Cystic Fibrosis Breakthrough Offers New Hope for Families

Seven-year-old Grant Leitch recently asked a heart-wrenching question to his mother: would his little brother Brett, who has cystic fibrosis, die? This query reflects a reality faced by families worldwide, including many in South Africa, who struggle to afford modern therapies for this condition.

However, as 2025 approaches, Carmen Leitch feels renewed optimism. A new treatment from Vertex Pharmaceuticals, previously priced at $370,000 a year, will soon be accessible for only about $2,000 annually through a generic manufacturer. This drastic price drop of 99.5% comes after years of advocacy from parents like Carmen.

Cystic fibrosis is a genetic disorder that leads to mucus accumulation in the lungs and digestive system, complicating breathing and making patients more vulnerable to infections. Historically, most individuals with CF did not live past early adulthood.

Recent advancements in CFTR modulators have enabled a significantly improved life expectancy for patients. Yet, strikingly, only one in four of the 190,000 individuals diagnosed globally has access to these crucial medications, known as ETI. Researchers have pointed out significant disparities in access and diagnosis, even though ETI has been designated an essential medicine by the World Health Organization.

Vertex holds the patent for ETI, which is sold under the names Trikafta or Kaftrio. The company has faced scrutiny over its pricing practices and its limited availability in various regions, including places where it has established patents.

I’ve seen children suffer and die while this treatment was unavailable due to its cost.

In response to criticism, Vertex claims its medications are accessible in over 60 countries and that they’re working to expand availability through a pilot donation program covering 14 nations. A spokesperson emphasized that their successful access expansion efforts stem from decades of investment in research and development, arguing that companies marketing generic versions did not invest similarly.

In the UK, Vertex and NHS England were previously embroiled in contentious negotiations regarding another CF medication called Orkambi. Families of CF patients banded together to secure a cut-price generic version from Argentina, forming what they called the CF buyers’ club, reminiscent of early AIDS activism depicted in the film “Dallas Buyers Club.”

This collective movement has since evolved into a broader initiative now dubbed CF buyers’ club 2.0, aimed at making generics more accessible globally. In a recent announcement, parent Gayle Pledger revealed that a Bangladeshi company would begin offering a generic ETI named Triko in spring 2026, at prices dramatically lower than Vertex’s offerings.

Triko’s annual price is set to be $12,750 for adults and $6,375 for children, meaning treatment for nearly six children could be afforded for the cost of one Trikafta treatment. Pledger, who became an activist after starting to fight for her daughter’s access to treatments, describes the transition from feeling powerless to witnessing the collective strength of families advocating for change.

With many individuals living with undiagnosed cystic fibrosis, the hope is that affordable medications will help identify more patients. Pledger notes that empowered families form an incredible force, especially mothers, driven to secure better futures for their children.

The ongoing challenge is ensuring patients get the medication, primarily through self-funding or government advocacy for affordable options. As a least developed country, Bangladesh can bypass many of the international patent laws that restrict generic manufacturing, further enabling access.

Furthermore, there are ways to reduce costs. For instance, doctors in South Africa are exploring a reduced dosing strategy for Triko, lowering the potential yearly cost to around $2,000.

Dr. Marco Zampoli, who has been involved in implementing this approach, describes it as a moral obligation, particularly after witnessing families struggling to afford medication. Although some patients now have access to Trikafta with insurance, many still remain in limbo.

With a donation of leftover doses, Zampoli aims to help the most severely affected children, utilizing an innovative dose-spreading strategy to maximize the impact of available medication.

At a recent gathering in Seattle, among many advocates was Carmen Leitch, who recalled her eldest son’s poignant question about his brother’s fate. Knowing there’s now a generic option changes her response to such inquiries, as it shapes a narrative of hope.

For Brett, an energetic five-year-old with dreams of being a monster truck driver, beginning ETI treatment could mean avoiding permanent lung damage. Leitch is determined to buy the Beximco product once it’s released and expressed excitement over the newfound affordability, saying, “We can afford that!” It’s a hopeful moment for their family.