Seven-year-old Grant Leitch had an important question for his mother. He asked if his little brother, Brett, who has cystic fibrosis (CF), was going to die.
The South African family, like tens of thousands around the world, have been priced out of access to modern cystic fibrosis therapies, and if Grant had asked at the start of 2025, he might have received a less optimistic answer.
But as the new year begins Carmen Leitch has fresh hope to offer her sons. A “revolutionary” treatment sold by pharmaceutical company Vertex for $370,000 (£274,000) a year will be available for as little as $2,000 a year from a generic manufacturer.
The 99.5% reduction in price is the result of years of parent-led campaigning.
Cystic fibrosis, a genetic condition, causes a buildup of mucus in the lungs and digestive system, making it hard to breathe and leaving patients susceptible to infections. Until recently, most sufferers would die as young adults.
Cystic fibrosis patient Lara, 10, at the Zeepreventorium centre in Belgium which specialises in treating young people with the condition. Her day includes treatment, aerosol drugs and a physiotherapy session. Photograph: BSIP/Universal Images Group/Getty Images
New drugs, called CFTR modulators, have become available over the past decade, allowing a normal life expectancy.
But just one in four of the estimated 190,000 people worldwide with cystic fibrosis has received modulator drugs, in a combination known as ETI (elexacaftor/tezacaftor/ivacaftor). Researchers have highlighted “profound global disparities in diagnosis and treatment access”, despite ETI being classed by the World Health Organization as an essential medicine.
Vertex Pharmaceuticals holds the patent for ETI, sold as Trikafta or Kaftrio. The company has faced criticism for its prices, and for failing to make the drug available in many countries, even where it has registered patents.
We’ve watched children suffer and die while a treatment sat on the shelf, priced out of reachGayle Pledger, CF campaigner
A spokesperson says Vertex’s CF medicines are available in more than 60 countries, and it continues to expand “reliable and safe access” including through a pilot donation programme that encompasses 14 countries. The spokesperson adds: “This progress is the result of Vertex’s decades of work and substantial investment in research and development. Companies that commercialise a generic version of Trikafta have not had to bear the notable cost of drug discovery and development.”
In the UK in 2019, Vertex and NHS England were locked in drawn-out and bad-tempered negotiations for a forerunner to ETI called Orkambi. Cystic fibrosis patients’ families grouped together to obtain a cut-price generic version made in Argentina. They called themselves the CF buyers’ club, a reference to mechanisms used by US Aids patients in the early years of HIV and portrayed in the film the Dallas Buyers Club.
The new, global effort to get generic drugs into families’ hands has grown out of that initial UK campaign, and is being called the CF buyers’ club 2.0.
Campaigners protest over the lack of availability of the cystic fibrosis drug Orkambi in London in 2017. Now, CFTR modulators are offering new hope to CF patients. Photograph: Facundo Arrizabalaga/EPA
The Argentinian route, later also used to access ETI, all but closed after rampant inflation increased prices of the drug, which – at about $60,000 a year – remained mostly unaffordable.
In a conference room in Seattle last month, UK parent Gayle Pledger announced that Bangladeshi company Beximco would make Triko, a generic ETI, available from spring 2026.
Triko will cost $12,750 for adults annually, and $6,375 for children, meaning 58 children treated for the cost of one with Vertex’s drug.
In 2017, Pledger had “never even thought about engaging with my MP”. But she was struck by the profound unfairness of a drug that could help her daughter, and others like her, being out of reach because of cost. Now she is a full-time campaigner with Just Treatment.
Campaigner Gayle Pledger, whose daughter has cystic fibrosis. Photograph: Allaa Aldaraji/Courtesy of CF buyers club
“We’ve watched children suffer and die while a treatment sat on the shelf, priced out of reach,” she says. “It has gone from the UK to this global thing. But it’s still the same thing.”
Just Treatment worked with campaign group Third World Network to approach Beximco three years ago about creating a generic ETI.
“[There have been] a number of calls where I’ve come off it in tears thinking, ‘this is actually going to happen’ and what that would mean to the community,” she says.
Almost 80,000 people are thought to be living with undiagnosed cystic fibrosis, and 82% of those are in low- and middle-income countries. Pledger hopes that an affordable treatment will help find them. “When there’s nothing available, what is the point in diagnosing them?”
As individuals, Pledger says, it is easy to feel powerless. But when families come together “it’s an incredible power, especially mums. It’s our drive to look after our children […] you give them the right tools, the legal tools, to fight – and we’ve seen again and again, it’s just the most incredible force.”
Now the fight is to get the drug to patients – either by self-funding through the buyers’ club or by lobbying governments to take up the affordable option.
Bangladesh, as a “least developed country”, is excluded from some international intellectual property laws, paving the way for Beximco to produce and export a generic version. Most countries allow some importation of medicines for personal use.
A month’s supply of Trikafta, made by Vertex. The list price of the ‘revolutionary’ cystic fibrosis treatment is $370,000 (£274,000) a year for each patient. Photograph: Michael Dwyer/Alamy
The annual cost of Triko could fall to $2,000 using a reduced dosing protocol, developed by South African doctor Marco Zampoli.
Making treatment affordable felt like “a moral obligation”, he says, pointing to how families had sold their homes to buy the Argentinian drug.
Trikafta is now available in South Africa for patients with the right insurance. But for those without, the wait continues.
In the drawer of a filing cabinet behind his desk, Zampoli has doses of the drug, donated by CF patients who had a surplus, to give to “the really sick ones who most need it” – using his dose sparing strategy.
“I have about 10 patients, kids, on this dose, and they do great. So where there’s no regulatory or legal restriction on doing it, I think it’s an effective strategy that people should use.”
The cystic fibrosis medication Trikafta, now available in South Africa for patients with the right insurance. Photograph: Madelene Cronjé/The Guardian
Alongside the “CF mums” in Seattle was Leitch. At previous events campaigning for modulator drug access, banners picturing children who had died had been noticed by her eldest.
“He said to me, ‘Mom, is Brettie going to die like all those other little kids?’
“I’m not going to lie: me knowing that there was a generic that was going to be launched really did shape that answer in a very different way than what it would have done, had I not known.”
A family member in Britain is on modulator therapy. It feels, Leitch says, “inhumane” that it is unaffordable for Brett in Africa.
Brett, an energetic five-year-old, dreams of becoming a monster truck driver. The sooner he begins ETI, the greater his chances of avoiding irreversible lung damage.
They will “absolutely” be buying the Beximco product when it becomes available in spring, Leitch says. “We were sitting there with our calculator, working out the exchange rate, and we were like – ‘We can afford that. We could afford that!’– it’s a celebration from start to finish.”
