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.<\/p>\n
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.<\/p>\n
But as the new year begins Carmen Leitch has fresh hope to offer her sons. A \u201crevolutionary\u201d treatment sold by pharmaceutical company Vertex for $370,000 (\u00a3274,000) a year will be available for as little as $2,000 a year from a generic manufacturer.<\/p>\n
The 99.5% reduction in price is the result of years of parent-led campaigning.<\/p>\n
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.<\/p>\n
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<\/p>\n
New drugs, called CFTR modulators, have become available over the past decade, allowing a normal life expectancy.<\/p>\n
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 \u201cprofound global disparities in diagnosis and treatment access<\/a>\u201d, despite ETI being classed by the World Health Organization as an essential medicine<\/a>.<\/p>\n Vertex Pharmaceuticals holds the patent for ETI, sold as Trikafta or Kaftrio. The company has faced criticism<\/a> for its prices, and for failing to make the drug available<\/a> in many countries, even where it has registered patents<\/a>.<\/p>\n We\u2019ve watched children suffer and die while a treatment sat on the shelf, priced out of reach<\/p><\/blockquote>\n Gayle Pledger, CF campaigner<\/p>\n A spokesperson says Vertex\u2019s CF medicines are available in more than 60 countries, and it continues to expand \u201creliable and safe access\u201d including through a pilot donation programme that encompasses 14 countries. The spokesperson adds: \u201cThis progress is the result of Vertex\u2019s 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.\u201d<\/p>\n In the UK in 2019, Vertex and N<\/a>HS England<\/a> were locked in drawn-out and bad-tempered negotiations for a forerunner to ETI called Orkambi. Cystic fibrosis patients\u2019 families grouped together to obtain a cut-price generic version made in Argentina<\/a>. They called themselves the CF buyers\u2019 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<\/a>.<\/p>\n The new, global effort to get generic drugs into families\u2019 hands has grown out of that initial UK campaign, and is being called the CF buyers\u2019 club 2.0.<\/p>\n 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<\/p>\n The Argentinian route, later also used to access ETI, all but closed after rampant inflation increased prices of the drug, which \u2013 at about $60,000 a year<\/a> \u2013 remained mostly unaffordable.<\/p>\n 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.<\/p>\n Triko will cost $12,750 for adults annually, and $6,375 for children, meaning 58 children treated for the cost of one with Vertex\u2019s drug.<\/p>\n In 2017, Pledger had \u201cnever even thought about engaging with my MP\u201d. 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<\/a>.<\/p>\n Campaigner Gayle Pledger, whose daughter has cystic fibrosis. Photograph: Allaa Aldaraji\/Courtesy of CF buyers club<\/p>\n \u201cWe\u2019ve watched children suffer and die while a treatment sat on the shelf, priced out of reach,\u201d she says. \u201cIt has gone from the UK to this global thing. But it\u2019s still the same thing.\u201d<\/p>\n Just Treatment worked with campaign group Third World Network<\/a> to approach Beximco three years ago about creating a generic ETI.<\/p>\n \u201c[There have been] a number of calls where I\u2019ve come off it in tears thinking, \u2018this is actually going to happen\u2019 and what that would mean to the community,\u201d she says.<\/p>\n 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. \u201cWhen there\u2019s nothing available, what is the point in diagnosing them?\u201d<\/p>\n As individuals, Pledger says, it is easy to feel powerless. But when families come together \u201cit\u2019s an incredible power, especially mums. It\u2019s our drive to look after our children [\u2026] you give them the right tools, the legal tools, to fight \u2013 and we\u2019ve seen again and again, it\u2019s just the most incredible force.\u201d<\/p>\n Now the fight is to get the drug to patients \u2013 either by self-funding through the buyers\u2019 club or by lobbying governments to take up the affordable option.<\/p>\n Bangladesh, as a \u201cleast developed country\u201d, 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.<\/p>\n A month\u2019s supply of Trikafta, made by Vertex. The list price of the \u2018revolutionary\u2019 cystic fibrosis treatment is $370,000 (\u00a3274,000) a year for each patient. Photograph: Michael Dwyer\/Alamy<\/p>\n The annual cost of Triko could fall to $2,000 using a reduced dosing protocol<\/a>, developed by South African doctor Marco Zampoli.<\/p>\n