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Trikafta drug US Name - Kaftrio drug UK Name

Joseph260268 profile image
9 Replies

Trikafta drug US Name - Kaftrio drug UK Name

Can someone please tell me if the above drug is only offered to people who have Cystic Fibrosis in the UK? i.e Would it not be offered to anyone who has Severe Bronchiectasis or anyone who could no longer have a Lung Transplant?

Kind regards as always

Joe x

9 Replies
Littlepom profile image

It is only offered to certain cf patients based on eligibility through DNA. Bronchiectasis does not have a DNA origin but is caused by many other factors. So sadly, kaftrio would not be of benefit to us bronchs. There is another member on here who has vast experience of cf and knows all about kaftrio. Hopefully he will see this and talk to you.

Joseph260268 profile image
Joseph260268 in reply to Littlepom

Thank you.

Hi Joe. As Littlepom says, the short answer is no, it’s not available to anyone other than cf patients at present, and even amongst cf patients, there is a specific criteria for accessing it. It’s quite complex, but I think it may be useful if I explain it a bit so you understand why that’s the case, as well as our experience with it. I hope so, anyway, and will apologise in advance if you disagree!

All the available modulating drugs like Kaftrio (which is a combination of 3 different modulators) are designed to work by correcting the underlying cause of disease symptoms. In cf, we know that the underlying cause is faulty transport of chloride ions in and between the cells in the body, causing thick secretions (everywhere, not just in the lungs), and that faulty transport is caused by having two genetic mutations of something called the cftr gene. To have cf, you have to have two mutations of this gene, one from each parent. There are 2000 known mutations that can occur, and they’re grouped by how they effect the chloride transport. There are 6 possible groups, but to give you some examples, some mutations mean the cells don’t produce enough chloride. Some mean that enough is produced but it’s faulty so doesn’t work. Or that there’s enough and it works but it never reaches where it’s needed on the cell surface. Someone with cf might have two of exactly the same mutation, or two mutations from the same group, or two mutations from very different groups. You only need to fix one gene to get rid of cf, but as there are so many possible mutations, any drug designed to treat the underlying dysfunction either has to be able to fix all the various ways the cells could be broken, which can be very different mechanisms, or will only work for some people. At present the available modulators are the latter, so only people with a mutation that we know (or think) can be fixed by the modulators are eligible, although about 90% of all cf patients have at least one copy of the most common gene, which is the main gene Kaftrio has been shown to work on, and one of the reasons people have got so excited about it.

More research is being done on which mutations definitely respond to kaftrio, in part because if 90% carry at least one right gene, then there are 10% of cf patients worldwide who don’t currently have access to any modulator at all due to having two incompatible genes. My 14 year old is technically in the 10%, but got Kaftrio off-licence last June after an experiment in the US showed one of her mutations responded in lab tests. Because she has more severe disease than many her age and she’s been losing lung function rapidly for the last three years, that was why they decided she could still have it. The tests since have shown that it’s made her chloride transport more normal than it was, but it’s still abnormal overall, and in terms of benefit, we haven’t had anything like the miraculous results that some have had. I personally think the press coverage has been a little bit misleading; for some people it’s been life-changing, absolutely, but even for those with the ‘right’ genetics, not everyone has found much benefit from it, and the results overall are quite individual even where someone on paper would be expected to respond well. There are also some people who can’t have it due to liver issues (liver disease occurs for around 30% of patients with cf, and modulators have been found to cause liver issues) and some people have stopped taking it due to not finding it a tolerable drug. So overall, yes, it’s been a positive for many, but those whose experiences haven’t been as positive either don’t speak up because they don’t want to upset anyone, or they’re not really heard over the people understandably thrilled that they’ve had jumps of 10% fev1 (sometimes more) and been able to be taken off the transplant list.

Although the lab test showed response for one of her two mutations, in Bod’s case, the only really meaningful benefit we’ve seen is a reduction in the rate of fev1 decline. We won’t be able to say for sure until we’ve done the full year, but it looks like she’s gone from losing 5 or 6% fev1 a year to around 2 or 3%; that’s not to be sniffed at and is really positive when you look at the buffer that might give us before transplant is needed (we were headed towards maybe needing one as young as 18 or 19, but with a halved rate of decline we might be talking late 20s or even older), but it’s a far cry from all the headlines suggesting we’ve somehow almost cured the disease for everyone. Other than the slowed rate of lung damage, she is a bit more stable generally, but she still has the same frequency and severity of exacerbations, she still needs all the treatments she’s been having for years (in part because the modulators can’t do anything about chronic infection, so if you’ve already got pseudo or another permanent bacteria, that still has to be managed regardless), and her life is still very much impacted by her cf day to day. It also doesn’t undo the damage already done; as I understand it, the gains in fev1 come from expelling old mucus trapped at the bottom of the lungs rather than fixing scarred and damaged lung tissue, and even with a purge as it’s referred to, some people still may not see any increase, Bod included. She had a purge and cleared huge volumes of sputum for the first 24 hours and the docs had high hopes we’d see an improvement in fev1 as a result, but that wasn’t the case.

All of the above said and done, there are studies happening in other conditions now, although I believe it’s primarily with COPD at this stage. The reason they’re being done is because although the genetic element isn’t present, the belief now is that most if not all obstructive diseases (including ncfb, all variants of COPD, and asthma) are actually a spectrum of cftr dysfunction that has cf at the most severe end. All of these other conditions are found as separate elements of the lung disease and damage seen in cf, and if cftr is the root to all of them, albeit with a different set of triggers, then it stands to reason that drugs like Kaftrio might be of benefit to people with these other conditions. The trials are in the early stages, and mostly using the earlier generation drugs like ivacaftor and orkambi rather than the Kaftrio combination at this stage, but they are happening and have been for the last couple of years. Only time will tell if they are viable outside of cf, though.

Patk1 profile image
Patk1 in reply to

My sons friend in mid 30s lung function was down to 24% & he wasn't able to do much with his young children,had to give up his sports until he was given kaftrio 2yrs ago.his lung functions gone up to 58%,he can play with children,and even ran a marathon last summer!So far,it's v successful for him x

Joseph260268 profile image
Joseph260268 in reply to Patk1

Does your friends son have CF?

in reply to Patk1

As I said, for some it absolutely has been life-changing, but even with the right genetics, it doesn’t work for everyone. That isn’t really acknowledged.

Alice70 profile image
Alice70 in reply to Patk1

As a broncheicstasis suffer ,thank you very much for the explanation very informative, the best of luck to your daughter I hope you find medication that works for her in the near future ,good luck to you both x

Joseph260268 profile image
Joseph260268 in reply to

Thanks for your reply it was very helpful.

cofdrop-UK profile image
cofdrop-UK in reply to

An amazingly informative explanation, as always Charlie.Love to you both ❣️❣️

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