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Associated Press
17-03-2025
- Health
- Associated Press
ITF Therapeutics LLC Presents Long-Term Data of DUVYZAT™ (givinostat) for Treatment of Duchenne Muscular Dystrophy at MDA Clinical and Scientific Conference
Seven abstracts highlight long-term effects, consistent safety profile, and delayed disease progression with givinostat Results also show that treatment is associated with delayed decline in respiratory function CONCORD, Mass., March 17, 2025 /PRNewswire/ -- ITF Therapeutics LLC, the U.S. affiliate of Italfarmaco, today announced the presentation of seven abstracts at the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference being held March 16-19, 2025, in Dallas, Texas. The poster presentations include data from the Phase 3 EPIDYS study and the company's ongoing open-label extension study in patients with Duchenne muscular dystrophy (DMD) treated with givinostat. Analyses assess the long-term safety and efficacy of givinostat as identified through measures including data on disease progression and respiratory function. Please see the Indication and Important Safety Information for DUVYZAT™ (givinostat) below. 'This year's MDA Clinical and Scientific Conference arrives at a meaningful moment for our team as we reflect on the one-year anniversary of the U.S. FDA approval of DUVYZAT for the treatment of DMD in people 6 years of age and older,' said Matt Trudeau, President, ITF Therapeutics. 'We are honored to participate in this important meeting and look forward to sharing new insights from our ongoing research, including clinical data from our open-label extension study in DMD. These abstracts assess the long-term efficacy and safety of DUVYZAT, and we are grateful for the opportunity to present this data to the Duchenne community. As a core focus of our work, we strive to provide people living with DMD and their families and clinicians with the resources and information they need to make informed treatment decisions.' One poster presented by Krista Vandenborne, Ph.D., Chair, Department of Physical Therapy, University of Florida, includes data from the Phase 3 EPIDYS study and from an ongoing open-label extension study evaluating the long-term safety, tolerability, and efficacy of givinostat in boys aged ≥ 6 years with DMD. Using propensity score matching based on baseline functional test results and type of steroid, 142 patients from EPIDYS and the extension study treated with givinostat and steroids were matched with 142 patients from two DMD natural history studies treated with steroids only. Results were assessed based on the median age at which DMD progression milestones such as persistent loss to perform 4-stair climb, loss of rise from floor, and loss of ambulation, occurred. The median age at loss of ambulation in patients treated with givinostat was 18.1 years, compared to 15.2 years in the control group. A second poster presented by Craig M. McDonald, M.D., Chair, Department of Physical Medicine & Rehabilitation, UC Davis Health, evaluates the effect of givinostat on pulmonary function in patients who experienced loss of ambulation during follow-up. Results were assessed based on a comparison of forced vital capacity (FVC) percent trajectories from patients treated with givinostat and steroids in the EPIDYS and extension studies to FVC percent trajectories from patients treated with steroids only in a natural history study of disease progression. A related poster presented by Erika L. Finanger, M.D., M.S., Professor of Pediatrics, Oregon Health & Science University, includes results from an analysis using a validated DMD clinical trial simulation tool to assess differences in the time course of worsening FVC in patients treated with givinostat or standard of care. A separate poster presented by John F. Brandsema, M.D., Pediatric Neurologist, Division of Neurology, Children's Hospital of Philadelphia, evaluates safety data from the ongoing open-label extension study of givinostat in patients who completed or who were screened but not randomized into previous studies of givinostat for the treatment of DMD. All treatment-emergent adverse events were consistent with the known safety profile of givinostat, with no new safety signals observed in patients continuing the treatment. Other presentations include disease progression models comparing the standard of care with givinostat and details related to the design of an observational study to assess real-world outcomes of patients treated with givinostat. A subgroup analysis of an off-target population will also assess givinostat efficacy in a population with relatively more advanced disease compared to target groups. 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) that 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 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, including anemia and neutropenia. Monitor platelets; dosage adjustment or discontinuation may be needed. Increased Triglycerides: An increase in triglycerides can occur; dosage modification may be needed. Discontinuation may be needed. Gastrointestinal Disturbances: Adjust dosage if moderate or severe diarrhea occurs. Antiemetics or antidiarrheal medications may be considered during treatment with DUVYZAT. Discontinue DUVYZAT if the symptoms persist. QTc Prolongation: Avoid use of DUVYZAT in patients who are at an increased risk for ventricular arrhythmias. Recommended Evaluation and Testing Before Initiation of DUVYZAT: Obtain and evaluate baseline platelet counts and triglycerides prior to initiation of DUVYZAT. Do not initiate DUVYZAT in patients with a platelet count less than 150 x 109/L. Monitor platelet counts and triglycerides as recommended during treatment to determine if dosage modifications are needed. In addition, in patients with underlying cardiac disease or taking concomitant medications that cause QT prolongation, obtain ECGs when initiating treatment with DUVYZAT, during concomitant use, and as clinically indicated. Most Common Adverse Reactions: Most common adverse reactions (≥10% in DUVYZAT-treated patients) are diarrhea, abdominal pain, thrombocytopenia, nausea/vomiting, hypertriglyceridemia, and pyrexia. C-DUV-US-0332 03/2025
