SHERIDAN, WYOMING – July 8, 2025 – KalVista Pharmaceuticals has received U.S. FDA approval for its oral treatment Ekterly (sebetralstat), marking a major milestone as the first and only on-demand tablet therapy for hereditary angioedema (HAE) in patients aged 12 and older.
The company confirmed that the drug will be available in the U.S. “immediately” under the brand name Ekterly. According to KalVista’s press release, it is “the first and so far only oral and on-demand treatment for hereditary angioedema (HAE).”
Regulatory delays and internal agency tensions
Ekterly’s road to approval was not smooth. Last month, the FDA informed KalVista that it would not meet its original target action date of June 17, citing “heavy workload and limited resources,” according to the biotech’s announcement at the time.
A few days after news of the delay broke, Endpoints News, citing anonymous internal sources, reported that FDA Commissioner Marty Makary had tried to get the application rejected. Makary ultimately relented after senior officials pushed back, raising potential legal issues that such an interference would be “arbitrary and capricious,” according to internal messages reviewed by the publication. It is unclear why Makary wanted the application rejected.
In a statement to Endpoints, a spokesperson for the Department of Health and Human Services denied Makary’s actions, calling the claims by the sources “totally false and untrue.”
Large-scale data confirm rapid treatment response
The FDA’s ultimate approval was backed by data from the Phase III KONFIDENT study, which according to the biotech is “the largest program ever conducted in HAE.” The trial enrolled 136 patients who were given either 600-mg or 300-mg Ekterly or placebo.
Results, released in February 2024, demonstrated significantly faster symptom relief:
- 300-mg dose: median 1.61 hours
- 600-mg dose: median 1.79 hours
- Placebo: median 6.72 hours
KalVista supported KONFIDENT with data from the KONFIDENT-S open-label extension study, providing a real-world peek at Ekterly’s efficacy, as per the biotech’s Monday release. Data as of September 2024 show that the drug helped patients address their HAE attacks “within a median of 10 minutes of onset.”
Clean safety label enhances clinical value proposition
Ekterly’s label is largely clean, with no boxed warnings. Precautions for adverse reactions flag headaches, which can occur in at least 2% of patients. Use of the drug should also be avoided in patients with severe hepatic impairment.
With a favorable safety profile and oral delivery, Ekterly offers a simplified and accessible option for managing acute HAE attacks—especially attractive in settings where injection-based solutions are impractical.
CEO underscores patient-centric breakthrough
In a prepared statement, CEO Ben Palleiko said that Ekterly’s approval is a “defining moment” for patients with HAE, providing them with a treatment option available “the moment symptoms begin.”
While the drug has not yet been launched in the U.S., KalVista on Monday said that “doctors can start prescribing Ekterly ‘today.’”
Strategic implications in the rare disease market
KalVista enters the market with first-mover advantage, bringing a tablet-based innovation to a space historically dominated by injectable therapies. Ekterly not only advances convenience and speed of administration but also reflects a growing industry shift toward patient-initiated treatment models in rare disease care.
Learn more at https://www.kalvista.com