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Lots of people are talking about the first-in-class PH drug sotatercept, which has recently been approved for use in America. Here, PHA UK Chair Dr Iain Armstrong explains what happens next - and what will be considered when it comes to making decisions for the UK...

Sotatercept is what’s known as ‘first-in-class’, and what makes it so exciting is that it’s treating part of the problem that’s not been treated before. It potentially is very important, but is it more important that the other PH drugs? Time will tell.  However, it’s the first new drug we’ve had for many years, so that’s the reason we’re talking about it so much.

It’s also a hot topic because it has recently been given a licence by the Federal Drug Agency, which means it can be used in America if patients fit the criteria and insurance companies will pay for it.

So where are we at in this part of the world? Sotatercept is yet to gain a European licence, but we think that is likely to happen before the end of 2024.

There are key differences between Europe, the UK, and America though. Once sotatercept has its European licence it will then need to get a UK licence, because we are no longer part of the European Union.

It’s important to remember that our health system is different to America’s, so obtaining a licence is only part of the issue. The next stage will be getting the drug commissioned by the NHS, and the challenge here is likely to be the cost. Because PH is a rare disease, drugs are very expensive.

Once it’s been licenced, the National Institute for Clinical Excellence (NICE) will put sotatercept through a process to decide whether the benefit of the drug is worth the cost to the NHS.

As a patient organisation, the PHA UK will be very much involved in this process – something we have done for different drugs over the last couple of decades. We can’t present clinical data, but we can present the patient narrative, and the stories that will help commissioners understand what it’s like to be impacted by PH. It is this involvement that we hope will persuade them that the benefits will be worth the cost.

Without a doubt, the biggest challenge in getting access to this drug will be its price. We have some idea how much it costs in different parts of the world, but drugs are sold at different prices in different countries. We need to be prepared for this challenge.

In summary, I think we can be very positive. It is very likely that sotatercept will get a UK licence, and this is a new drug that works in a different way by targeting the root cause of the disease.

However, we do need to be prepared for commissioning challenges. Here at the PHA UK we put every effort into any drug that we think has a place in the world of PH, and as Chair of this organisation I can assure you we will fight on behalf of the PH community.

What is sotatercept and what makes it different?

Sotatercept is a totally new type of drug for pulmonary arterial hypertension. There are already a number of existing drugs that are known as ‘vasodilators’ – which work by opening up some of the arteries that have narrowed or closed down. But these drugs are treating the symptoms of PH, rather than what’s causing the disease.

Sotatercept arose from some of the work done in genetic forms of pulmonary arterial hypertension, and the drug works by changing the expression of proteins that underly them.

So, rather than just opening up vessels, it works more directly on the underlying ‘problem proteins’ that are causing the disease in the first place. It’s the first time a PH drug is getting to the root cause of the problem, rather than treating the consequences of it.

Although it was developed by studying genetic forms of PAH, trials have shown that sotatercept can be used in non-genetic forms of PAH too. It is designed to be taken alongside other therapies, via an injection administered at home every few weeks.

Watch a video of Iain explaining more about the NICE drug review process here