Icatibant Acetate

iStock, kentoh After winning a surprise approval for its hereditary angioedema drug Ekterly, KalVista is confident the oral offering will capture the lion’s share of the market for on-demand use. KalVista’s road to an FDA approval in hereditary angioedema was anything but straightforward. In June 2025, the agency said it would miss an expected PDUFA date due to “resource constraints.” Then, reports emerged that Commissioner Marty Makary had personally tried to get the company’s drug, Ekterly, rejected. In July, the on-demand oral drug for hereditary angioedema (HAE) attacks got the nod anyway. CEO Benjamin Palleiko, when asked about the company’s dealings with the FDA in an interview on the sidelines of the 2026 J.P. Morgan Healthcare Conference, demurred, saying that FDA staff had been nothing but professional. “We took [the missed PDUFA date] at face value; it was a blip for us,” he said. Palleiko and other KalVista executives came to JPM26 for a mix of business development meetings and Ekterly promotion, he continued. “The focus of the team on the BD side has been on commercial collaborations,” he said, but the company is also focused on marketing Ekterly as the best drug on the market for treating HAE attacks as they happen. Shrugging Off Competitors HAE is a rare genetic disorder that causes potentially life-threatening swelling in different parts of the body. Different reports put the size of the global HAE market at between $3.8 billion and $6.5 billion in 2025. According to Palleiko, two-thirds of the patient population is on-demand while one-third takes prophylactics. On the prophylactics side, patients with HAE have a couple of new options. Ionis got an August 2025 approval for its RNA-targeting therapy Dawnzera, while CSL’s Andembry was approved in June 2025. Both are injectable prophylactic drugs, meant to be taken regularly to prevent occurrences. KalVista stressed repeatedly that Ekterly is the only drug among the three that can be taken on-demand, in pill form, to prevent an attack, like heading off a sudden migraine. “[Ionis and CSL are] evolving in very, very different ways,” Palleiko said. “People think it’s one thing but it’s really a tale of two markets,” the prophylactic and on-demand market. Rare diseases As New Hereditary Angioedema Drugs Flood the Market, Will Uptake Match Innovation? This year has seen the approval of several first-in-class therapies for HAE, but in a fragmented space, experts question whether they will be enough to net their developers a significant share of the entrenched market. December 1, 2025 · 5 min read · Ben Hargreaves Read more In the on-demand space, “notionally there are other products in the space,” he added—for example, Takeda’s Firazyr and Takhzyro. “But they’re mature, one is generic, it’s not very competitive,” Palleiko said. “For us, the opportunity is that they’re all injectable or IV, and we’re just a better mousetrap. It’s just a better drug.” When asked about other competitors in the space, Palleiko nodded. “There is someone developing an oral on demand,” he said, and immediately followed that statement with, “No, I’m not worried about it.” The late-stage biopharma Pharvaris is developing its own oral on-demand drug in HAE called deucrictibant. That drug yielded positive topline Phase III data in early December 2025 that will be used to support marketing authorization applications in the first half of this year. But Palleiko expects Ekterly to be well established by the time Pharvaris’ offering potentially hits the market. “They’ll show up in mid-2027, but it’s a big market. It’s a space that even when they enter, it’ll have fundamentally different market dynamics.” Go About Your Day Palleiko shared a story of a patient taking Ekterly. A woman driving her car felt an HAE attack come on, took an Ekterly pill and simply went about her day. Otherwise, she would have had to go home to take an icatibant injection—Firazyr’s generic—since it can’t be kept in a hot car’s glove compartment. As Ekterly further penetrates the market, KalVista expects people to switch. “People respond the same everywhere,” Palleiko said. “If you have a tablet, you’re going to generally switch” from another delivery modality. He added that the Ekterly pill “is slippery,” meaning a patient doesn’t even need water to swallow it. The next push for KalVista is into the pediatric space. While Ekterly is approved for patients aged 12 and older, the company is aiming for kids as young as 2, who in the past were not thought to even experience HAE attacks, according to Palleiko. He said that mindset produced situations where children wouldn’t tell parents about symptoms, and parents would be loathe to bring children to the hospital, where treatment would likely be an invasive IV therapy. KalVista has a Phase III trial studying Ekterly in patients aged 2 to 11, with an expected end date in August 2027. Interim data from that trial, called KONFIDENT-KID, showed that the drug brought symptom relief in an average of 1 1/2 hours across 65 attacks in 26 children in the trial. In the hotel room at JPM26 where the interview took place, Palleiko said the push into treating children is not motivated by money. “[Pediatric HAE is] not a big dollar thing,” Palleiko said. “It’s just a high unmet need thing.”

Dive Brief:Pharvaris said Wednesday its pill for the rare swelling disorder hereditary angioedema succeeded in a Phase 3 trial, helping to begin relieving symptoms of an attack within 1.28 hours, significantly faster than a placebo.The Netherlands-based biotechnology company said it plans on asking the Food and Drug Administration in the first half of 2026 to approve deucrictibant. An extended release formulation of the medication is also in a Phase 3 trial to test whether it can prevent HAE attacks, with data expected next year.If approved, deucrictibant would be entering an increasingly competitive market. The first oral HAE drug to relieve attacks, Kalvistas Ekterly, got an FDA nod this year, while two injectable drugs, including one to relieve attacks, also recently launched.Dive Insight:Treatment of HAE began with plasma products designed to replace a protein called C1 that regulates blood vessel dilation, but many of these early products could only be administered intravenously. That began to change in the early 2010s with Firazyr, now sold by Takeda, as the first self-administered injection for HAE attacks.More medicines designed to relieve symptoms of attacks or reduce their frequency have been introduced in the years since. However, 2025 broke new ground with the approval of three drugs: Ekterly, and the preventive shots Andembry from CSL and Dawnzera from Ionis Pharmaceuticals.Ekterlys launch pointed to strong demand for an oral drug. Sales of $13.7 million for the third quarter of 2025 well exceeded Leerink Partners forecast of $4 million. In assessing the deucrictibant data, Leerink analyst Joseph Schwartz wrote the market is very receptive to new options in the HAE space.We continue to believe that the HAE market is large enough for multiple players, especially as it continues to expand with the entrance of new options, Schwartz wrote.On its primary endpoint, time to onset of symptom response, deucrictibants median 1.28 hours is numerically better than Ekterlys 1.79 hours for its FDA-approved first dose, Schwartz added, although the two drugs havent been tested head-to-head. More impressive, he wrote, was the 11.95 hours median time to complete resolution of symptoms for deucrictibant, a measure on which Ekterly took more than 24 hours.Stifel analyst Paul Matteis, who covers Kalvista, said the differences in effectiveness might not necessarily drive patients to switch once they have begun taking Ekterly, however.If you look historically at the on demand market, subtle differences in efficacy haven't really been the determinant of majority share, Matteis wrote. Within a few months, Kalvista penetrated the market by about 10% and will have the opportunity to entrench itself further before deucrictibant might gain approval. '