CAMBRIDGE, Mass. & SALISBURY, England - KalVista Pharmaceuticals, Inc. (NASDAQ: KALV) has received Orphan Drug Designation for sebetralstat from Japan's Ministry of Health, Labour and Welfare (MHLW), the company announced today. Alongside this designation, the company has submitted a New Drug Application (NDA) for sebetralstat in Japan, which, if approved, would mark it as the first oral on-demand treatment for hereditary angioedema (HAE) in the country.

The NDA submission is based on results from the KONFIDENT phase 3 clinical trial and the ongoing KONFIDENT-S open-label extension trial. The KONFIDENT trial, which is the largest ever conducted for HAE, involved 136 patients across 20 countries. Participants in this trial were given sebetralstat to treat acute HAE attacks. The KONFIDENT-S trial continues to evaluate the long-term safety and efficacy of the drug, with plans for a novel oral disintegrating tablet (ODT) formulation intended for a 2026 supplemental New Drug Application (sNDA) filing. With analyst price targets ranging from $18 to $39, significantly above the current trading price of $8.30, market expectations appear optimistic despite recent financial challenges.

Sebetralstat is a novel oral plasma kallikrein inhibitor aimed at treating HAE attacks in adults and adolescents aged 12 and older. The drug has also been submitted for regulatory review by the U.S. FDA, with a Prescription Drug User Fee Act (PDUFA) goal date of June 17, 2025. Marketing Authorization Applications have been completed for sebetralstat to the European Medicines Agency and other global regulatory authorities.

Hereditary angioedema is a rare genetic disorder characterized by spontaneous episodes of swelling in various parts of the body, which can be life-threatening depending on the location. Current on-demand treatments for HAE require either intravenous or subcutaneous administration, which highlights the significance of an oral treatment option like sebetralstat.

The company's CEO, Ben Palleiko, emphasized the critical need for new, effective treatments for HAE in Japan and expressed pride in KalVista's role in advancing care for the HAE community.

This news is based on a press release statement and contains forward-looking statements that involve risks and uncertainties, as detailed in KalVista's filings with the Securities and Exchange Commission. The company's financial health score from InvestingPro is rated as "Fair," with notable strengths in cash flow management despite current unprofitability. The stock has shown recent momentum with a 12% gain over the past week, though it remains 33% lower year-over-year.

In other recent news, KalVista Pharmaceuticals has seen significant progress in the development of its drug sebetralstat. Analysts at TD Cowen project that the drug could achieve over $750 million in peak sales, while BofA Securities estimates peak risk-adjusted sales of $604 million by 2035. Both firms have assigned a Buy rating to KalVista's stock, with TD Cowen setting a price target of $30 and BofA Securities at $22.

In terms of leadership, KalVista has appointed Jeb Ledell as its new Chief Operating Officer and Laurence Reid, Ph.D., to its Board of Directors. The company has also appointed Brian Piekos as the new CFO. These appointments come as KalVista prepares for the global launch of sebetralstat, which is anticipated in the second half of 2025.

On the other hand, H.C. Wainwright maintained a Buy rating for KalVista, citing the potential market strength for sebetralstat. The firm believes there will be significant demand for the drug upon its launch, as it offers a pain-free, convenient, and effective alternative to existing on-demand therapies for hereditary angioedema.

Lastly, KalVista has secured over $160 million in capital through financing transactions and equity offerings, and has announced a public offering and a concurrent private placement of shares, aiming to raise a total of $60 million. It's important to note that these are recent developments and do not include a summary or conclusion.

Japan grants orphan drug status to HAE treatment

KalVista Pharmaceuticals, Inc. has received Orphan Drug Designation from Japan's Ministry of Health, Labour and Welfare for its HAE treatment sebetralstat. The company has submitted a New Drug Application in Japan, based on the KONFIDENT phase 3 trial results. Analysts predict significant sales potential for sebetralstat, with TD Cowen projecting over $750 million in peak sales.