Pendopharm, Kalvista to bring sebetralstat for HAE to Canada
Application seeking approval in the US has been delayed by 4 weeks: FDA
Pendopharm has obtained the exclusive rights to manage the regulatory approval process and commercialization of sebetralstat, an on-demand investigational treatment for hereditary angioedema (HAE), in Canada under a licensing agreement with Kalvista Pharmaceuticals, the developer of the therapy.
“We look forward to collaborating with Pendopharm, whose deep knowledge of the Canadian market and proven track record make them a strong partner as we work to bring sebetralstat to people living with HAE,” Ben Palleiko, CEO of KalVista, said in a company press release.
Meanwhile, the U.S. Food and Drug Administration has notified Kalvista that its review of an application seeking sebetralstat’s approval in the U.S. has been delayed due to a heavy workload and limited resources. The agency indicated that it expects to deliver a decision within about four weeks of the previous action date of June 17.
“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,” Palleiko said in a separate press release. “At the same time, we remain confident in the near-term approval of sebetralstat.”
Applications submitted in EU, UK, other countries
HAE is marked by overactivity by the enzyme kallikrein, which controls the production of a signaling molecule called bradykinin. Excessive kallikrein activity boosts bradykinin levels, leading to blood vessel leakage and fluid accumulation in nearby tissues, which causes swelling.
Sebetralstat works by blocking the activity of kallikrein in the bloodstream, thereby stopping bradykinin levels from rising and easing HAE swelling attacks. The therapy is administered as an oral tablet, unlike available on-demand HAE treatments that are given via intravenous (into-the-vein) or subcutaneous (under-the-skin) injections.
Kalvista has submitted applications requesting approval for sebetralstat in the U.S., the European Union, the U.K., Switzerland, Australia, and Singapore.
Data from the Phase 3 KONFIDENT trial (NCT05259917) and its extension study, KONFIDENT-S (NCT05505916), supported the approval applications. Results showed on-demand treatment with sebetralstat led to rapid symptom relief, with attacks easing within two hours of taking the tablets. In some cases, a single dose was sufficient to start bringing symptom relief.
This partnership supports our broader goal of making sebetralstat available globally, as the first and only oral on-demand treatment that has the potential to transform HAE care.
In a recent analysis of KONFIDENT-S data, the median time to the end of attack progression after on-demand treatment was about 20 minutes, which was similar to the time observed in the KONFIDENT trial. The therapy was also effective in attacks rated as severe or very severe, as well as those involving the abdomen and the larynx, or voice box, the latter of which can be life-threatening as they may obstruct breathing.
Regulatory agencies in the U.S. and EU have granted orphan drug designation to sebetralstat, a status that provides financial incentives and expedited review for therapies targeting rare diseases.
Palleiko called the partnership with Pendopharm, details of which have not been disclosed, an essential step in making sebetralstat available to HAE patients worldwide.
“This partnership supports our broader goal of making sebetralstat available globally, as the first and only oral on-demand treatment that has the potential to transform HAE care,” he said.
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