The U.S. Food and Drug Administration (FDA) on Monday approved KalVista Pharmaceuticals Inc.’s KALV Ekterly (sebetralstat) for acute attacks of hereditary angioedema (HAE) in adult and pediatric patients aged 12 years and older.
Ekterly is the first and only oral on-demand treatment for HAE. HAE is characterized by recurrent episodes of severe swelling (angioedema).
The efficacy and safety of Ekterly were established by the results from KalVista’s phase 3 KONFIDENT clinical trial, which was the largest clinical trial program ever conducted in HAE, the company said in a statement on Monday.
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Data from KONFIDENT was published in the New England Journal of Medicine in May 2024, showing that Ekterly achieved significantly faster symptom relief, reduced attack severity and resolution than placebo, and was well-tolerated with a safety profile similar to placebo.
The trial randomized 136 HAE patients.
These results were further supported by the more real-world KONFIDENT-S open-label extension trial, which, as of September 2024, showed that Ekterly enabled patients to treat attacks in a median of 10 minutes following onset.
The most recent data from KONFIDENT-S show that the beginning of symptom relief occurred in a median of 1.3 hours among attacks involving the larynx and the abdomen and for breakthrough attacks among patients receiving long-term prophylaxis.
KalVista will launch Ekterly in the U.S. immediately, and physicians can begin writing prescriptions today.
Due to heavy workload and limited resources, the FDA missed the PDUFA (Prescription Drug User Fee Act) goal date of 17 June for the New Drug Application (NDA) for sebetralstat for HAE.
In other developments, KalVista Pharmaceuticals also granted Pendopharm, a division of Pharmascience Inc., exclusive rights to manage the regulatory approval and commercialization of sebetralstat in Canada. The financial terms of this agreement were not disclosed.
Meanwhile, Intellia Therapeutics, Inc. NTLA is working on a different kind of treatment for HAE: an investigational in vivo CRISPR-based gene editing therapy called NTLA-2002.
In vivo gene editing means the gene-editing tools are delivered directly into the patient’s body to make changes to their DNA, aiming for a one-time, potentially curative treatment.
Intellia released promising Phase 2 data from its ongoing Phase 1/2 study of NTLA-2002 in October 2024. A single dose of NTLA-2002 showed mean monthly attack rates relative to placebo were reduced by 75% and 77% for the 25 mg and 50 mg arms during weeks 1–16 and by 80% and 81% during weeks 5–16, respectively.
In the 50 mg arm, eight of 11 patients experienced a complete response after a single dose of NTLA-2002, with no attacks at all during the 16-week primary observation period; these eight patients continued to be attack-free through the latest follow-up (median of eight months), and no subsequent treatment has been required.
Price Action: KALV stock is trading higher by 15.9% to $13.89 premarket at last check Monday.
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