- Donidalorsen will be a first-in-class RNA-targeted medicine
for hereditary angioedema, assuming approval
- Donidalorsen PDUFA date set for August 21, 2025
- Donidalorsen has the potential to be Ionis' second
independent commercial launch
CARLSBAD, Calif., Nov. 4, 2024
/PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq:
IONS) announced today that the U.S. Food and Drug Administration
(FDA) has accepted for review the New Drug Application (NDA) for
donidalorsen, an investigational RNA-targeted medicine for
prophylaxis to prevent attacks of hereditary angioedema (HAE) in
adult and pediatric patients 12 years of age and older. The FDA has
set an action date of August 21, 2025
under the Prescription Drug User Fee Act (PDUFA). The FDA
application was based on positive results with monthly and
bi-monthly dosing in the pivotal Phase 3 OASIS-HAE and OASISplus
(open label extension (OLE) and switch) studies, as well as the
ongoing Phase 2 OLE study.
HAE is a rare and potentially life-threatening genetic condition
that involves recurrent attacks of severe swelling (angioedema) in
various parts of the body, including the hands, feet, genitals,
stomach, face and/or throat. Donidalorsen is designed to
reduce the production of prekallikrein (PKK), interrupting the
pathway that leads to HAE attacks.
"Despite currently available treatments, many people living with
HAE continue to experience painful and potentially life-threatening
breakthrough attacks. Based on the totality of clinical evidence
from the Phase 3 OASIS-HAE and OASISplus studies, as well as new
three-year results from our Phase 2 OLE study, we believe that
donidalorsen has the potential to advance the prophylactic
treatment paradigm for people living with HAE," said Brett Monia, Ph.D., chief executive officer of
Ionis. "With the FDA acceptance of our donidalorsen NDA, we are
poised for our second independent launch next year, assuming
approval, which will allow us to continue to deliver on our goal to
bring a steady cadence of medicines to patients with serious
diseases."
The FDA previously granted donidalorsen Orphan Drug Designation
in 2023. Otsuka, which has exclusive rights to commercialize
donidalorsen in Europe and Asia
Pacific, is preparing to submit a Marketing Authorization
Application (MAA) to the European Medicines Agency (EMA) this
year.
Ionis recently presented new results from the Phase 3 and Phase
2 OLE studies at the 2024 American College of Allergy, Asthma &
Immunology (ACAAI) Annual Scientific Meeting, which demonstrate
that donidalorsen delivered significant and sustained reductions in
HAE attacks, with overall sustained mean reduction in HAE attack
rates of 96% from baseline maintained up to three years in the
ongoing Phase 2 OLE study.
Across all three studies, donidalorsen was well-tolerated, with
no serious treatment-emergent adverse events (TEAEs) related to
donidalorsen. Most adverse events (AEs) were mild or moderate in
severity, and injection site reactions were the most common
AE.
The donidalorsen ACAAI e-poster presentations can be found on
Ionis' website.
About Hereditary Angioedema (HAE)
HAE is a rare and
potentially life-threatening genetic condition that involves
recurrent attacks of severe swelling (angioedema) in various parts
of the body, including the hands, feet, genitals, stomach, face
and/or throat. HAE is estimated to affect more than 20,000 patients
in the U.S. and Europe. In the
U.S., doctors frequently use prophylactic treatment approaches to
prevent and reduce the severity of HAE attacks in
patients.
About Donidalorsen
Donidalorsen is an
investigational RNA-targeted medicine designed to target
prekallikrein (PKK), which plays an important role in activating
inflammatory mediators associated with acute attacks of hereditary
angioedema (HAE). By reducing the production of PKK, donidalorsen
could be an effective prophylactic approach to preventing HAE
attacks, if approved.
Donidalorsen is an investigational medicine that has not been
approved for the treatment of any disease by regulatory
authorities.
About Ionis Pharmaceuticals, Inc.
For three
decades, Ionis has invented medicines that bring better futures to
people with serious diseases. Ionis currently has five marketed
medicines and a leading pipeline in neurology, cardiology, and
other areas of high patient need. As the pioneer in RNA-targeted
medicines, Ionis continues to drive innovation in RNA therapies in
addition to advancing new approaches in gene editing. A deep
understanding of disease biology and industry-leading technology
propels our work, coupled with a passion and urgency to deliver
life-changing advances for patients. To learn more about Ionis,
visit Ionis.com and follow us on X
(Twitter) and LinkedIn.
Ionis Forward-looking
Statements
This press release includes forward-looking
statements regarding Ionis' business and the therapeutic and
commercial potential of our commercial medicines, donidalorsen,
additional medicines in development and technologies. Any statement
describing Ionis' goals, expectations, financial or other
projections, intentions or beliefs is a forward-looking statement
and should be considered an at-risk statement. Such statements are
subject to certain risks and uncertainties including those inherent
in the process of discovering, developing and commercializing
medicines that are safe and effective for use as human
therapeutics, and in the endeavor of building a business around
such medicines. Ionis' forward-looking statements also involve
assumptions that, if they never materialize or prove correct, could
cause its results to differ materially from those expressed or
implied by such forward-looking statements. Although Ionis'
forward-looking statements reflect the good faith judgment of its
management, these statements are based only on facts and factors
currently known by Ionis. Except as required by law, we undertake
no obligation to update any forward-looking statements for any
reason. As a result, you are cautioned not to rely on these
forward-looking statements. These and other risks concerning Ionis'
programs are described in additional detail in Ionis' annual report
on Form 10-K for the year ended December 31,
2023, and most recent Form 10-Q, which are on file with the
Securities and Exchange Commission. Copies of these and other
documents are available from the Company.
In this press release, unless the context requires otherwise,
"Ionis," "Company," "we," "our" and "us" all refer to Ionis
Pharmaceuticals and its subsidiaries.
Ionis Pharmaceuticals® is a registered trademark
of Ionis Pharmaceuticals, Inc.
Ionis Pharmaceuticals Investor Contact:
D.
Wade Walke, Ph.D.
– IR@ionis.com – 760-603-2331
Ionis Pharmaceuticals Media Contact:
Hayley
Soffer – media@ionis.com – 760-603-4679
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SOURCE Ionis Pharmaceuticals, Inc.