South Africa Sees Breakthrough in Affordable Cystic Fibrosis Treatment
A new generic version of cystic fibrosis treatment is set to become available for the first time, thanks to years of campaigning by patients and their families. The life-saving medicine could change the outlook for millions worldwide.
For 7-year-old Grant Leitch, asking his mother if his little brother Brett would die from cystic fibrosis was a tough question to ask, but one that he had to pose. Cystic fibrosis is a genetic condition that causes build-up of mucus in lungs and digestive system, making it hard to breathe and leaving patients susceptible to infections.
Brett's situation is dire, as the treatment is currently priced out of reach for many families worldwide. However, with Vertex Pharmaceuticals now selling its revolutionary treatment sold by pharmaceutical company Vertex for $370,000 a year, will be available for as little as $2,000 a year from a generic manufacturer. The price reduction is a result of years of parent-led campaigning.
Cystic fibrosis affects an estimated 190,000 people worldwide and only one in four has received modulator drugs, which allow a normal life expectancy. Researchers have highlighted profound global disparities in diagnosis and treatment access despite the World Health Organization classifying CFTR modulators as 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.
In the UK, parent Gayle Pledger had "never even thought about engaging with my MP" but was struck by the unfairness of a treatment that could help her daughter being out of reach because of cost. Now she is a full-time campaigner with Just Treatment.
Pledger's efforts have contributed to a global movement with an effort to get generic drugs into families' hands, dubbed the CF buyers club 2.0. The UK campaign has grown from the initial success in getting a cut-price generic version made in Argentina for Orkambi.
Bangladeshi company Beximco will now make Triko, a generic ETI, available from spring 2026. This could save families thousands of dollars per year.
South African doctors have tested this approach and found it effective. Leitch is one of the parents who have been campaigning for more affordable treatment options.
A new generic version of cystic fibrosis treatment is set to become available for the first time, thanks to years of campaigning by patients and their families. The life-saving medicine could change the outlook for millions worldwide.
For 7-year-old Grant Leitch, asking his mother if his little brother Brett would die from cystic fibrosis was a tough question to ask, but one that he had to pose. Cystic fibrosis is a genetic condition that causes build-up of mucus in lungs and digestive system, making it hard to breathe and leaving patients susceptible to infections.
Brett's situation is dire, as the treatment is currently priced out of reach for many families worldwide. However, with Vertex Pharmaceuticals now selling its revolutionary treatment sold by pharmaceutical company Vertex for $370,000 a year, will be available for as little as $2,000 a year from a generic manufacturer. The price reduction is a result of years of parent-led campaigning.
Cystic fibrosis affects an estimated 190,000 people worldwide and only one in four has received modulator drugs, which allow a normal life expectancy. Researchers have highlighted profound global disparities in diagnosis and treatment access despite the World Health Organization classifying CFTR modulators as 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.
In the UK, parent Gayle Pledger had "never even thought about engaging with my MP" but was struck by the unfairness of a treatment that could help her daughter being out of reach because of cost. Now she is a full-time campaigner with Just Treatment.
Pledger's efforts have contributed to a global movement with an effort to get generic drugs into families' hands, dubbed the CF buyers club 2.0. The UK campaign has grown from the initial success in getting a cut-price generic version made in Argentina for Orkambi.
Bangladeshi company Beximco will now make Triko, a generic ETI, available from spring 2026. This could save families thousands of dollars per year.
South African doctors have tested this approach and found it effective. Leitch is one of the parents who have been campaigning for more affordable treatment options.