A new NHS-approved pill could eliminate symptoms of cystic fibrosis—if regulators approve it in time

A groundbreaking new drug that could dramatically improve the lives of thousands of cystic fibrosis (CF) patients in the UK has been approved by the Medicines and Healthcare products Regulatory Agency (MHRA). If given final approval by NHS regulators, the pill—called Alyftrek—could be available by August.

CF is a life-threatening genetic disorder that affects over 11,000 people in the UK. It clogs the lungs and digestive system with thick, sticky mucus, making breathing and digestion increasingly difficult. Over time, the damage to the lungs can become so severe that they fail completely.

Alyftrek: A Game-Changer for CF Sufferers

Alyftrek, a triple-combination drug, works by correcting a faulty protein known as CFTR. This faulty protein disrupts the balance of water and salt in the body, leading to mucus buildup. By fixing this process, the pill effectively reduces symptoms, helping patients breathe more easily and absorb nutrients properly.

Clinical trials have shown staggering results. Patients who took just one pill a day saw their symptoms vanish in vital tests. More strikingly, their sweat chloride levels—used to diagnose CF—returned to those of healthy individuals, suggesting the drug corrects the underlying genetic issue.

Regulatory Hurdles Remain

Despite its approval by the MHRA, Alyftrek is not yet guaranteed to be available on the NHS. The National Institute for Health and Care Excellence (NICE) is currently assessing whether the drug is cost-effective. If approved, it could be rolled out to thousands of eligible CF patients by late summer.

David Ramsden, Chief Executive of the Cystic Fibrosis Trust, welcomed the news but urged regulators to act swiftly. “Today’s MHRA approval is another important step in making sure as many people with CF as possible can benefit from the best available treatments,” he said. “We now hope that NICE will move quickly to complete its assessment to enable NHS access.”

A Lifeline, But Not a Cure

While Alyftrek offers hope, it is not a cure for CF. It only works for those with the most common genetic mutation of the disease, leaving some patients without access to its benefits. Ramsden emphasised that while the drug is a major breakthrough, work must continue to ensure all CF patients receive effective treatment.

For thousands of sufferers, Alyftrek could mean the difference between struggling for every breath and leading a near-normal life. Now, all eyes are on NICE to see whether this ‘miracle pill’ will be made available to those who need it most.