We were sitting with our calculator saying we can afford that!' Joy for families as cystic fibrosis drug prices fall within reach

"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."

"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."

A South African family faces life-or-death uncertainty for a child with cystic fibrosis due to unaffordable modern therapies. A revolutionary ETI treatment previously sold by Vertex for $370,000 a year will become available from a generic manufacturer for about $2,000 annually, reflecting a 99.5% price reduction driven by sustained parent-led campaigning. CFTR modulators now enable a normal life expectancy, yet only about one in four of an estimated 190,000 people worldwide has received ETI. Global disparities in diagnosis and treatment access persist despite ETI being listed as an essential medicine by WHO, and Vertex faces criticism over pricing and availability.

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