UK approves first oral treatment for hereditary angioedema attacks

CAMBRIDGE, Mass. & SALISBURY, England - The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has granted marketing authorization for EKTERLY (sebetralstat) for the treatment of hereditary angioedema (HAE) attacks in patients aged 12 years and older, KalVista Pharmaceuticals announced Tuesday.

EKTERLY is the first and only oral on-demand treatment for HAE approved in the UK. The drug will be added to the MHRA’s Orphan Register, providing it with up to 10 years of market exclusivity. According to InvestingPro data, while KalVista maintains a strong balance sheet with more cash than debt, analysts expect continued investments in drug development to impact profitability in the near term.

The approval follows the drug’s recent authorization by the U.S. Food and Drug Administration on July 3, 2025. KalVista (NASDAQ:KALV) has also submitted marketing applications in the EU, Japan and other global markets.

"This approval holds particular significance for KalVista as EKTERLY was discovered in our Salisbury, UK labs," said Ben Palleiko, CEO of KalVista.

The MHRA’s decision was based on results from the phase 3 KONFIDENT clinical trial, which enrolled 136 HAE patients across 66 clinical sites in 20 countries. The study showed that EKTERLY achieved faster symptom relief, reduced attack severity, and accelerated attack resolution compared to placebo, with a safety profile similar to placebo.

EKTERLY has been available to UK patients with unmet needs through the Early Access to Medicines Scheme since March. It will continue to be available through this program until the National Institute for Health and Care Excellence (NICE) makes a decision on its use under the National Health Service, expected in the first half of 2026.

Hereditary angioedema is a rare genetic disease characterized by painful and sometimes life-threatening swelling attacks in various parts of the body.

According to a company press release, KalVista is also conducting studies of EKTERLY in children aged two to 11 years. With analyst price targets ranging from $20 to $40 per share, InvestingPro analysis suggests the stock is currently trading above its Fair Value. InvestingPro subscribers have access to 13 additional key insights about KalVista’s financial health and market position.

In other recent news, Kalvista Pharmaceuticals has reported several significant developments. The company recently received FDA approval for Ekterly, an oral treatment for hereditary angioedema (HAE), marking it as the first of its kind for on-demand use. Ekterly’s launch in the U.S. has commenced, with a wholesale acquisition cost set at $16,720 per dose. Analysts from Leerink Partners and BofA Securities have expressed optimism, with Leerink maintaining an Outperform rating and BofA raising its price target to $37.00 from $22.00. JMP Securities also increased its price target to $27.00, citing the drug’s pricing strategy and potential market penetration. Kalvista anticipates Ekterly’s international launch in Germany in the latter half of 2025, followed by Japan and the UK. The company has entered commercialization agreements with Kaken Pharmaceutical and Pendopharm for Japan and Canada, respectively. H.C. Wainwright reiterated a Buy rating, emphasizing Ekterly’s favorable safety profile and broad label. These developments are expected to position Ekterly strongly in the HAE market, with analysts projecting substantial sales growth in the coming years.

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