KalVista Pharmaceuticals Announces FDA Approval of EKTERLY® (sebetralstat), First and Only Oral On-demand Treatment for Hereditary Angioedema

CAMBRIDGE, Mass. & SALISBURY, England--(BUSINESS WIRE)--Jul 7, 2025--

KalVista Pharmaceuticals, Inc. (Nasdaq: KALV) today announced that the U.S. Food and Drug Administration (FDA) has approved EKTERLY ® (sebetralstat), a novel plasma kallikrein inhibitor, for the treatment of 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.

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“The FDA approval of EKTERLY is a defining moment for people living with HAE,” said Ben Palleiko, CEO of KalVista. “EKTERLY enables people to treat attacks the moment symptoms begin, wherever they are. This approval affirms the strength of our science and deep commitment to the HAE community. I am profoundly grateful to the KalVista team for their dedication and perseverance, and to the patients and healthcare providers, as well as the HAEA and HAEi, for making this possible. EKTERLY has the potential to become the foundational treatment for HAE and our focus now is on delivering it to the people who need it.”

“As the first orally administered on-demand therapy for HAE attacks, EKTERLY provides patients and physicians with an important and welcome advance in HAE treatment options,” said Anthony J. Castaldo, chief executive officer of the U.S. Hereditary Angioedema Association.

Prior to EKTERLY’s approval, all on-demand HAE treatment options approved in the U.S. required intravenous or subcutaneous administration, which carries a significant treatment burden. 1 Even with the use of long-term prophylaxis as a preventative therapy, most people living with HAE continue to have unpredictable attacks and require ready access to on-demand medication. 1

“This is an important moment for patients, giving people living with HAE a treatment option that could provide greater independence and control over managing their condition ,” said Marc A. Riedl, MD, Professor of Medicine and Clinical Director, U.S. Hereditary Angioedema Association Center at the University of California, San Diego, and an investigator for the KONFIDENT phase 3 trial. “Until now, on-demand treatment relied on injectable subcutaneous or intravenous administration, often resulting in delayed intervention. Having an oral option empowers patients to treat attacks early, which aligns with treatment guidelines and advances our goal as physicians to reduce the overall burden of disease.”

The efficacy and safety of EKTERLY was established by the results from KalVista’s phase 3 KONFIDENT clinical trial, which was the largest clinical trial program ever conducted in HAE. Data from KONFIDENT was published in the New England Journal of Medicine in May 2024, showing that EKTERLY achieved significantly faster symptom relief, reduction in attack severity and attack resolution than placebo, and was well-tolerated with a safety profile similar to placebo .2 The trial randomized 136 HAE patients from 66 clinical sites across 20 countries. 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 shows that beginning of symptom relief occurred in a median of 1.3 hours among attacks involving the larynx, the abdomen, and for breakthrough attacks among patients receiving long-term prophylaxis. The safety profile of EKTERLY 600 mg in KONFIDENT-S, in a much larger number of attacks (>1700), was consistent with that observed in KONFIDENT.

KalVista will launch EKTERLY in the U.S. immediately and physicians can begin writing prescriptions today. As part of the Company’s commitment to supporting patients, KalVista has established KalVista Cares™, a comprehensive patient support program that offers personalized services and resources for eligible individuals. This includes assistance with navigating insurance coverage, access support, and ongoing help throughout the treatment journey.

For more information, visit EKTERLY.com.

Conference Call and Webcast
KalVista will host a live conference call and webcast to discuss the FDA approval of EKTERLY today, July 7, 2025, at 8:30 a.m. ET. The webcast will be accessible through the Investors section of the Company’s website at ir.kalvista.com. A replay will be available shortly after the conclusion of the live event.

About Hereditary Angioedema
Hereditary angioedema (HAE) is a rare genetic disease resulting in deficiency or dysfunction in the C1 esterase inhibitor (C1INH) protein and subsequent uncontrolled activation of the kallikrein-kinin system. People living with HAE experience painful and debilitating attacks of tissue swelling in various locations of the body that can be life-threatening depending on the area affected. Treatment guidelines recommend treating attacks as early as possible to prevent progression of swelling and shorten the time to attack resolution, and to consider treatment for all attacks, regardless of anatomic location or severity.

About EKTERLY ® (sebetralstat)
EKTERLY (sebetralstat), a novel plasma kallikrein inhibitor, is the first and only oral on-demand therapy approved by the U.S. FDA for the treatment of acute attacks of hereditary angioedema (HAE) in people 12 years of age and older. With ongoing studies exploring its use in children aged two to 11 and multiple regulatory applications under review in key global markets, EKTERLY has the potential to become the foundational therapy for HAE management worldwide.

