Presenting seven abstracts demonstrating consistency of effect, long-term safety, and reduced progression of disease with givinostat
Results also demonstrate delayed respiratory decline with givinostat
CONCORD, Mass., Feb. 20, 2025 /PRNewswire/ -- ITF Therapeutics LLC, the U.S. affiliate of Italfarmaco, today announced that seven abstracts have been accepted as poster presentations at the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference to be held March 16-19, 2025, in Dallas, Texas.
The presentations include data highlighting the long-term safety and efficacy of treatment with givinostat in patients with Duchenne muscular dystrophy (DMD). Presentations also include analyses of the effect of givinostat on respiratory function in patients with DMD before and after loss of ambulation, disease progression models comparing the standard of care with givinostat, and updates on the design of an observational study to assess real-world outcomes of patients treated with givinostat. Please see the Indication and Important Safety Information for DUVYZAT™ (givinostat) below.
"In 2024, we attended our first MDA Clinical and Scientific Conference to listen to the Duchenne community and learn about their unique needs and goals as we prepared for the U.S. FDA approval of DUVYZAT for the treatment of DMD," said Matt Trudeau, President, ITF Therapeutics. "These insights were instrumental in shaping our commercialization strategy that enabled us to provide access to hundreds of patients within just the first few months. One year later, we are looking forward to returning to this important meeting to provide the community with updates including a range of data on DUVYZAT clinical performance and information about our ongoing research on the role of HDAC inhibition in the management of DMD."
Poster presentations
As an Impact Sponsor of the MDA Clinical and Scientific Conference, ITF Therapeutics is also supporting two special events during the meeting:
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 www.DUVYZAT.com.
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 www.itftherapeutics.com.
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 www.italfarmaco.com.
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
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.
To report SUSPECTED ADVERSE REACTIONS, contact ITF Therapeutics LLC at 1-833-582-4312 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
Please see full Prescribing Information and Medication Guide.
DUVYZAT is a registered trademark of Italfarmaco S.p.A.
U.S.A.
Media inquiries: Brian Connor |+1 212 253 8881| bconnor@cglife.com
Other inquiries: Patient Advocacy and Communications Lead U.S.A.| c.allen@itftherapeutics.com
Global
Media inquiries: Charlotte Spitz or Jacob Verghese |+49 (0)151 7441 6179| italfarmaco@trophic.eu
Other inquiries: Patient Advocacy and Communications Lead| s.parker@italfarmaco.com
C-DUV-US-0303 02/2025
SOURCE ITF Therapeutics LLC