Action Duchenne is pleased to share great news with the Duchenne community. Givinostat has been approved by the Medicines and Healthcare products Regulatory Agency (MHRA) for the treatment of Duchenne muscular dystrophy (DMD). The MHRA confirm full approval for patients, 6 years and older, that start treatment when ambulatory, and issue a conditional approval for patients that start treatment when no longer ambulatory
This UK approval is supported by evidence from the results of the EPIDYS Phase III 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 Givinostat twice daily or placebo, in addition to glucocorticosteroid treatment. The EPIDYS study met its primary endpoint demonstrating that patients on Givinostat showed a statistically significant and clinically meaningful difference in time to complete the four-stair climb assessment. Givinostat also showed favourable results on key secondary endpoints including North Star Ambulatory Assessment (NSAA), and fat infiltration evaluation by magnetic resonance imaging. Most adverse effects observed with Givinostat in the EPIDYS study were mild to moderate in severity.
Florence Boulton, CEO of Action Duchenne, expressed her delight at this milestone:
“We are pleased that the MHRA has approved Givinostat as a treatment for Duchenne muscular dystrophy. This is a significant step forward for the Duchenne community, bringing new hope to people living with Duchenne and their families.
At Action Duchenne, we are proud to have represented the Duchenne community’s perspective during the approval process, ensuring that the voices of those living with Duchenne were heard.
Givinostat offers healthcare professionals a new treatment option, independent of genetic mutation or ambulatory status. We remain committed to supporting families as they explore this option and to working with the MHRA, clinicians, and the community to improve outcomes for everyone affected by Duchenne.”
Further data is required for the efficacy in non-ambulatory patients and will be subject to review following completion of additional clinical trials, as detailed in the condition associated with approval.
Professor Francesco Muntoni (Director of the Dubowitz Neuromuscular Centre at UCL Great Ormond Street Institute of Child Health) & Professor Tracey Willis (Consultant Paediatric Neurologist, The Robert Jones and Agnes Hunt Orthopaedic Hospital, and part of the EPIDYS study group), say, “With MHRA approval, givinostat provides healthcare professionals with a novel non-corticosteroid-based treatment option for DMD, independent of the underlying genetic mutation, and ambulatory status.”
Italfarmaco has significantly expanded its rare disease presence through the formation of a new fully owned subsidiary, ITF Pharma UK. ITF Pharma UK will be responsible for the commercialisation of Givinostat in the U.K.
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. This approval highlights the dedication of Italfarmaco’s research and clinical teams, and of ITF Pharma UK, to support the DMD community.”
MHRA Press release can be found here: https://www.gov.uk/government/news/givinostat-conditionally-approved-to-treat-patients-with-duchenne-muscular-dystrophy-dmd
Overview of Givinostat can be found here: https://www.actionduchenne.org/givinostat-epigenetics-an-overview/
Please do not hesitate to reach out to us if you have any questions: info@actionduchenne.org
