NICE has recommended givinostat (Duvyzat) for ambulant Duchenne muscular dystrophy patients aged 6 years and older through the NHS Innovative Medicines Fund following a commercial agreement with NHS England. The treatment is expected to benefit around 530 patients in England.
Written By: Chikkula Pavan Kumar, PharmD
Reviewed By: Pharmacally Editorial Team
The UK’s National Institute for Health and Care Excellence (NICE) has recommended givinostat for treating Duchenne muscular dystrophy (DMD) in ambulant patients aged six years and older who are able to walk or stand, with or without support, at treatment initiation.
The recommendation follows a commercial agreement between NHS England and ITF Pharma UK, enabling immediate funding through the Innovative Medicines Fund. Marketed as Duvyzat, givinostat becomes the third NICE-recommended therapy for DMD.
NICE said around 530 people in England are expected to benefit from access to the treatment. The recommendation applies to the patient population studied in clinical trials rather than the full licensed indication.
Givinostat received UK regulatory approval from MHRA in December 2024 and has been available through an early access programme since November 2024.
DMD is a rare, progressive, and fatal muscle-wasting disorder caused by the absence of dystrophin, a protein essential for muscle function. The disease mainly affects boys, with symptoms typically appearing around age three. Patients gradually lose mobility and eventually require full-time care and respiratory support as the disease progresses to affect the heart and lungs.
Clinical evidence reviewed by NICE suggests givinostat may extend the period ambulant patients remain able to walk by about five years on average compared with standard care, including corticosteroids and supportive management. The findings were based on outcomes measured in the North Star Ambulatory Assessment (NSAA) and other functional assessments evaluated in clinical trials. However, NICE noted uncertainty remains regarding the magnitude of long-term benefit because of limitations in the available evidence.
Helen Knight director of medicines evaluation at NICE said the agreement enables access to a promising treatment while recognising uncertainties around its overall benefit. She also acknowledged testimony from patient representatives describing the significant burden of DMD on patients and families and the need for additional treatment options.
Dr Zubir Ahmed Health Innovation and Safety Minister said the approval gives patients access to a treatment that may help slow disease progression and reflects NHS efforts to secure timely access to innovative therapies.
NICE added that the appraisal committee accepted a greater degree of uncertainty than usual because of the challenges of collecting evidence in rare diseases, including quality-of-life data for patients and carers. After applying the maximum severity weighting of 1.7, the committee concluded givinostat represented a cost-effective use of NHS resources.
Reference
NICE approves Duchenne muscular dystrophy treatment givinostat after commercial deal sealed | NICE
About the Writer
Chikkula Pavan Kumar (LinkedIn) is a Doctor of Pharmacy with a keen interest in clinical pharmacy, pharmacovigilance, and evidence-based practice. In his words, he is passionate about patient safety and translating complex medical information into clear, research-driven communication.