthcare products Regulatory Agency (MHRA) for patients 6 years and older who are ambulatory and granted conditional marketing approval for non-ambulatory patients. About Duchenne Muscular Dystrophy Duchenne muscular dystrophy (DMD) is a rare, progressive neuromuscular disorder caused by mutations in the DMD gene. Mutations in the DMD gene 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. 3-6 DMD primarily affects males, with symptoms typically appearing between the ages of two and five. As the condition progresses, muscle weakness worsens, leading to difficulty walking and eventually to loss of ambulation. Over time, the heart and respiratory muscles are also affected, which are the leading causes of premature death. 7 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. 8 About Duvyzat ® 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. 9,10 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.eu Other enquiries: Samantha Parker | Patient Advocacy and Communications Lead | RDEnquiries@italfarmacogroup.com References: Mercuri, E, Vilchez, JJ, Boespflug-Tanguy, O, Zaidman, CM, Mah, JK, Goemans, N. Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial. Lancet Neurol. 2024;23:393-403. Vandenborne, K., McDonald, C., Servais, L., Munell, F., Schara, U., Bertini, E., Comi, G., Blaschek, A., Cazzaniga, S., Bettica, P. U., & Mercuri, E. (2025, March). Givinostat in Duchenne muscular dystrophy: Effect on disease milestones [Poster presentation]. MDA Clinical & Scientific Conference, Dallas, TX. https://www.mdaconference.org/abstract-library/givinostat-in-duchenne-muscular-dystrophy-effect-on-disease-milestones/ ​Sandonà M, Cavioli G, Renzini A, et al. Histone Deacetylases: Molecular Mechanisms and Therapeutic Implications for Muscular Dystrophies. Int J Mol Sci. 2023;24(5):4306. https://doi.org/10.3390/ijms24054306 . Consalvi S, Saccone V, Giordani L, Minetti G, Mozzetta C, Puri PL. Histone Deacetylase Inhibitors in the Treatment of Muscular Dystrophies: Epigenetic Drugs for Genetic Diseases. Mol Med. 2011;17(5):457–465. https://doi.org/10.2119/molmed.2011.00049 . Bez Batti Angulski A, Hosny N, Cohen H, et al. Duchenne muscular dystrophy: disease mechanism and therapeutic strategies. Front Physiol. 2023;14:1183101. https://doi.org/10.3389/fphys.2023.1183101 . Giuliani G, Rosina M, Reggio A. Signaling pathways regulating the fate of fibro/adipogenic progenitors (FAPs) in skeletal muscle regeneration and disease. FEBS J. 2022;289(21):6484–6517. https://doi.org/10.1111/febs.16080 . Walter MC, Reilich P. Recent developments in Duchenne muscular dystrophy: facts and numbers. J Cachexia Sarcopenia Muscle. 2017;8(5):681–685. https://doi.org/10.1002/jcsm.12245 . Crisafulli S, Sultana J, Fontana A, Salvo F, Messina S, Trifirò G. Global epidemiology of Duchenne muscular dystrophy: an updated systematic review and meta-analysis. Orphanet J Rare Dis. 2020;15(1):141. https://doi.org/10.1186/s13023-020-01430-8 . 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 . 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May 6, 2025 – Italfarmaco S.p.A. announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to givinostat for the treatment of patients with polycythemia vera (PV), a rare haematologic cancer, for which treatment options are limited. “The FDA decision to grant givinostat Fast Track designation underscores the urgent need for innovative treatments for PV and highlights the potential of givinostat to make a meaningful difference,” said Paolo Bettica, MD, PhD, Chief Medical Officer at Italfarmaco Group. “We look forward to working closely with the FDA as we plan for completion of our Phase III clinical trial.” Givinostat is an orally administered histone deacetylase inhibitor (HDACi) with potential applications in both neuromuscular disorders and oncology. HDACis work by modulating key cellular pathways that regulate gene expression, offering promising therapeutic benefits across a range of diseases. Givinostat is currently being studied for its potential to treat PV, a rare blood cancer characterised by the overproduction of erythroid, myeloid, and megakaryocytic components in the bone marrow. PV commonly causes symptoms like headache, weakness, and itching, with severe complications including stroke, heart attack, and deep vein thrombosis, which are leading causes of mortality. Furthermore, PV carries a variable risk of progression to myelofibrosis or acute myeloid leukaemia. By targeting and modulating abnormal gene expression, givinostat may help control excessive cell proliferation driven by mutations such as JAK2V617F, commonly found in PV patients. This mechanism aims to reduce disease burden, alleviate symptoms, and improve long-term outcomes. The Phase III trial (NCT06093672) is currently enrolling patients with clinical sites open in Europe, the UK, Israel, and North America, with more sites expected soon. FDA Fast Track designation is a process designed to facilitate the development and expedite the review of new drugs or biologics that are intended to treat serious or life-threatening conditions and address unmet medical needs. This designation aims to accelerate the drug development and review process by encouraging early and frequent communication between the FDA and drug companies. Givinostat has received orphan drug designation by the FDA and the European Medicines Agency (EMA) for PV. In addition, givinostat (Duvyzat®) has marketing authorisations for Duchenne muscular dystrophy from the FDA and the MHRA1, with a positive opinion adopted by the CHMP2 and other regulatory processes currently ongoing. 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 60 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. [1] UK’s Medicines and Healthcare products Regulatory Agency [2] European Medicines Agency Committee for Medicinal Products for Human Use The content above comes from the network. if any infringement, please contact us to modify.