Elexacaftor/Ivacaftor/Tezacaftor

Trikafta still possesses numerous patent protections around the world, but the buyers club has plotted a workaround by tapping Bangladeshi manufacturer Beximco to produce the generic. To topple access barriers to Vertex Pharmaceuticals’ potentially lifesaving, yet often out-of-reach cystic fibrosis therapy Trikafta, a buyers club has resurfaced with a plan to introduce a discounted generic of the drug in certain parts of the world.Under the strategy, the new generic of Trikafta—which combines the CFTR modulator drugs ivacaftor, tezacaftor and elexacaftor—will cost $12,750 a year for adults and $6,375 annually for kids, well below the $370,000 list price the medicine carries in the U.S., the buyers club said in a Thursday press release.Trikafta, which generated full-year sales of $11.02 billion last year, still possesses numerous patent protections around the world. But the buyers club believes it has plotted a workaround by tapping Bangladeshi manufacturer Beximco to produce a generic option.Given its classification as a least developed country (LDC), Bangladesh is not beholden to pharmaceutical patents under the World Trade Organization agreement on trade-related aspects of intellectual property rights (TRIPS). That means Beximco can legally manufacture and export generics of patented drugs, the buyers group explained.As for the potential reach of the Trikafta generic, many countries have legal exemptions in place that allow people to purchase and import medicines for personal use, the group explained, noting that patients, families, doctors and health service employees can now “register their interest” in the cheaper copycat on the buyers club website.The buyers club effort looks poised to predominantly benefit patients in low- and middle-income countries (LMICs) where Trikafta is either cost-prohibitive or unavailable outright. Patients in the U.S., however, are less likely to benefit, given that it is often illegal for people to import drugs into the country for personal use if they haven’t been approved by the FDA.On the other hand, certain countries like the U.K.—where at least one of the organizations behind the buyer's club is based—do allow patients to import certain medicines for personal use, under strict conditions. The generic will carry the name Triko and become available for purchase starting in the spring of 2026, according to the buyers club, which was borne out of the efforts of multiple advocacy and cystic fibrosis (CF) patient groups.Vertex’s pricing and patenting strategy around Trikafta and predecessor CF meds has long been controversial, and the buyers club did not hold back in calling out the company for creating “deep inequality in access to the treatment.” Cystic fibrosis, a progressive, genetic disease of the lungs, pancreas and other organs, is thought to affect more than 188,000 people around the world, the buyers club pointed out, citing a 2024 paper critical of current Trikafta access levels. Among that group, some 60% are diagnosed and just 27% are on treatment for their disease, according to the organization. The Cystic Fibrosis Foundation estimates that around 105,000 people have been diagnosed with CF across 94 countries.Treatment disparities appear rife when looking at LMICs, where researchers last year estimated that around 82% of undiagnosed CF patients live. Meanwhile, just one LMIC reported patient reimbursement for Trikafta, versus 35 high-income countries at the time the paper was written.Vertex, for its part, did not directly respond to Fierce Pharma’s request for comment on the purchasing group’s announcement. Instead, a spokesperson pointed to a “CF facts and figures” page on the company’s website, where Vertex asserts that it is “committed to develop and provide access to our CF medicines to as many eligible people living with CF who can potentially benefit from them.”Vertex contends that its approved modulators are suitable for around 90% of people living with CF and states that its medicines are currently treating more than 75,000 people with CF globally, or about two-thirds of the disease’s diagnosed patient population.The company’s CF drugs are available in over 60 countries, more than 50 of which have secured “broad access agreements,” Vertex notes. Additionally, the company has rolled out a pilot donation program in tandem with the group Direct Relief to provide Trikafta to eligible patients in 14 countries, including Ukraine, Nepal, India, Egypt, Ivory Coast, Sri Lanka and El Salvador.But for the buyers club and advocacy groups behind its access push, those efforts are not enough.“We’ve watched children suffer and die while a treatment sat on the shelf, priced out of reach,” Gayle Pledger, part of organizations Just Treatment and Right to Breathe, said in a statement.