Italfarmaco in the NEWS
From Milan, Italy: Today, Italfarmaco announced that the U.S. Food and Drug Administration (FDA) has approved Duvyzat™ (givinostat) – a novel histone deacetylase (HDAC) inhibitor, for the treatment of patients 6 years or older with Duchenne muscular dystrophy (DMD) – a rare X-linked progressive and life-limiting neuromuscular condition with symptoms from early childhood.
From Italfarmaco
Paolo Bettica, MD, PhD, Chief Medical Officer at Italfarmaco Group said, “The FDA’s approval of Duvyzat for DMD, based on our robust and successful clinical development program, reflects Italfarmaco’s commitment to providing a safe and proven-effective therapy that can have a meaningful impact for people living with DMD. We are grateful for the support of those living with DMD and their dedicated caregivers, which played a central role in helping us reach this landmark FDA approval. Our focus now is to make Duvyzat available as a treatment for DMD management in the U.S. as quickly as possible.”
Dr Francesco De Santis, President of Italfarmaco Holding and Chairman of Italfarmaco Group added, “Duchenne muscular dystrophy is a disease with significant unmet medical need and Duvyzat has the potential to benefit a broad DMD patient population independent of the underlying gene mutation that causes the disease. The FDA approval highlights the dedication of Italfarmaco’s research and clinical teams to achieve this milestone for the company.”
The approval is based on the results of the pivotal multicenter, randomized, double-blind, placebo-controlled phase 3 EPIDYS 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 glucocorticosteroid treatment.
The EPIDYS study met its primary endpoint demonstrating that patients on Duvyzat showed a statistically significant and clinically meaningful difference in time to complete the four-stair climb assessment. Duvyzat also showed favorable results on key secondary endpoints including North Star Ambulatory Assessment (NSAA), and fat infiltration evaluation by magnetic resonance imaging. The majority of adverse effects observed with Duvyzat were mild to moderate in severity.
Results from this study were published in The Lancet Neurology in March 2024.
More From the Experts
Craig M. McDonald, MD, Professor at the Department of Pediatrics and Physical Medicine Rehabilitation at the University of California Davis Health and investigator for the EPIDYS trial added, “There is a tremendous unmet need for novel therapies in DMD that can achieve meaningful benefits for a broad range of patients. Duvyzat’s unique mechanism of action has shown a positive risk/benefit profile and the ability to delay disease progression, supporting its potential to become a key component of the standard of care for people living with DMD. I would like to thank all patients and their families for participating in the clinical trials and for making this approval possible.”
Pat Furlong, Founding President & CEO at Parent Project Muscular Dystrophy (PPMD) said, “We are thrilled with the FDA’s approval of Duvyzat, a new therapy for DMD. It is an oral medication that will be available to every person 6 years and older with DMD. This brings great hope for the Duchenne community, and we believe this will be a key therapy to prevent disease progression in Duchenne. Italfarmaco has significantly expanded its U.S. presence through the formation of a new fully owned subsidiary ITF Therapeutics LLC.”
ITF Therapeutics will be responsible for the commercialization of Duvyzat in the U.S. and the company is working closely with healthcare providers, patient advocacy groups and payors to make Duvyzat available to patients.
Duvyzat received priority review, orphan drug and rare pediatric disease designations from the FDA. A Marketing Authorization Application (MAA) for givinostat as a potential treatment for DMD has been submitted to the European Medicine Agency (EMA) and is currently under review. Italfarmaco has a global presence and is also working with other regulatory agencies.
Duchenne Muscular Dystrophy
DMD is a severe neuromuscular genetic disease characterized by progressive muscle weakness and degeneration and is the most common type of muscular dystrophy globally. DMD is caused by mutations in the dystrophin gene that result in the absence of a functional dystrophin protein. Without dystrophin, muscle fibers 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.
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. DMD incidence is approximately one in every 3500 – 6000 male births worldwide.
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 commercialization with proven success in many therapeutic areas including immuno-oncology, gynecology, neurology, cardiovascular disease and rare diseases. Italfarmaco’s rare disease unit includes programmed in Duchenne muscular dystrophy, Becker muscular dystrophy, amyotrophic lateral sclerosis and polycythemia vera.
About Duvyzat™
Duvyzat is an investigational drug discovered through Italfarmaco’s research and development efforts in collaboration with Telethon and Duchenne Parent Project (Italy). Duvyzat is a HDAC inhibitor that modulates the deregulated activity of HDACs in the dystrophic muscle, which is a major consequence of the lack of dystrophin associated with DMD. Duvyzat’s mechanism of action has the potential to inhibit HDAC pathological overactivity in an effort to address the cascade of events leading to muscle damage, thereby counteracting the disease pathology and slowing down muscle deterioration.
The product is an oral prescription medicine used for the treatment of DMD in people 6 years of age and older. It is not known if Duvyzat is safe and effective in children under 6 years of age.
Please see Full Prescribing Information and Medication Guide.
See the most important information about Duvyzat adverse effects that Includes Low platelet count, increased triglycerides levels, frequent watery loose stools (diarrhea) and vomiting.
Before taking Duvyzat, the DMD Patient should tell his healthcare provider about all his medical problems, including heart problems. The patient should also tell his physician about the drugs he is currently using including prescription products and over-the-counter products, vitamins, and herbal supplements.
Taking Duvyzat with certain other medicines may interact and cause serious side effects.
Prohost Observations
Duvyzat was needed for patients with DMD who have not been treated successfully or who haven’t been treated at all. The novel oral product, Duvyzat, can, alone and with other combination drugs, fill a gap left by current treatments.
