UK approves new drug aficamten for thousands of patients
A major breakthrough has been announced in the United Kingdom for thousands of people living with obstructive hypertrophic cardiomyopathy (HCM), a serious heart condition that can significantly affect quality of life. The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) and the National Institute for Health and Care Excellence (NICE) have simultaneously approved and recommended a new treatment, Aficamten, marketed under the brand name MYQORZO.
The decision marks an important milestone in the treatment of obstructive HCM and is expected to benefit around 6,600 patients in England. The coordinated approval process between MHRA and NICE enabled the medicine to become available more quickly than usual, ensuring that eligible patients gain faster access to this innovative therapy.
Aficamten is a once-daily oral tablet designed to treat obstructive hypertrophic cardiomyopathy by reducing the excessive contraction of the heart muscle. In people with HCM, the heart muscle becomes abnormally thick, making it harder for the heart to pump blood effectively. This can lead to symptoms such as shortness of breath, chest pain, fatigue, dizziness, fainting, and irregular heart rhythms. By limiting the heart muscle’s excessive contraction, Aficamten improves blood flow from the heart, enhances overall cardiac function, and helps relieve these debilitating symptoms.
Clinical trials have shown that Aficamten significantly improves patients’ ability to perform physical activities and reduces many of the common symptoms associated with the disease. Participants experienced better exercise capacity and reported less breathlessness, fatigue, chest discomfort, dizziness, and episodes of abnormal heartbeat. These findings indicate that the medication can substantially improve the quality of life for patients living with obstructive HCM.
According to medical experts, the effectiveness of Aficamten is comparable to Mavacamten, another approved treatment for the condition. However, one potential advantage of Aficamten is that patients may require fewer echocardiograms, or heart ultrasound scans, during treatment. This could reduce the need for frequent hospital visits and ongoing monitoring, making treatment more convenient for both patients and healthcare providers.
NICE stated that Aficamten was recommended after a comprehensive cost-effectiveness assessment showed that it represents good value for the National Health Service (NHS). The organization also highlighted that close collaboration with the MHRA helped accelerate the review and approval process without compromising safety or effectiveness.
Helen Knight, Director of Medicines Evaluation at NICE, described the approval as an important advancement in the treatment of this complex heart disease. She said the new medicine offers fresh hope to patients and expands the range of available treatment options.
Julian Beach, Executive Director at the MHRA, said the joint effort between the two organizations demonstrates the importance of collaboration in ensuring that safe and effective medicines reach patients as quickly as possible.
The decision has also been welcomed by Cardiomyopathy UK. Chief Executive Katharine McIntosh said the availability of Aficamten would provide patients with more effective treatment options and help improve their overall quality of life.
According to NHS England, the new medicine will be available to eligible patients within 30 days of NICE issuing its final guidance. The pharmaceutical company has also agreed to provide the medicine to the NHS at a discounted price, making the treatment more affordable and accessible. The approval represents a significant step forward in the management of obstructive hypertrophic cardiomyopathy and offers renewed hope for thousands of patients across England.