Ekterly’s road to approval was not a smooth one. Last month, the FDA informed KalVista it would not meet its PDUFA date due to resource constraints and reports surfaced that Commissioner Marty Makary tried to have the application rejected.
The FDA on Monday approved the use of KalVista Pharmaceuticals’ sebetralstat to treat acute hereditary angioedema attacks in patients 12 years and up. The Massachusetts biotech expects to make the drug available in the U.S. “immediately” under the brand name Ekterly, the first and so far only oral and on-demand treatment for hereditary angioedema (HAE), according to KalVista’s press release.
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.”
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, showed that both Ekterly doses achieved symptom relief significantly more rapidly, with a median of 1.61 hours and 1.79 hours in the 300-mg and 600-mg dose arms, respectively, versus 6.72 hours for the placebo group.
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.
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.
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.”
