A huge turning point: CF medicine’s Trikafta/Kaftrio added to WHO Essential Medicines List

Allaa Aldaraji
8 hours ago
3 min read

Today marks a historic breakthrough in the fight for global access to lifesaving cystic fibrosis (CF) treatment.

The World Health Organization (WHO) has officially added elexacaftor/tezacaftor/ivacaftor (ETI) - better known as Trikafta/Kaftrio - to its 2025 Model List of Essential Medicines (EML).

This decision is a turning point. For the first time, a medicine specifically aimed at treating CF has been included on the EML and the world’s leading health authority has recognised that Trikafta is not a luxury drug, but an essential medicine - and that every child and adult living with CF should have the right to access it. As Dr Yukiko Nakatani, WHO Assistant Director-General for Health Systems, Access and Data, explained: “The new editions of essential medicines lists mark a significant step toward expanding access to new medicines with proven clinical benefits and with high potential for global public health impact.” This is a powerful statement from the WHO: not only is Trikafta recognised as essential, but its inclusion is about breaking down barriers to access and ensuring patients everywhere – not just in the richest countries – can benefit from its lifesaving impact.

Why this matters

The WHO Essential Medicines List is one of the most powerful tools in global health. Inclusion can drive down prices through global negotiations, enable generic production and local manufacturing, and guide governments to prioritise sustainable access in their health systems. For CF, this decision is nothing short of transformative.

“Essential medicine status gives us leverage to challenge extortionate pricing and push governments into action,” said Gayle Pledger, Senior Organiser with Right to Breathe.

Dr Andrew Hill, University of Liverpool, added: “This decision recognises ETI is not a luxury – it’s an essential medicine capable of transforming lives.”

*Gayle Pledger & Ajsela Kushova at the WHO EML Expert Committee meeting in May*

A collective effort

This milestone is the result of a collective global effort. A comprehensive 98-page application, built on months of research and testimony, was submitted to the WHO with the backing of 35 cystic fibrosis organisations and more than 50 human rights groups. This milestone has been made possible by an amazing team of doctors, researchers, and activists: Dr Andrew Hill, Grace Hennessy, Dr Cassandra Fairhead, Ollie James, Ambar Noor Qavi, Lizzie O’Brien, Dr Andrea Perez Navarro, Gayle Pledger, Benedict Young and Dr Jonathan Guo.

At the WHO Expert Committee meeting in Geneva this May, CF parents and advocates – including Gayle Pledger and Ajsela Kushova – testified about the reality families face: children being priced out of survival.

“When the committee chair closed the meeting by recognising the importance of the EML for rare diseases, it felt like a crack in the wall of injustice we’ve been pushing against,” – Ajsela.

*Ajesela’s son, Flori, who has cystic fibrosis*

Families on the frontlines

Behind this win are families who have fought tirelessly for their children.

Ajsela, from Albania, had to move her son Flori to Italy for treatment. “Since starting ETI, my son can go to school and live like other children,” she said. “But not every family can move countries to save their child.”

Carmen Leitch, from South Africa, told us: “This listing proves Trikafta is essential, not optional. Vertex must act immediately to ensure long-term, sustainable access for all families.”

In India, Tanya Kumar called out Vertex’s so-called donation scheme: “It helps only a handful of families and offers no long-term security. Access to this essential medicine cannot be left to the whims of a profit-hungry company.”

What's next?

This decision is a major step forward, but the fight is not over.

We call on governments to urgently secure sustainable access to generic versions of Trikafta, and to use every tool available – including compulsory licensing – to break Vertex’s monopoly. At the same time, we demand that Vertex drop its prices and stop enforcing patents in countries where patients are being locked out of treatment.

As Grace Hennessy, one of the academics behind the application, said: “This is a monumental step towards equitable access for patients worldwide.” For other parents that still do not have access to this lifesaving treatment, the battle to save their children's lives continues…