Around 6,600 people in England will gain access to the drug aficamten following simultaneous MHRA authorisation and NICE approval.
The once-daily tablet, also known as MYQORZO, is for people with symptomatic obstructive hypertrophic cardiomyopathy.
Known as obstructive HCM, the condition causes the heart muscle to become thick and stiff, making it harder to pump blood around the body.
Symptoms can include exhaustion, dizziness, breathlessness, chest pain and a racing or irregular heartbeat.
Helen Knight, director of medicines evaluation at NICE, said: “We’re pleased to be able to recommend a treatment that has the potential to alter the course of obstructive hypertrophic cardiomyopathy and offers new hope to people with this serious condition.
“This treatment being approved demonstrates the value of closer working between NICE and the MHRA, ensuring that safe, effective medicines get to people earlier by aligning licencing and value assessment decisions.”
Around one in 500 people in the UK has hypertrophic cardiomyopathy, with roughly half of cases inherited.
Clinical trials found that aficamten reduced symptoms and helped people exercise more easily.
NICE said it is likely to be as effective as mavacamten, the other NICE-approved treatment for this group of patients, while potentially requiring fewer scans to find the right dose.
NICE recommended the medicine through a cost comparison process, which required its cost to be similar to or lower than that of mavacamten.
The organisation published its final guidance two weeks faster than under its standard process because of closer working with the MHRA.
NICE said it is now publishing guidance 30 per cent faster for medicines and nine per cent faster for healthtech than before April 2024.
Julian Beach, executive director of healthcare quality and access at the MHRA, said: “Today’s authorisation of aficamten, alongside NICE’s publication of final guidance, marks an important milestone for patients with obstructive hypertrophic cardiomyopathy and for the UK’s medicines regulatory system.
“This demonstrates how closer working can help patients access innovative treatments sooner, while maintaining the robust standards of safety, quality and effectiveness that patients rightly expect.
“We’re assured that the appropriate regulatory standards for the approval of this medicine have been met and, as with all medicines, we will keep its safety under close review.”
Katharine McIntosh, chief executive of Cardiomyopathy UK, said: “We’re delighted that aficamten is going to be made available to people living with obstructive hypertrophic cardiomyopathy (HCM).
“As part of the NICE appraisal, we submitted insights from our patient community, including the impact of symptoms, experiences of NHS treatment and care, and what another treatment option could mean.
“Obstructive HCM can have a considerable impact, with symptoms such as breathlessness, fatigue and a reduced ability to exercise making day-to-day life significantly more challenging.
“Mavacamten is currently the only treatment of this kind available, so having an additional option is very welcome. And because aficamten may need fewer echocardiograms in the early stages of treatment, it could represent a more accessible treatment route.
“This outcome means greater choice for patients and the opportunity for more people living with obstructive HCM to benefit from treatment, feel better and live well.”