“Today, that changes,” she continued. “[W]e’ve proven that patient power can change what billion-dollar corporations refuse to. Governments must act fast to take all the necessary steps to make this lifesaving drug available for every eligible patient.”This is hardly the first time Vertex has taken flak over the availability of its CF meds, and the company had a previous run-in with the buyers club in 2019 over an earlier combo therapy, according to Stat News.In that instance, the group elected to work with Argentinian drugmaker Gador to produce a cheaper generic of Vertex’s Trikafta predecessor Orkambi, which weds the cystic fibrosis transmembrane conductance regulator (CFTR) potentiator ivacaftor with the CFTR corrector lumacaftor. Meanwhile, a paper published in The Journal of Cystic Fibrosis in 2022 argued that while Vertex’s arsenal of CFTR modulator drugs has the “unparalleled opportunity to increase quality and length of life for almost all CF patients,” the therapeutics are “so expensive they are essentially unavailable unless reimbursed by the government or health system authorities.”That paper, which had the same lead author who penned the studies cited in the buyers club press release, estimated that just 12% of some 162,000 people thought to have CF worldwide were receiving Trikafta.The company's access agenda again came under fire in 2023, when a coalition of CF patients and families, helmed by Vertex Save US and the U.K.’s Just Treatment, petitioned governments in South Africa, Brazil, India and Ukraine to either revoke or suspend Trikafta patents. Outside the U.S., Trikafta goes by the name Kaftrio. 

iStock, Afry Harvy Despite showing no meaningful benefit on lung function, Arcturus’ mRNA therapy ARCT-032 reduced mucus volume in patients with cystic fibrosis—an outcome William Blair found “potentially promising.” Arcturus Therapeutics’ inhalable mRNA therapeutic ARCT-032 failed to induce meaningful lung function improvements in a Phase II cystic fibrosis trial, prompting the biotech to dig into exploratory outcomes to search for signals of efficacy. By the end of trading Wednesday, shares of Arcturus were down to $11.54 apiece, down 50% from its previous closing price of $23.16. Despite the strong negative market reaction, analysts at William Blair pointed to “some potentially promising efficacy signals.” In its news release, Arcturus touted results from AI-enhanced high-resolution computed tomography screening, demonstrating what the company said is an “encouraging” reduction of mucus, which “is a meaningful trend indicative” of the asset’s therapeutic benefit. William Blair appeared to agree on this point, writing on Wednesday that this mucus outcome “offers potential for clinical benefit at [a] high dose.” Still, the firm maintained that the mucus data are “also slightly mixed,” with only four of six evaluated patients showing reductions in mucus volume and in the number of mucus plugs. The analysts added that Arcturus will need to establish whether or not “longer treatment duration or a higher dose of ARCT-032 can boost efficacy signals beyond mucus clearance.” This phase of the trial tested six patients with cystic fibrosis receiving 10 mg of ARCT-032 over 28 days. The company is preparing a longer-duration trial “to substantiate the clinical relevance” of the mucus results, it stated in Wednesday’s release. Arcturus is also looking to enroll six more patients who will receive a higher dose of ARCT-032, findings from which will guide dosing, duration and endpoints for future studies. The company is also gearing up for a 12-week safety and preliminary efficacy trial set to begin in the first half of 2026. Designed to be aerosolized and inhaled, ARCT-032 is an mRNA therapy that helps restore expression and activity of the cystic fibrosis transmembrane conductance (CFTR) regulator gene in the lungs. In patients with cystic fibrosis, the CFTR protein is either deficient or completely absent, resulting in compromised airway function, giving rise to key symptoms such as inflammation and respiratory failure. Current therapies for cystic fibrosis, such as Vertex Pharmaceuticals’ Trikafta and the recently approved Alfytrek , also address CFTR dysfunction. Also on Wednesday, Arcturus revealed in an SEC filing that the FDA asked the company to delay its biological license application for its COVID-19 vaccine, citing the need for further data. The regulator had previously agreed that Arcturus’ current data “could support a single-dose indication,” but it has since walked back on that determination and found that “additional data from a clinical endpoint efficacy study will be needed.”