European regulator recommends approval of first oral HAE treatment

Published 25/07/2025, 13:34
European regulator recommends approval of first oral HAE treatment

CAMBRIDGE, Mass. & SALISBURY, England - The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended approval of sebetralstat, an oral treatment for hereditary angioedema (HAE) attacks in patients aged 12 and older, KalVista Pharmaceuticals, Inc. (NASDAQ:KALV) announced Friday.

If approved by the European Commission, which is expected to make its final decision by early October, sebetralstat would become the first and only oral on-demand treatment for HAE available in the European Union.

The positive opinion follows recent regulatory approvals in the United States and United Kingdom, where the drug is marketed under the brand name EKTERLY.

CHMP based its recommendation on results from the phase 3 KONFIDENT clinical trial, which enrolled 136 HAE patients across 66 clinical sites in 20 countries. According to data published in the New England Journal of Medicine in May 2024, the drug demonstrated significantly faster symptom relief, reduced attack severity, and accelerated attack resolution compared to placebo, with a safety profile similar to placebo. Analysts maintain a strong buy consensus on the stock, with price targets ranging from $20 to $40, as revealed by InvestingPro analysis.

HAE is a rare genetic disease characterized by painful and potentially life-threatening swelling attacks in various body locations. Current on-demand treatments require injection, while sebetralstat offers an oral alternative.

"Access to on-demand treatments that offer easy self-administration and rapid symptom relief is critical for patients living with HAE," said Emel Aygören-Pürsün, Assistant Professor at University Hospital Frankfurt, in the company’s press release.

The most common side effect reported with sebetralstat is headache, according to the company’s statement.

KalVista is also pursuing regulatory approvals in Japan and other territories, while conducting additional studies in children aged two to 11 years. While the company maintains strong liquidity with a current ratio of 5.35 and more cash than debt on its balance sheet, InvestingPro analysis indicates rapid cash burn as a key concern. InvestingPro subscribers have access to 13 additional key insights about KalVista’s financial health and market position.

In other recent news, Kalvista Pharmaceuticals has achieved several significant milestones. The company announced that its hereditary angioedema (HAE) treatment, Ekterly (sebetralstat), has received marketing authorization from the UK’s Medicines and Healthcare products Regulatory Agency (MHRA), marking it as the first oral on-demand treatment for HAE approved in the UK. This approval grants Ekterly up to 10 years of market exclusivity as it is added to the MHRA’s Orphan Register. Additionally, Ekterly has launched in the U.S. following FDA approval, which includes a favorable label with no listed contraindications and approval for a higher 600 mg dose.

In light of these developments, several financial firms have reiterated their positive outlook on Kalvista. JMP Securities has maintained its Market Outperform rating and set a $27.00 price target, citing the potential of the stabilized on-demand HAE market. H.C. Wainwright also reiterated a Buy rating with a $27.00 price target, highlighting the favorable aspects of Ekterly’s label. Furthermore, Leerink Partners has reiterated an Outperform rating with a $20.00 price target as the U.S. launch of Ekterly begins. These developments underscore the strategic progress Kalvista is making in expanding its market presence with Ekterly.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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