Further to our previous article on the publication of the phase 3 EPIDYS trial positive results for Givinostat, a histone deacetylase (HDAC) inhibitor drug that works by targeting pathogenic processes to reduce inflammation and loss of muscle; we are excited to share the news that as a result of these published findings, the United States Food and Drug Administration (FDA) has granted approval to Italfarmaco S.p.A. for Givinostat (also known as Duvyzat) making it available for the treatment of patients aged 6 years and older in the USA.
“DMD denies the opportunity for a healthy life to the children it affects. The FDA is committed to advancing the development of new therapies for DMD,” said Emily Freilich, M.D., director of the Division of Neurology 1, Office of Neuroscience in the FDA’s Center for Drug Evaluation and Research. “This approval provides another treatment option to help reduce the burden of this progressive, devastating disease for individuals impacted by DMD regardless of genetic mutation.”
“The FDA’s approval of Duvyzat [Givinostat] 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,” said Paolo Bettica, MD, PhD, Chief Medical Officer at Italfarmaco Group. “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.”
Givinostat is a twice-daily orally administered treatment and will be available to patients 6+ years old, with the dosage determined by the individual’s body weight. The treatment is not mutation specific, which means it will be more broadly available to patients with DMD – a much needed additional treatment that has the potential to improve the standards of care for the Duchenne patients.
“There is a tremendous unmet need for novel therapies in DMD that can achieve meaningful benefits for a broad range of patients. Duvyzat’s [Givinostat] 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,” added 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. “I would like to thank all patients and their families for participating in the clinical trials and for making this approval possible.”
A Marketing Authorisation 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 S.p.A. are also continuing to work tirelessly with the MHRA and other relevant stakeholders towards Givinostat’s approval in the UK. The FDA’s approval, alongside significant results from the phase 3 clinical trials, will serve as positive support for the applications seeking to gain approval of Givinostat in the EU and UK.
Notable side effects of Givinostat:
The most common side effects of Givinostat are diarrhoea, abdominal pain, a decrease in platelets—which can lead to increased bleeding—nausea/vomiting, an increase in triglycerides (a type of fat in the body) and fever. Clinicians and health care providers will be advised to evaluate the patient’s platelet counts and triglycerides before prescribing Givinostat, and will require continued monitoring during treatment and dosages amended where necessary.
Action Duchenne remains steadfast in continuing to campaign and push for approval of viable treatments in the UK that improve the standards of care for all members of the Duchenne community.
Would you like to know more about Duchenne muscular dystrophy? Increase your knowledge and understanding of Duchenne with our bite-sized science video series.
Section 1 – Facts about Duchenne muscular dystrophy
Section 2 – Signs and Symptoms of Duchenne muscular dystrophy
Section 3 – Diagnosis of Duchenne muscular dystrophy
Section 4 – Crucial Genetic Terminology
Section 5 – Genetics – Blueprint of Duchenne muscular dystrophy
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Our 2024 Annual International Conference will be held on Friday 8th and Saturday 9th November 2024. The conference brings together families, clinicians, researchers and experts for 2 days of sharing knowledge and experience. It is an amazing opportunity to come together as part of the Duchenne community
Results from Italfarmaco Pivotal Phase 3 EPIDYS Study of Givinostat in Duchenne Muscular Dystrophy (DMD) Published in The Lancet Neurology
