KalVista Announces Orphan Drug Designation and NDA Submission for Sebetralstat in Japan for Hereditary Angioedema
2025年1月21日 - 9:00PM
ビジネスワイヤ（英語）
–Orphan drug designation paves the way for
potential of sebetralstat to be first oral on-demand treatment for
HAE in Japan–
– Submission advances Company’s strategic plan
to address unmet needs in HAE on global scale–
KalVista Pharmaceuticals, Inc. (NASDAQ: KALV), today announced
that Japan’s Ministry of Health, Labour and Welfare (MHLW) has
granted sebetralstat Orphan Drug Designation. The Company has also
submitted a New Drug Application (NDA) for sebetralstat to the
Agency. If approved, sebetralstat, a novel, investigational oral
plasma kallikrein inhibitor for the on-demand treatment of
hereditary angioedema (HAE) attacks in adults and adolescents aged
12 years and older, would be the first oral on-demand treatment for
HAE in Japan.
“The submission of our NDA for sebetralstat in Japan represents
another key step toward our efforts to make this important new
treatment available to as many people living with HAE as possible,”
said Ben Palleiko, CEO of KalVista. “The Orphan Drug Designation
not only acknowledges the critical need for new, effective
treatments for HAE in Japan but underscores the potential of
sebetralstat to provide meaningful relief for people who have faced
ongoing challenges with existing options. We are proud to be at the
forefront of advancing care for the HAE community.”
The NDA submission is supported by previously disclosed results,
including data from the KONFIDENT phase 3 clinical trial and
ongoing KONFIDENT-S open-label extension trial.
About the KONFIDENT Phase 3 Trial
The KONFIDENT phase 3 clinical trial was a randomized,
double-blind, 3-way crossover trial evaluating the safety and
efficacy of sebetralstat 300 mg and 600 mg versus placebo for the
on-demand treatment of HAE in adult and pediatric patients aged 12
years and older. The trial randomized 136 HAE patients from 66
clinical sites across 20 countries, making it the largest clinical
trial ever conducted in HAE. In the trial, participants treated
each eligible attack with up to two doses of study drug and treated
up to three attacks over the course of the study. The trial
included type 1 and type 2 HAE patients who had at least two
documented HAE attacks 90 days prior to randomization and also
included patients receiving long-term prophylaxis.
About the KONFIDENT-S Trial
KONFIDENT-S is an open-label extension trial with numerous
real-world elements evaluating the long-term safety and efficacy of
sebetralstat for the on-demand treatment of HAE attacks in adults
and pediatric patients aged 12 years and older with HAE Type I or
Type II. KalVista is currently transitioning ongoing participants
in the trial to a novel oral disintegrating tablet (ODT)
formulation to support a planned 2026 sNDA filing. If approved, the
ODT formulation would provide people living with HAE with an
alternative, novel option for oral, on-demand treatment.
About Sebetralstat
Sebetralstat is an investigational, novel oral plasma kallikrein
inhibitor for the treatment of hereditary angioedema (HAE). We have
filed multiple regulatory applications seeking approval of
sebetralstat as the first oral, on-demand treatment for HAE in
individuals aged 12 and older, with ongoing studies exploring its
use in children aged 2 to 11. If approved, sebetralstat has the
potential to become the foundational therapy for HAE management
worldwide.
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. All currently approved on-demand treatment options
require either intravenous or subcutaneous administration.
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. Our lead investigational product is
sebetralstat, a novel, oral, on-demand treatment for hereditary
angioedema (HAE). Sebetralstat is under regulatory review by the
U.S. FDA, with a PDUFA goal date of June 17, 2025. In addition, we
have completed Marketing Authorization Applications for
sebetralstat to the European Medicines Agency and multiple other
global regulatory authorities.
For more information about KalVista, please visit
www.kalvista.com or follow us on social media at
@KalVista and LinkedIn.
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, timing or
outcomes of communications with the FDA, our expectations about
safety and efficacy of our product candidates and timing of
clinical trials and its results, our ability to commence clinical
studies or complete ongoing clinical studies, including our
KONFIDENT-S and KONFIDENT-KID trials, and to obtain regulatory
approvals for sebetralstat and other candidates in development, the
success of any efforts to commercialize sebetralstat, the ability
of sebetralstat and other candidates in development to treat HAE or
other diseases, and the future progress and potential success of
our oral Factor XIIa program. 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.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20250121977033/en/
Media: Jenn Snyder Vice President, Corporate Affairs
(857) 356-0479 jennifer.snyder@kalvista.com Investors: Ryan
Baker Head, Investor Relations (617) 771-5001
ryan.baker@kalvista.com
KalVista Pharmaceuticals (NASDAQ:KALV)
過去 株価チャート
から 12 2024 まで 1 2025
KalVista Pharmaceuticals (NASDAQ:KALV)
過去 株価チャート
から 1 2024 まで 1 2025