News & Comments
March 22, 2024
Italfarmaco Received FDA Approval for Duvyzat™ for the Treatment of Duchenne Muscular Dystrophy
Italfarmaco in the NEWS
From Milan, Italy: Today, Italfarmaco announced that the U.S. Food and Drug Administration (FDA) has approved Duvyzat™ (givinostat) – a novel histone deacetylase (HDAC) inhibitor, for the treatment of patients 6 years or older with Duchenne muscular dystrophy (DMD) – a rare X-linked progressive and life-limiting neuromuscular condition with symptoms from early childhood.
From Italfarmaco
Paolo Bettica, MD, PhD, Chief Medical Officer at Italfarmaco Group said, “The FDA’s approval of Duvyzat for DMD, based on our robust and successful clinical development program, reflects Italfarmaco’s commitment to providing a safe and proven-effective therapy that can have a meaningful impact for people living with DMD. We are grateful for the support of those living with DMD and their dedicated caregivers, which played a central role in helping us reach this landmark FDA approval. Our focus now is to make Duvyzat available as a treatment for DMD management in the U.S. as quickly as possible.”
Dr Francesco De Santis, President of Italfarmaco Holding and Chairman of Italfarmaco Group added, “Duchenne muscular dystrophy is a disease with significant unmet medical need and Duvyzat has the potential to benefit a broad DMD patient population independent of the underlying gene mutation that causes the disease. The FDA approval highlights the dedication of Italfarmaco’s research and clinical teams to achieve this milestone for the company.”
The approval is based on the results of the pivotal multicenter, randomized, double-blind, placebo-controlled phase 3 EPIDYS 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 glucocorticosteroid treatment.
The EPIDYS study met its primary endpoint demonstrating that patients on Duvyzat showed a statistically significant and clinically meaningful difference in time to complete the four-stair climb assessment. Duvyzat also showed favorable results on key secondary endpoints including North Star Ambulatory Assessment (NSAA), and fat infiltration evaluation by magnetic resonance imaging. The majority of adverse effects observed with Duvyzat were mild to moderate in severity.
Results from this study were published in The Lancet Neurology in March 2024.
More From the Experts
Craig M. McDonald, MD, Professor at the Department of Pediatrics and Physical Medicine Rehabilitation at the University of California Davis Health and investigator for the EPIDYS trial added, “There is a tremendous unmet need for novel therapies in DMD that can achieve meaningful benefits for a broad range of patients. Duvyzat’s unique mechanism of action has shown a positive risk/benefit profile and the ability to delay disease progression, supporting its potential to become a key component of the standard of care for people living with DMD. I would like to thank all patients and their families for participating in the clinical trials and for making this approval possible.”
Pat Furlong, Founding President & CEO at Parent Project Muscular Dystrophy (PPMD) said, “We are thrilled with the FDA’s approval of Duvyzat, a new therapy for DMD. It is an oral medication that will be available to every person 6 years and older with DMD. This brings great hope for the Duchenne community, and we believe this will be a key therapy to prevent disease progression in Duchenne. Italfarmaco has significantly expanded its U.S. presence through the formation of a new fully owned subsidiary ITF Therapeutics LLC.”
ITF Therapeutics will be responsible for the commercialization of Duvyzat in the U.S. and the company is working closely with healthcare providers, patient advocacy groups and payors to make Duvyzat available to patients.
Duvyzat received priority review, orphan drug and rare pediatric disease designations from the FDA. A Marketing Authorization Application (MAA) for givinostat as a potential treatment for DMD has been submitted to the European Medicine Agency (EMA) and is currently under review. Italfarmaco has a global presence and is also working with other regulatory agencies.
Duchenne Muscular Dystrophy
DMD is a severe neuromuscular genetic disease characterized by progressive muscle weakness and degeneration and is the most common type of muscular dystrophy globally. DMD is caused by mutations in the dystrophin gene that result in the absence of a functional dystrophin protein. Without dystrophin, muscle fibers 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.
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. DMD incidence is approximately one in every 3500 – 6000 male births worldwide.
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 commercialization with proven success in many therapeutic areas including immuno-oncology, gynecology, neurology, cardiovascular disease and rare diseases. Italfarmaco’s rare disease unit includes programmed in Duchenne muscular dystrophy, Becker muscular dystrophy, amyotrophic lateral sclerosis and polycythemia vera.
About Duvyzat™
Duvyzat is an investigational drug discovered through Italfarmaco’s research and development efforts in collaboration with Telethon and Duchenne Parent Project (Italy). Duvyzat is a HDAC inhibitor that modulates the deregulated activity of HDACs in the dystrophic muscle, which is a major consequence of the lack of dystrophin associated with DMD. Duvyzat’s mechanism of action has the potential to inhibit HDAC pathological overactivity in an effort to address the cascade of events leading to muscle damage, thereby counteracting the disease pathology and slowing down muscle deterioration.
The product is an oral prescription medicine used for the treatment of DMD in people 6 years of age and older. It is not known if Duvyzat is safe and effective in children under 6 years of age.
Please see Full Prescribing Information and Medication Guide.
See the most important information about Duvyzat adverse effects that Includes Low platelet count, increased triglycerides levels, frequent watery loose stools (diarrhea) and vomiting.
Before taking Duvyzat, the DMD Patient should tell his healthcare provider about all his medical problems, including heart problems. The patient should also tell his physician about the drugs he is currently using including prescription products and over-the-counter products, vitamins, and herbal supplements.
Taking Duvyzat with certain other medicines may interact and cause serious side effects.
Prohost Observations
Duvyzat was needed for patients with DMD who have not been treated successfully or who haven’t been treated at all. The novel oral product, Duvyzat, can, alone and with other combination drugs, fill a gap left by current treatments.
Other Articles