Posted on January 2nd 2025
The new PAH treatment sotatercept has now been approved for use in the UK by the Medicines and Healthcare products Regulatory Agency (MHRA). The announcement came just before New Year, and it’s important to remember that the approval does not mean it can now be prescribed.
The therapy is yet to be commissioned, and the next stage will involve the National Institute for Clinical Excellence (NICE) putting it 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 PAH. It is this involvement that we hope will persuade them that the benefits will be worth the cost.
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 every few weeks.