INDICATION AND IMPORTANT SAFETY INFORMATION

What is EKTERLY ® (sebetralstat)?
EKTERLY is a prescription medicine used to treat sudden (acute) attacks of hereditary angioedema (HAE) in adults and children aged 12 years of age and older. It is not known if EKTERLY is safe and effective in children under 12 years of age.

IMPORTANT SAFETY INFORMATION

Before taking EKTERLY, tell your healthcare provider about all of your medical conditions, including if you:

Tell your healthcare provider about all of the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Taking EKTERLY with certain other medicines can cause side effects or affect how well EKTERLY or the other medicines work. Especially tell your healthcare provider if you take any of the following, as their use with EKTERLY is not recommended: itraconazole, phenytoin, efavirenz.

Know the medicines you take. Keep a list of them to show your healthcare provider or pharmacist when you get a new medicine.

What are the possible side effects of EKTERLY?

The most common side effects of EKTERLY include headache. For more information, ask your healthcare provider or pharmacist. Talk to your doctor for medical advice about side effects.

You are encouraged to report side effects related to KalVista products by calling 1-855-258-4782. If you prefer, you may contact the U.S. Food and Drug Administration (FDA) directly. Visit www.fda.gov/medwatch or call 1-800-FDA-1088.

Please click here for fullPrescribing Information, including Patient Information.

About KalVista Pharmaceuticals, Inc.
KalVista Pharmaceuticals, Inc., is a global biopharmaceutical company dedicated to developing and delivering life-changing oral therapies for individuals affected by rare diseases with significant unmet needs. In the U.S., KalVista markets EKTERLY ®, the first and only oral on-demand treatment for hereditary angioedema (HAE). The Company has multiple regulatory applications under review in key global markets. For more information about KalVista, please visit www.kalvista.com and follow us on LinkedIn, X, Facebook and Instagram.

Forward-Looking Statements
This press release contains "forward-looking" statements within the meaning of the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of 1995. Forward-looking statements can be identified by words such as: "anticipate," "intend," "plan," "goal," "seek," "believe," "project," "estimate," "expect," "strategy," "future," "likely," "may," "should," "will" and similar references to future periods. These statements are subject to numerous risks and uncertainties that could cause actual results to differ materially from what we expect. Examples of forward-looking statements include, among others, information relating to our business and business plans, the success of our efforts to commercialize EKTERLY ® (sebetralstat), our ability to successfully obtain foreign regulatory approvals for sebetralstat, our expectations about the safety and efficacy of sebetralstat and our other product candidates, the timing of clinical trials and their results, our ability to commence clinical studies or complete ongoing clinical studies, including our KONFIDENT-S and KONFIDENT-KID trials, and the ability of EKTERLY to treat HAE. Further information on potential risk factors that could affect our business and financial results are detailed in our filings with the Securities and Exchange Commission, including in our annual report on Form 10-K for the year ended April 30, 2024, our quarterly reports on Form 10-Q, and our other reports that we may make from time to time with the Securities and Exchange Commission. We undertake no obligation to publicly update any forward-looking statement, whether written or oral, that may be made from time to time, whether as a result of new information, future developments or otherwise.

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WASHINGTON (AP) — Becky Pepper-Jackson finished third in the discus throw in West Virginia last year though she was in just her first year of high school. Now a 15-year-old sophomore, Pepper-Jackson is aware that her upcoming season could be her last.

West Virginia has banned transgender girls like Pepper-Jackson from competing in girls and women's sports, and is among the more than two dozen states with similar laws. Though the West Virginia law has been blocked by lower courts, the outcome could be different at the conservative-dominated Supreme Court, which has allowed multiple restrictions on transgender people to be enforced in the past year.

The justices are hearing arguments Tuesday in two cases over whether the sports bans violate the Constitution or the landmark federal law known as Title IX that prohibits sex discrimination in education. The second case comes from Idaho, where college student Lindsay Hecox challenged that state's law.

Decisions are expected by early summer.

President Donald Trump's Republican administration has targeted transgender Americans from the first day of his second term, including ousting transgender people from the military and declaring that gender is immutable and determined at birth.

Pepper-Jackson has become the face of the nationwide battle over the participation of transgender girls in athletics that has played out at both the state and federal levels as Republicans have leveraged the issue as a fight for athletic fairness for women and girls.

“I think it’s something that needs to be done,” Pepper-Jackson said in an interview with The Associated Press that was conducted over Zoom. “It’s something I’m here to do because ... this is important to me. I know it’s important to other people. So, like, I’m here for it.”

She sat alongside her mother, Heather Jackson, on a sofa in their home just outside Bridgeport, a rural West Virginia community about 40 miles southwest of Morgantown, to talk about a legal fight that began when she was a middle schooler who finished near the back of the pack in cross-country races.

