KalVista Completes Enrollment in Pediatric HAE Trial for Oral Therapy Sebetralstat
Key Insights
KalVista Pharmaceuticals has completed enrollment in the KONFIDENT-KID trial of sebetralstat, an oral plasma kallikrein (search) inhibitor for children aged 2-11 with hereditary angioedema (search), a full year ahead of schedule.
Due to overwhelming interest from families affected by HAE (search), the trial was expanded from 24 to nearly 36 children across seven countries in Asia, Europe, and North America.
If approved, sebetralstat would become the first oral on-demand therapy for pediatric HAE (search) patients, with initial results expected later this year and a supplemental New Drug Application planned by mid-2026.
KalVista Pharmaceuticals has announced the completion of enrollment in its KONFIDENT-KID clinical trial, testing sebetralstat in pediatric patients with hereditary angioedema (search) (HAE (search)). The trial, which targets children between the ages of two and 11, reached full enrollment a full year ahead of schedule, highlighting the significant unmet need for oral treatment options in this patient population.
Sebetralstat, an investigational novel oral plasma kallikrein (search) inhibitor, could potentially become the first oral on-demand therapy for HAE (search) in children if approved by regulatory authorities.
Trial Expansion Due to High Demand
The KONFIDENT-KID trial was initially designed to enroll 24 subjects but has since been expanded to include nearly 36 children due to overwhelming interest from families affected by HAE (search). The expanded study now spans seven countries across Asia, Europe, and North America.
"We're proud to share that we achieved target enrollment in our KONFIDENT-KID trial a full year ahead of schedule and expanded the trial size due to overwhelming interest," said Ben Palleiko, CEO of KalVista. "The high level of participation from families living with HAE (search) underscores the significant need for an oral treatment option for this population."
Despite the increased trial size, KalVista anticipates that initial results will be available later this year, with a supplemental New Drug Application (sNDA) expected to be filed by mid-2026.
Study Design and Objectives
The open-label trial aims to gather comprehensive data on sebetralstat's performance in pediatric HAE (search) patients. Over a 12-month period, researchers will collect pharmacokinetic, efficacy, and safety data using a pediatric oral disintegrating tablet formulation specifically developed for children.
This specialized formulation addresses the challenges of medication administration in young patients, potentially improving treatment adherence and quality of life for children with HAE (search).
Regulatory Status and Potential Impact
Sebetralstat is currently under review by the U.S. Food and Drug Administration (FDA), with a Prescription Drug User Fee Act (PDUFA) goal date set for June 17, 2024. If approved, it would represent a significant advancement in HAE (search) treatment options for children.
The approval would make sebetralstat:
- The first oral on-demand therapy for children aged 2-11 years
- The second on-demand therapy of any type approved for this age group
KalVista has also submitted marketing authorization applications for sebetralstat to the European Medicines Agency and other international regulatory bodies, positioning the therapy for potential global availability.
Understanding Hereditary Angioedema
HAE (search) is a rare genetic condition characterized by a deficiency or dysfunction in the C1 esterase inhibitor protein. This deficiency leads to uncontrolled activation of the kallikrein-kinin system (search), resulting in painful and potentially life-threatening swelling attacks that can affect various parts of the body.
Currently, all approved on-demand treatment options for HAE (search) require either intravenous or subcutaneous administration, creating significant barriers to treatment, especially for children. An oral therapy could substantially improve disease management and quality of life for young patients and their families.
KalVista believes that if approved, sebetralstat could become a foundational therapy for managing HAE (search) worldwide, addressing a critical gap in treatment options for this debilitating condition.