MHRA approves givinostat for all people with DMD aged six and older
We are overjoyed at the decision by the Medicines and Healthcare products Regulatory Agency (MHRA) to give approval to Duchenne muscular dystrophy (DMD) treatment givinostat for patients aged six years and older.
The MHRA confirm full approval for patients, six years and older, that start treatment when ambulatory (walking), and issue a conditional approval for patients that start treatment when no longer ambulatory (non-walking). Read Jack’s blog about the MHRA decision and his experience of taking part in the givinostat clinical trial here: joiningjack.org/about-joining-jack/blog/an-update-from-jack/
Trial data.
Results from the Italfarmaco Pivotal Phase 3 EPIDYS Study of givinostat in DMD showed that the clinical trial met its primary and secondary endpoints, showing efficacy in slowing down disease progression.
The drug was tested in clinical trials on tested on ambulant boys only and our concern was that the MHRA would approve the drug in this group only. Duchenne UK has been campaigning since April for the MHRA to approve givinostat and give it what is known as a broad label. More than 260 families have written in to express their views.
A huge thank you to everyone who has joined us in our campaign. This is a very special day for our community.
Our co-founder, Alex Johnson OBE, said:
"There is a cruel and bitter double punch in DMD. Once your child stops walking, not only can he not walk, but because clinical trials often measure the effectiveness of drug by seeing its impact on how far a boy can walk, boys who can’t walk are not included in clinical trials. This means that when a drug is shown to be effective, it’s only been proven to be effective in that clinical trial population i.e boys who can walk. And it historically only got approved for that patient group. With this decision by the MHRA, we are entering a new dawn. A dawn where regulators are demonstrating that where clinical data is compelling, compassion and flexibility can be used to benefit patients. We would like to thank everyone in the DMD UK community."
Some of you might know a lot about givinostat (sometimes known by the brand name Duvyzat), while others might not.
It is produced by Italfarmaco, and is an ‘HDAC inhibitor’. This means that it blocks enzymes called histone deacetylases (HDACs), which are involved in turning genes ‘on’ and ‘off’ within cells. It works by targeting pathogenic processes to reduce inflammation and muscle loss. You can read more about how it works from Professor Annemieke Aartsma-Rus on the website of the World Duchenne Organisation here.
Results from the Italfarmaco Pivotal Phase 3 EPIDYS Study of givinostat in DMD showed that the clinical trial met its primary endpoint: change in four-stair climb assessment from baseline to 72 weeks, which demonstrated the potential of givinostat to delay disease progression when added to corticosteroid treatment. Givinostat-treated boys also showed favorable outcomes on key secondary endpoints assessed in the study.
Accessing givinostat.
While the approval of givinostat as a DMD treatment in the UK is a key milestone, there is another hurdle to overcome. For givinostat to be made available to every patient who could benefit, it needs to be assessed for cost effectiveness by the National Institute for Health and Care Excellence (NICE) which will decide if it can be available on the NHS. The company will present to NICE in May 2025, which means it likely won’t be available on the NHS until the end of 2025.
Givinostat and the Early Access Programme.
In the meantime, givinostat is being made available under an Early Access Programme (EAP). Local NHS Trusts can take part in the scheme and gain access for eligible patients on a case-by-case basis. You can read the full eligibility criteria in our first update on the givinostat EAP here. If you think you are eligible, talk to your neuromuscular consultant who will be able to give you medical advice, and tell you if their Trust is taking part in the EAP.
The company has committed to providing access to givinostat through the EAP until the regulators make a decision. We currently do not know what impact the MHRA decision will have on the eligibility criteria EAP. We will update you when we know more.