Associated Press
06-03-2025
- Business
- Associated Press
Italfarmaco Provides Overview on Givinostat as Treatment for Duchenne Muscular Dystrophy: Progress in Global Access, Regulatory Milestones and Clinical Trials
Italfarmaco S.p.A. today announced a comprehensive update on the regulatory and clinical advancements for givinostat, the company's drug for the treatment of Duchenne muscular dystrophy (DMD). The update highlights key regulatory milestones and ongoing clinical trials. Givinostat has been granted regulatory approval in the US (March 2024) and the UK (December 2024), marking critical milestones in the global effort to make the therapy available to patients. In the UK, givinostat was granted full approval for ambulant patients six years and older, while conditional approval was given for non-ambulant patients. In the US, full approval was granted for patients six years and older regardless of their ambulatory status, further emphasizing the treatment's potential across the disease spectrum. The European Medicines Agency (EMA) is currently reviewing the Marketing Authorisation Application (MAA), with a Committee for Medicinal Products for Human Use (CHMP) opinion expected in the first half of 2025. Regulatory filings in additional countries are actively progressing as part of Italfarmaco's commitment to global patient access. Carlos Barallobre, CEO of Italfarmaco, said, 'At Italfarmaco, our unwavering commitment is to the Duchenne community. The givinostat approvals in the US and UK, along with the promising long-term data we are generating, bring us closer to our goal: ensuring that every patient, regardless of location, has access to a therapy that can make a difference in disease progression. We are diligently working with global regulatory bodies to expedite availability, because we understand that for patients and families affected by DMD, every moment counts.' Ongoing Clinical Trials in DMD Three clinical trials are currently underway in Europe and Canada, designed to expand the potential use of givinostat and to provide long-term follow up on treated patients: NCT05933057: Evaluating non-ambulant patients aged 9 years and older NCT06769633: Evaluating pharmacokinetics and safety in younger patients aged 2 to 5 years, with recruitment recently completed for the first cohort of 4- to 5-year-olds NCT03373968: Confirming long-term tolerability and efficacy of givinostat treated patients, with up to eleven years follow-up Paolo Bettica, MD, PhD, Chief Medical Officer at Italfarmaco Group, stated, 'Our continuing clinical trial evaluation seeks to further affirm givinostat's clinical value in Duchenne muscular dystrophy, where treatment options remain limited, and by precisely targeting the biological drivers of muscle degeneration. We look forward to our ongoing interactions with the regulatory bodies and thank the clinicians for their tireless efforts and the DMD community and families for their continuing trust.' About Duchenne Muscular Dystrophy DMD is a progressive neuromuscular disorder caused by a mutation in the DMD gene which affects the production of a protein called dystrophin. 3 Dystrophin is a critical component of the dystrophin-associated protein complex (DAPC) which supports the strength, stability, function and repair of muscle cells. In DMD, muscle fibres are highly susceptible to injury and this continuous muscle injury leads to chronic inflammation, impairment of muscle regeneration and muscle replacement by fibrotic and fat tissue. 4, 5-7 The disease primarily affects boys, with symptoms usually first seen between two and five years of age. Symptoms worsen over time affecting the ability to walk. Eventually, heart and respiratory muscles are affected, which are the two main causes of premature death. 8 DMD is one of the most severe and common forms of childhood muscular dystrophy, with a worldwide birth incidence of around 1 in 5,050 boys. 9 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 deacetylases (HDAC) inhibitor. HDAC is activity upregulated in DMD muscle and has the potential to modify the expression of certain genes and biological processes involved in muscle repair and inflammation. 10, 11 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 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. 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. 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. Supplementary appendix. Lancet Neurol. 2024;23. Ryder S, Leadley RM, Armstrong N, et al. The burden, epidemiology, costs and treatment for Duchenne muscular dystrophy: an evidence review. Orphanet J Rare Dis. 2017;12(1):79. doi:10.1186/s13023-017-0631-3 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. 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. Bez Batti Angulski A, Hosny N, Cohen H, et al. Duchenne muscular dystrophy: disease mechanism and therapeutic strategies. Front Physiol. 2023;14:1183101. 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. Walter MC, Reilich P. Recent developments in Duchenne muscular dystrophy: facts and numbers. J Cachexia Sarcopenia Muscle. 2017;8(5):681–685. 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. 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. Other enquiries: Patient Advocacy and Communications Lead| [email protected] KEYWORD: ITALY EUROPE SOURCE: Italfarmaco S.p.A. Copyright Business Wire 2025. PUB: 03/06/2025 05:00 AM/DISC: 03/06/2025 05:03 AM
