Orkambi: 1,000 days on

I first started Orkambi as part of a clinical trial nearly 1,000 days ago. Originally the drug was not funded by the Irish Government and campaigns and debates were under way during the time I was on the trial to get the drug funded so all Irish patients with the most common cystic fibrosis (CF) gene, Double Delta F508, could have the first ever drug that would help with our gene – Orkambi.

Vertex Pharmaceuticals initially supplied the drug to 500 Irish CF patients over the age of 12. The drug was made available to six to 11-year-olds in 2018 and, in January 2019, it was announced 70 patients under six years of age would also be given access to the drug.

CF is an inherited disease that primarily affects the lungs and digestive system of about 1,300 children and adults in Ireland. It causes the body to produce a thick, sticky mucus that clogs the lungs and obstructs the pancreas. It also stops natural enzymes from helping the body break down and absorb food.

There is no cure.

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The trial I participated in, which lasted more than six months, was a double-blinded trial. That means that the drug I was taking could have been a placebo and not Orkambi. While I and my consultant did not know whether it was the real deal or not, I am convinced it was. My confidence is based on the amazing results I saw after a few months on the trial.

Results

While I was never underweight, I noticed my weight started rapidly going up. I’d say in a month I gained about 10kg which, for someone with CF, is amazing. If we get an infection, we tend to lose weight quite easily, so to have a reserve for when anything like that happens is amazing.

After finishing the drug trial, I also noticed how much I had grown in height.

My lung function didn’t increase much, maybe about 4-5 per cent, but it helped to prevent it from lowering and it kept it stable.

From being on Orkambi I feel so much better overall, I have more energy and feel I can do much more. I can go for longer walks, I can do physical activity longer and it just makes me feel so much more energetic.

A few months ago I had a CT scan of my lungs, which shows if there has been any further damage. To my and my family’s surprise, no further damage showed, and the CT scan actually looked the same as it did one to two years ago.

Nearly 1,000 days later I feel Orkambi has given me more energy.

My weight has maintained itself and my lung function hasn’t decreased at all and has stayed the same, and I am still slowly growing.

I am glad I had the opportunity to start Orkambi earlier and effectively get a head-start. It was so great to see all these changes happen.

Orkambi doesn’t work for everyone, and everyone notices different benefits, some better than others but there is no doubt for me that Orkambi has and did change my life.

Now it’s time to get excited for the future drugs that are coming down the line such as the triple combo drug which could be out next year. This drug will hopefully really transform the lives of people living with CF.