FDA approves first ‘breakthrough’ drug that could treat up to 90% of cystic fibrosis patients

The US Food and Drug Administration (FDA) announced that it has approved the first drug combination therapy to treat cystic fibrosis. 

Trikafta, created by Boston-based Vertex Pharmaceuticals Inc, combines three medications into one pill.

It has been approved for patients 12 years and older who have at least one F508del mutation, the most common genetic mutation of the damaging lung disease.

Doctors and health professionals say this means the new drug could treat up to 90 percent of cystic fibrosis patients, meaning roughly 27,000 people.

‘In the past few years, we have seen remarkable breakthroughs in therapies to treat cystic fibrosis and improve patients’ quality of life, yet many subgroups of cystic fibrosis patients did not have approved treatment options,’ said acting FDA commissioner Ned Sharpless in a statement. 

‘That’s why we used all available programs … to help advance today’s approval in the most efficient manner possible, while also adhering to our high standards.’ 

The FDA has approved Trikafta, made by Vertex (pictured), which has been approved for patients 12 years and older who have the most common genetic mutation of cystic fibrosis

According to the Securities and Exchange Commission, the drug will priced at $311,503 per year, or $23,896 for a 28-day pack.  

But a Vertex representative told ABC News that it is expected that the cost for Trikafta will be widely reimbursed by private insurers and Medicaid/Medicare’.

Cystic fibrosis is caused by mutations in the CFTR gene, which creates a defective protein. 

Normally the protein functions as a channel, controlling the movement of water in tissues, which is necessary for mucus to be thin and flow easily. 

The most common mutation is delta F508, which is a deletion of one amino acid at position 508 in the CFTR protein.

This impairs the movement of water into and out of cells, which causes thick and sticky mucus to be produced  

But Trikafta helps the protein created by the gene mutation function more effectively.  

In clinical trials, Trikafta was found to improve lung function by approximately 14 percent compared to a currently available drug, according to Reuters.

Another clinical trial found that the drug increased sweat production and body mass index in patients compared to a placebo. 

Some side effects included headaches, abdominal pain, upper respiratory tract infections and diarrhea. 

‘Today marks a tremendous breakthrough and exciting news for people with cystic fibrosis,’ said Dr Preston Campbell III, president and CEO of the Cystic Fibrosis Foundation in a statement.

‘This milestone is the result of an extraordinary community working together against great odds, and we are overjoyed that this will mean more people will have effective treatments for their disease.’ 

Cystic fibrosis causes a build-up of mucus in the airwaves and makes it increasingly difficult to breathe over time.

Bacteria can become trapped, which can cause the lungs to become damaged or infected, and in some cases send the sufferer into respiratory failure, according to the Cystic Fibrosis Foundation.

Symptoms include persistent coughing, frequent lung infections, shortness of breath and inflamed nasal passages.

The Cystic Fibrosis Foundation estimates that more than 30,000 people have the condition in the US and that 1,000 new cases are diagnosed every year.

The median age of survival is currently 33.4 years, with half of patients living into their fifties or sixties.   

Read more at DailyMail.co.uk