Pepper-Jackson has grown into a competitive discus and shot put thrower. In addition to the bronze medal in the discus, she finished eighth among shot putters.

She attributes her success to hard work, practicing at school and in her backyard, and lifting weights. Pepper-Jackson has been taking puberty-blocking medication and has publicly identified as a girl since she was in the third grade, though the Supreme Court's decision in June upholding state bans on gender-affirming medical treatment for minors has forced her to go out of state for care.

Her very improvement as an athlete has been cited as a reason she should not be allowed to compete against girls.

“There are immutable physical and biological characteristic differences between men and women that make men bigger, stronger, and faster than women. And if we allow biological males to play sports against biological females, those differences will erode the ability and the places for women in these sports which we have fought so hard for over the last 50 years,” West Virginia's attorney general, JB McCuskey, said in an AP interview. McCuskey said he is not aware of any other transgender athlete in the state who has competed or is trying to compete in girls or women’s sports.

Despite the small numbers of transgender athletes, the issue has taken on outsize importance. The NCAA and the U.S. Olympic and Paralympic Committees banned transgender women from women's sports after Trump signed an executive order aimed at barring their participation.

The public generally is supportive of the limits. An Associated Press-NORC Center for Public Affairs Research poll conducted in October 2025 found that about 6 in 10 U.S. adults “strongly” or “somewhat” favored requiring transgender children and teenagers to only compete on sports teams that match the sex they were assigned at birth, not the gender they identify with, while about 2 in 10 were “strongly” or “somewhat” opposed and about one-quarter did not have an opinion.

About 2.1 million adults, or 0.8%, and 724,000 people age 13 to 17, or 3.3%, identify as transgender in the U.S., according to the Williams Institute at the UCLA School of Law.

Those allied with the administration on the issue paint it in broader terms than just sports, pointing to state laws, Trump administration policies and court rulings against transgender people.

"I think there are cultural, political, legal headwinds all supporting this notion that it’s just a lie that a man can be a woman," said John Bursch, a lawyer with the conservative Christian law firm Alliance Defending Freedom that has led the legal campaign against transgender people. “And if we want a society that respects women and girls, then we need to come to terms with that truth. And the sooner that we do that, the better it will be for women everywhere, whether that be in high school sports teams, high school locker rooms and showers, abused women’s shelters, women’s prisons.”

But Heather Jackson offered different terms to describe the effort to keep her daughter off West Virginia's playing fields.

“Hatred. It’s nothing but hatred,” she said. "This community is the community du jour. We have a long history of isolating marginalized parts of the community.”

Pepper-Jackson has seen some of the uglier side of the debate on display, including when a competitor wore a T-shirt at the championship meet that said, “Men Don't Belong in Women's Sports.”

“I wish these people would educate themselves. Just so they would know that I’m just there to have a good time. That’s it. But it just, it hurts sometimes, like, it gets to me sometimes, but I try to brush it off,” she said.

One schoolmate, identified as A.C. in court papers, said Pepper-Jackson has herself used graphic language in sexually bullying her teammates.

Asked whether she said any of what is alleged, Pepper-Jackson said, “I did not. And the school ruled that there was no evidence to prove that it was true.”

The legal fight will turn on whether the Constitution's equal protection clause or the Title IX anti-discrimination law protects transgender people.

The court ruled in 2020 that workplace discrimination against transgender people is sex discrimination, but refused to extend the logic of that decision to the case over health care for transgender minors.

The court has been deluged by dueling legal briefs from Republican- and Democratic-led states, members of Congress, athletes, doctors, scientists and scholars.

The outcome also could influence separate legal efforts seeking to bar transgender athletes in states that have continued to allow them to compete.

If Pepper-Jackson is forced to stop competing, she said she will still be able to lift weights and continue playing trumpet in the school concert and jazz bands.

“It will hurt a lot, and I know it will, but that’s what I’ll have to do,” she said.

Heather Jackson, left, and Becky Pepper-Jackson pose for a photograph outside of the U.S. Supreme Court in Washington, Sunday, Jan. 11, 2026. (AP Photo/Jose Luis Magana)

Heather Jackson, left, and Becky Pepper-Jackson pose for a photograph outside of the U.S. Supreme Court in Washington, Sunday, Jan. 11, 2026. (AP Photo/Jose Luis Magana)

Becky Pepper-Jackson poses for a photograph outside of the U.S. Supreme Court in Washington, Sunday, Jan. 11, 2026. (AP Photo/Jose Luis Magana)

The Supreme Court stands is Washington, Friday, Jan. 9, 2026. (AP Photo/J. Scott Applewhite)

FILE - Protestors hold signs during a rally at the state capitol in Charleston, W.Va., on March 9, 2023. (AP Photo/Chris Jackson, file)