KalVista Pharmaceuticals submits FDA application for sebetralstat
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Key takeaways:
- Sebetralstat is the first on-demand oral therapy that would be available to patients with hereditary angioedema.
- New study aims to gather data on patients aged 2 to 11 years for future drug approval.
KalVista Pharmaceuticals Inc. submitted a new drug application to the FDA for the approval of sebetralstat for patients aged 12 years and older with hereditary angioedema, according to a company press release.
“If approved, we believe sebetralstat could become a foundational treatment that will transform the way people living with HAE treat their disease,” Ben Palleiko, MBA, MA, CEO of KalVista, said in the release.
The company also announced the start of a new clinical trial, KONFIDENT-KID, that will examine the use of sebetralstat for hereditary angioedema for patients aged 2 to 11 years, according to its June press release.
Sebetralstat works as an oral plasma kallikrein inhibitor for on-demand treatment of hereditary angioedema. The new drug application is based on results from two previous clinical trials, the KONFIDENT phase 3 clinical trial and the KONFIDENT-S extension trial.
Sebetralstat achieved symptom relief faster than the placebo in the phase 3 trial (P < .0001 for 300 mg; P = .0013 for 600 mg). The median time to beginning of symptom relief for sebetralstat 300 mg was 1.61 hours (95% CI, 1.28-2.27), 1.79 hours with 600 mg (95% CI, 1.33-2.27) and 6.72 hours with placebo (95% CI, 2.33 to > 12). The safety profile was no different from the placebo and no treatment-related serious adverse events were observed.
The KONFIDENT-KID study for patients aims to collect safety, pharmacokinetic and efficacy data in 24 children across seven countries in North America, Europe and Asia. Data will be collected for up to 1 year, and the study will utilize a proprietary pediatric oral disintegrating tablet formulation of sebetralstat. If approved, it would be the first oral on-demand therapy in pediatric patients aged 2 to 11 years.
“Currently, the only approved on-demand treatment for this population is administered intravenously, making this a critical area of unmet need for people living with HAE,” Palleiko said.
The FDA has 60 days to review the submission, and the company anticipates a result in September. The company also plans to present further data from the KONFIDENT and KONFIDENT-S trials at the 2024 Annual Scientific Conference of the American College of Allergy, Asthma, and Immunology Conference on Oct. 24 to 28 in Boston.
Reference:
- KalVista initiates KONFIDENT-KID trial for on-demand treatment of hereditary angioedema attacks with sebetralstat in children aged 2 to 11. https://ir.kalvista.com/news-releases/news-release-details/kalvista-initiates-konfident-kid-trial-demand-treatment. Published June 27, 2024. Accessed July 15, 2024.
Perspective
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Current limitations in HAE treatment include a need for more oral options, especially for an acute, on-demand therapy. Patients have never had an oral medication that they could use for an acute HAE attack. This is important, as IV infusions or subcutaneous injections pose a barrier to some patients and may delay their decisions to treat an acute attack.
Plus, many of these patients have experienced a “needle fatigue” through many years of treating with medications requiring needles. If sebetralstat is FDA-approved, it would fill that gap for patients so they could have that oral, on-demand therapy. It would be the first of its kind. I am optimistic it will be approved by the FDA.
The KONFIDENT-KID study is so significant because options for children are so limited, and needles often scare them or make them more anxious. Having an oral option potentially available could really shift the dynamic for children as well as adults.
HAE carries a significant burden. When I say burden, I mean the disease is a burden for many patients, as it can be unpredictable and potentially life-threatening. Further, there has been a burden of treatment where past treatment options, especially for acute attacks, have not been easy and require needles. This can be daunting for children, in particular.
Having an oral option in this case adds to the current menu of medications available, and I think it may alleviate the burden of disease and treatment as long as it demonstrates safety and efficacy in the clinical trials.
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