The World Health Organization today announced it has added the cystic fibrosis combination treatment elexacaftor + tezacaftor + ivacaftor (brand name Trikafta) to its Model Lists of Essential Medicines (EML) and Essential Medicines for Children. This is a huge win for cystic fibrosis patients, and their parents, offering the possibility that access to this critical medicine – which has been difficult due to high prices and a lack of registration in developing countries – might be expanded. This is the first time a cystic fibrosis medication has been added to the EML.
“These lists are among WHO’s most important products, used in over 150 countries to shape medicines procurement, health insurance, and reimbursement schemes,” said WHO Director General Adhanom Ghebreyesus at a press conference on 5 September 2025.
“Essential medicine status is not just symbolic – it’s a tool,” said Gayle Pledger of the Right to Breathe campaign, which has worked on access to Trikafta, in a press release. “It gives us leverage to challenge extortionate pricing, to unlock generic production, and to push governments into action.”
Cystic fibrosis is an early-onset, degenerative genetic disorder that causes mucus build-up in organs, impacting their functioning; progressive lung disease is a leading cause of death among cystic fibrosis patients. It is relatively rare, impacting some 160,000 people worldwide. In recent years, new medical breakthroughs brought hope to those suffering from the illness – allowing them longer, healthier lives.
But a list price of over $326,000 per patient per year has limited their reach; the Right to Breathe campaign estimated that only 28% of patients have access to life-saving medicines. As a result, Trikafta has been the target of several access campaigns. On 3 September 2025, civil society groups in Peru (Innovarte, Los Pacientes Importan, Acción Internacional para la Salud Peru, and Public Citizen) formally requested a compulsory licence, hoping to access generic versions of Trikafta at a lower price and improve access; compulsory licence requests were also filed in Uruguay in 2024 and in Brazil, India, South Africa, and the Ukraine in 2023, and for an earlier version of the medicine in the United Kingdom in 2019. Medicines Law & Policy tracks compulsory license requests in its TRIPS Flexibilities Database.
Other notable updates to the EML
Overall, the WHO Expert Committee on the Selection and Use of Essential Medicines reviewed 59 proposals, recommending the addition of 20 new medicines to the EML and 15 to the EML Children; it also recommended new indications and for 7 medicines already on the list. Among the new medicines added are pembrolizumab (brand name Keytruda), a treatment for lung cancer, colorectal cancer, and cervical cancer (among others), and several glucagon-like peptide-1 (GLP-1) receptor agonists (semaglutide, dulaglutide and liraglutide), which have recently gained notoriety for their impact in treating diabetes and obesity.
Absent from the list is risdiplam, a treatment for another devastating and rare childhood genetic condition, called Spinal Muscular Atrophy. A proposal by Knowledge Ecology International for the inclusion of risdiplam was made for the 2025 and 2023 lists; the Expert Committee has requested a resubmission in 2027 to allow time for more evidence gathering from peer-reviewed sources. It can be difficult to gather this type of data for rare diseases due to small, and often disperse, populations of patients. The National Institutes of Health lists the annual cost of Risdiplam as $969.86 a day, or $354,000 a year. In April of this year, generic drugmaker Natco Pharma announced it would begin making a generic version available for approximately ₹30,000 a month ($340 a month, or $4,086 a year).
The Executive Summary to the new EML also notes that the Committee made several choices to “minimise financial toxicity” in health spending. The 2021 Expert Committee also raised concerns about rising prices, and encouraged the WHO to work with the Medicines Patent Pool to facilitate lower prices for medicines already on the list and those being considered for future inclusion.
Kaitlin Mara, MSc, has been writing about international intellectual property and innovation policy for a over 15 years.