The U.S. Food and Drug Administration has notified KalVista Pharmaceuticals that it will not meet the June 17, 2025 PDUFA goal date for sebetralstat, the company's investigational oral treatment for hereditary angioedema (HAE), citing heavy workload and limited resources. The agency expects to deliver a decision within approximately four weeks of the original deadline.
Regulatory Status and Timeline
KalVista received notification on June 13, 2025, just four days before the scheduled PDUFA date. The FDA has not requested additional data or studies and has not raised concerns regarding the safety, efficacy, or approvability of sebetralstat. According to the company, all prior information requests have been addressed in a timely manner, with only labeling finalization remaining under FDA review.
"We are disappointed by this delay, most importantly because we know how much people living with HAE are looking forward to an oral on-demand option to treat their HAE attacks," said Ben Palleiko, CEO of KalVista. "At the same time, we remain confident in the near-term approval of sebetralstat."
Therapeutic Significance
Sebetralstat is positioned to become the first oral, on-demand treatment for HAE in individuals aged 12 and older. The investigational drug is a novel oral plasma kallikrein inhibitor that could address a significant unmet medical need in HAE management. Currently, all approved on-demand treatment options require either intravenous or subcutaneous administration.
If approved, sebetralstat has the potential to become the foundational therapy for HAE management worldwide, according to KalVista. The company is also investigating its use in children aged 2 to 11 years old.
Disease Background and Market Need
Hereditary angioedema is a rare genetic disease resulting from deficiency or dysfunction in the C1 esterase inhibitor (C1INH) protein and subsequent uncontrolled activation of the kallikrein-kinin system. Patients experience painful and debilitating attacks of tissue swelling in various body locations that can be life-threatening depending on the affected area.
The current treatment landscape requires injectable therapies, creating barriers to immediate treatment access during acute attacks. An oral on-demand option would represent a significant advancement in patient care and treatment convenience.
Global Regulatory Strategy
Beyond the U.S. application, KalVista has completed Marketing Authorization Applications for sebetralstat to the European Medicines Agency and multiple other global regulatory authorities, indicating a comprehensive international development strategy for the therapy.
