A MIRACLE pill that makes many signs of cystic fibrosis disappear has been approved in a boost for thousands of UK sufferers.
The genetic condition affects over 11,000 people in the UK, by slowly clogging their lungs with a thick mucus.
The NHS miracle pill makes signs of the genetic disease vanish[/caption]
Over the years, the mucus causes so much damage that the person’s lungs can stop working properly.
The drug, Alyftrek, works by regulating the flow of water and salt in and out of organs in CF patients, improving their overall function.
It helps fix a faulty protein (CFTR) in their cells, which reduces thick mucus buildup in the lungs and digestive system.
This makes it easier for patients to breathe and digest food.
In trials with just one tablet a day CF sufferers no longer exhibit symptoms in vital tests.
Medicine regulatory bosses at the MHRA approved the triple combination medicine for those with the most common CF causing mutation.
The National Institute for Health and Care Excellence are now studying the cost-effectivness of the pill and if it is backed it could be available on the NHS by late August.
David Ramsden, Cystic Fibrosis Trust Chief Executive, said: “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.
“We now hope that NICE will move quickly to complete its assessment of the medicine to enable it to be prescribed on the NHS.
“Today is good news, but we never forget that these medicines are not a cure, and do not work for some people.
“Cystic Fibrosis Trust will not stop until everyone with CF can live a life that’s not limited by their condition.”
CF causes thick, sticky mucus in the lungs and digestive system because it disrupts the transport of chloride (a type of salt) in and out of cells.
Because of this, people with CF also have higher levels of salt in their sweat.
A sweat chloride test is used to diagnose CF by measuring the salt in sweat.
Phase 3 trials showed that Alyftrek helps people reach “carrier levels of sweat chloride,” meaning it fixes the gene problem and makes their sweat levels normal, like someone without CF.
What are the symptoms of cystic fibrosis?
While individuals can have different symptoms, some of the main indicators as listed by the NHS can be:
- Recurring chest infections
- Difficulty putting on weight
- Frequent, wet-sounding coughs
- Diarrhoea
- Occasional wheezing and shortness of breath
Those diagnosed with the disease are also at risk of other conditions, including diabetes, osteoporosis and liver problems.
