Update on givinosat - Friday 22 November
If you missed the news last week, there was a major development for some Duchenne families – an Early Access Programme (EAP) for a new Duchenne muscular dystrophy (DMD) treatment called givinostat is now open in the UK, which you can access through your neuromuscular consultant
How the EAP works
To learn more about what givinostat is, who is eligible for the EAP, and how it works, check out last week’s article on our website here.
This is big news, however we know that it has not been straightforward for everyone. And we have been listening to you. We know many of you have already reached out to your neuromuscular consultant, and have been disappointed by the response. We knew this wasn’t going to be easy, and it would take time to set up the EAP – but it’s still very frustrating. It’s a challenging time for the NHS, and we recognise the challenges they face, but that doesn’t mean we’re helpless. And, most of all, we can pull together as a community. That’s why we wanted to suggest some actions you could take.
In this article you’ll find:
– Actions for everyone in our community!,
– Actions for families who are eligible for the EAP,
– Actions for families who aren’t.
Duchenne UK has been doing what it can. Along with Muscular Dystrophy UK, we’ve written to the Chief Executive of all of the NHS Trusts in the UK that have a doctor who is part of the NorthStar clinical network, telling them to give neuromuscular teams the resources they need to deliver the EAP to patients. You can find a copy of that letter here. Duchenne UK is also in close contact with the NorthStar clinicians on this subject, as well as regulators.
Suggestions for everyone
The EAP is only temporary. While we are working on getting as many as possible access to the EAP now, we also need to look ahead to the future.
The Medicines and Healthcare products Regulatory Agency (MHRA) will decide on whether to approve givinostat as a treatment early next year. If they grant it a licence, we want that licence to be as broad as possible. In the USA, the Food and Drug Administration (FDA) approved givinostat for all DMD patients aged 6 years old and over that are ambulant and non-ambulant – and we want the MHRA to mirror this decision.
We are urging everyone in our community to write to the MHRA and let them know how important it is for givinostat to have a broad label which includes non-ambulant boys as well. We have made a template you can use here, and the best email address to use is: [email protected].
We encourage you to personalise the letter – share your story and explain why you want access.
Suggestions if your son is eligible
Have a look at the full exclusion and inclusion criteria at the end of this article on our website. If you think your son is eligible, we recommend doing the following:
– Contact your neuromuscular consultant, and;
– ask them if they think your son would benefit from givinostat,
– ask them to record your interest in taking part in the EAP,
– ask them to keep you updated on the availability of the EAP in their clinic.
– If your consultant does not think their Trust will take part in the EAP, write to the Trust’s CEO and ask them to change their mind. You can find a suggested template letter here.
– Keep us updated! Let us know if you can or cannot access the EAP, and where you are in the country. We will be tracking this information, message our Senior Policy Manager, Will Pender, with the details: [email protected].
Suggestions if your son is not eligible
We know that many people will be devastated by the eligibility criteria of the EAP, which excludes boys who are non-ambulant. It is hugely painful news for so many families, and we feel that pain too. We want this treatment available for everyone who could benefit, which is why we are asking the whole community to write to the MHRA in the section above.
What is next?
We will keep you informed as givinostat progresses through the British regulatory system, and as the EAP develops. If you have any further questions or comments on givinostat, please contact our Senior Policy Manager, Will Pender: [email protected].