Summary
We are excited to announce that Givinostat, a new HDAC (histone deacetylase) inhibitor treatment currently under regulatory review, is now available to eligible Duchenne patients via the UK’s Early Access Programme (EAP), sometimes termed as Expanded Access Programme.
Although the treatment has yet to achieve regulatory approval in the UK and EU, ITF Pharma UK (the treatment manufacturer) has successfully applied for the EAP for Givinostat. The aim of the EAP is to allow free-of-charge access to Givinostat until regulatory approval is in place, driven by the lack of currently available treatment for Duchenne patients.
Access to the EAP must be through one of the 23 NorthStar Centres across the UK. Which of these 23 will be able to provide access to the EAP will be decided at the individual NHS Trust level. The treatment is provided free-of-charge by the manufacturer to both the NHS Trust and patient. However, any associated administrative costs with the clinical practice’s enrollment on the EAP will be for the individual NHS Trusts to cover.
If you are interested in the EAP for Givinostat, and feel you meet the eligibility criteria, you must contact your regular neuromuscular clinician to enquire about the programme and whether the centre is able to provide you access. Decisions are made on a case-by-case basis for patients aligned to eligibility criteria.
If you have additional questions about accessing services at a NorthStar Centre, reaching out to your clinician or contacting Muscular Dystrophy UK’s helpline at 0800 652 6352 is recommended.
What is Givinostat?
Givinostat is a new form of epigenetic drug designed to be a supportive treatment alongside current corticosteroid use. Givinostat is an innovative treatment designed to reduce muscle inflammation, limit scarring and promote muscle regeneration, aiming to slow the progress of Duchenne muscular dystrophy.
The treatment is not mutation specific, but is currently only available for ambulatory boys aged 6 years and older, who meet the below eligibility criteria.
Eligibility Criteria
- A confirmed diagnosis of Duchenne muscular dystrophy
- Age six and above, and ambulant (able to walk)
- On stable corticosteroid for at least six months prior to start the treatment
- Time to stand up: less than 10 seconds
- Not a candidate for any licensed or standard-of-care Duchenne muscular therapy option, except for Corticosteroids, available at the time of inclusion
- Patient is not eligible for any ongoing clinical trial for Duchenne muscular dystrophy
- Patient must be willing to use adequate contraception
- Residing lawfully in the UK on a properly settled basis.
Exclusion Criteria
- Patient registered for another EAP
- Use of any current Duchenne muscular dystrophy investigational drug
- Patient is participating in any ongoing givinostat clinical trial
- Patient is participating in an ongoing other clinical trial
- Have platelets count, at < Lower Limit of Normal (LLN)
- Have Triglycerides > 300 mg/dL (3.42 mmol/L) in fasting condition
- Patients who are at an increased risk for ventricular arrhythmias / concomitant use with other drugs that prolong the QTc interval
- Symptomatic cardiomyopathy or heart failure and/or left ventricular ejection fraction <45%
- Have any hypersensitivity to the components of the EAP medication
- Have a sorbitol intolerance or sorbitol malabsorption or have the hereditary form of fructose intolerance.
Regulatory Standing
For families in the UK:
Givinostat is not yet approved in the UK, The MHRA (The Medicines and Healthcare products Regulatory Agency) are currently reviewing Givinostat for authorised use, where it will then be assessed by the NICE (National Institute for Health and Care Excellence) and SMC (Scottish Medicines Consortium) to determine if it is to be recommended for use under the NHS across the UK. An initial decision by the NICE is expected in 2025. This EAP offers a vital opportunity for eligible patients to access the treatment earlier.
For families in the EU:
The EMA are currently reviewing Givinostat to determine if it will be recommended for use in EU countries.
For Families in the US:
The FDA has approved Givinostat for use in Duchenne boys aged 6 years and older in the United States.
FAQs
– How does the EAP differ from a clinical trial?
Early access programs provide a potential pathway for patients diagnosed with serious and/or life-threatening diseases or conditions to gain access to investigational products. This allows patients to receive treatment outside of a clinical trial setting, when no comparable or satisfactory alternative therapy options are available.
– What happens if a patient is no longer ambulant during the EAP?
If a patient starts Givinostat via the EAP (by definition must meet the inclusion criteria) then the treatment would continue to be provided free-of-charge for this patient until either national reimbursement (by NICE/SMC), or if not reimbursed, until the clinician deems it is no longer needed. This is the case for both loss of ambulation, and reduction in time to rise test.
Contact Us
We understand that navigating treatment options for Duchenne muscular dystrophy can be an overwhelming and emotional journey for families. The Early Access Programme for Givinostat offers a new pathway for eligible patients to access this promising treatment ahead of regulatory approval. We hope this opportunity provides a sense of hope and progress in the fight against Duchenne. If you have any questions or need support, please reach out to your neuromuscular care team or connect us at info@actionducehnne.org who are here to guide you through this progress.
Action Duchenne particularly want to acknowledge and support those who, at his moment, may not be eligible for this treatment and may find this news challenging. Please know that our science team and support officers are here to offer guidance and assistance during this difficult time
Our science team has produced a series of bite-sized science videos to help you digest the latest scientific advancements with ease. Click the links below to find our resources:
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
Winter Webinar – NICE & Drug Approval
