- If Approved, Lanadelumab Will be the First
Long-Term Prophylactic Treatment of HAE Available in the EU for
Patients Under the Age of Six.
- Positive Opinion Based on Pivotal Study SHP643-301, Evaluating the Safety Profile
and Pharmacokinetics (PK) of Lanadelumab, in Combination
with Extrapolation of Data from the Pivotal Adult and
Adolescent Study DX-2930-03.
- HAE is a Rare, Genetic Disorder Estimated to Affect About
1 in 10,000 to 1 in 50,000 People Worldwide. The Condition Results
in Recurring Attacks of Oedema (Swelling) in Various Parts of the
Body that can be Debilitating and
Painful.[1],[2],[3]
ZURICH, Sept. 22,
2023 /PRNewswire/ -- Takeda (TSE:4502) (NYSE:TAK)
today announced the European Medicines Agency's (EMA) Committee for
Medicinal Products for Human Use (CHMP) has recommended the
approval of lanadelumab (trade name TAKHZYRO®) for the routine
prevention of Hereditary Angioedema (HAE) in patients aged 2 years
and older. If approved, lanadelumab will be the first long-term
prophylactic treatment available in the EU for patients under the
age of six.[4],[5],[6] The European Commission (EC) will
consider the CHMP positive opinion and decide upon potential
marketing authorization in the coming months. Lanadelumab is
currently indicated for the routine prevention of recurrent attacks
of HAE in patients aged 12 years and
older.[7]
HAE attacks, which can involve serious and severely debilitating
swelling in the abdomen, face, feet, genitals, hands and throat,
may occur very early in childhood.[8] Potentially fatal upper
airway angioedema has been reported in patients as young as 3 years
old.[9] HAE diagnosis can take an average of 8.4 years after
symptom onset.2
"We are so pleased to achieve this positive step towards
providing the first long-term prophylactic treatment option to
prevent attacks in this vulnerable population," said Didier Relin, Head of International Regulatory
at Takeda. "We know that HAE can be a complex, debilitating
condition, and we are committed to being a champion for all
individuals living with HAE."
The positive opinion is supported by data from Phase 3 Study
SHP643-301, also known as the SPRING
study, a multicenter, open-label Phase 3 study to evaluate the
safety profile and pharmacokinetics (PK) of lanadelumab in patients
2 to <12 years of age. The safety profile was consistent with
that seen in the clinical program for patients 12 years of age and
older; there were no serious adverse events and no dropouts due to
adverse events. The study also successfully reached the secondary
objective evaluating the clinical activity/outcome of lanadelumab
in preventing hereditary angioedema (HAE) attacks as well as
characterizing the pharmacokinetics of lanadelumab in paediatric
patients 2 to <12 years of age.[10]
In February, the U.S. Food and Drug Administration (FDA)
approved the supplemental Biologics License Application (sBLA) for
the expanded use of TAKHZYRO® (lanadelumab-flyo) for prophylaxis to
prevent attacks of hereditary angioedema (HAE) in paediatric
patients 2 to <12 years of age.[11]
Notes to Editors
About HAE
Hereditary angioedema (HAE) is a rare genetic disorder that
results in recurring attacks of oedema – swelling – in various
parts of the body, including the abdomen, face, feet, genitals,
hands and throat. The swelling can be debilitating and
painful.1 Attacks that obstruct the airways can
cause asphyxiation and are potentially life
threatening.2 HAE affects an estimated 1 in 50,000
people worldwide.3 It is often under recognized,
under diagnosed and under treated.3
HAE, like so many other rare diseases, is highly complex, and
patients, their families and caregivers often undergo years of
strain trying to understand their disease, get a definitive
diagnosis and gain access to the medicines they need. At Takeda we
are a committed champion for the patients we serve. Every
individual living with HAE is unique and by listening and reacting
to their needs, we translate the insights we gain into innovative
solutions – from diagnosis to ongoing management. Advancing the
science is crucial to the way we operate and we are bold in our
mission to accelerate diagnosis and develop treatments that will
make a difference to the lives of HAE patients, their support
networks and those medical professionals who care for them.
About Lanadelumab (TAKHZYRO®)
Lanadelumab is a fully human monoclonal antibody that
specifically binds and decreases plasma kallikrein and is indicated
for routine prevention of recurrent attacks of HAE in patients aged
12 years and older. It was studied in one of the largest prevention
studies in HAE with the longest active treatment duration, and
Lanadelumab consistently demonstrated HAE attack reduction.
Lanadelumab is formulated for subcutaneous administration and has a
half-life of approximately two weeks. Lanadelumab is intended for
self-administration or administration by a caregiver once trained
by a healthcare professional.4
About Study SHP643-301 (SPRING
Study)
SHP643-301 is A Multicenter,
Open-Label Phase 3 Study to Evaluate Safety, PK, Pharmacodynamics,
And Clinical Activity/Outcomes of TAKHZYRO for Prevention Against
Acute Attacks of HAE in Pediatric Patients 2 To <12 Years of
Age. The safety profile was consistent with that seen in the
clinical program for patients 12 years of age and older; there were
no serious adverse events and no dropouts due to adverse
events. The study also successfully reached the secondary
objective evaluating the clinical activity/outcome of TAKHZYRO in
preventing hereditary angioedema (HAE) attacks as well as
characterizing the pharmacodynamics of TAKHZYRO in paediatric
patients 2 to <12 years of age.10
About Takeda Pharmaceutical Company
Takeda is a global, values-based, R&D-driven
biopharmaceutical leader headquartered in Japan, committed to discover and deliver
life-transforming treatments, guided by our commitment to patients,
our people and the planet. Takeda focuses its R&D efforts on
four therapeutic areas: Oncology, Rare Genetics and Hematology,
Neuroscience, and Gastroenterology (GI). We also make targeted
R&D investments in Plasma-Derived Therapies and Vaccines. We
are focusing on developing highly innovative medicines that
contribute to making a difference in people's lives by advancing
the frontier of new treatment options and leveraging our enhanced
collaborative R&D engine and capabilities to create a robust,
modality-diverse pipeline. Our employees are committed to improving
quality of life for patients and to working with our partners in
health care in approximately 80 countries and regions.
Important Notice
For the purposes of this notice, "press release" means this
document, any oral presentation, any question-and-answer session
and any written or oral material discussed or distributed by Takeda
Pharmaceutical Company Limited ("Takeda") regarding this release.
This press release (including any oral briefings and any
question-and-answer in connection with it) is not intended to, and
does not constitute, represent or form part of any offer,
invitation or solicitation of any offer to purchase, otherwise
acquire, subscribe for, exchange, sell or otherwise dispose of, any
securities or the solicitation of any vote or approval in any
jurisdiction.
References
[1] Maurer, M., Magerl, M., et al. (2022). The international
WAO/EAACI guideline for the management of hereditary angioedema—The
2021 revision and update. Allergy, 77(7), 1961–1990.
https://doi.org/10.1111/all.15214.
[2] Banerji, A., Davis, K. H., Brown, T. M., Hollis, K.,
Hunter, S. M., Long, J., ... & Devercelli, G. (2020).
Patient-reported burden of hereditary angioedema: findings from a
patient survey in the United States. Annals of Allergy, Asthma
& Immunology, 124(6), 600-607.
[3] Longhurst, H. J., & Bork, K. (2019). Hereditary
angioedema: an update on causes, manifestations and
treatment. British Journal of Hospital Medicine, 80(7),
391-398.
[4] HAEGARDA® (C1 Esterase Inhibitor Subcutaneous [Human]).
Product Characteristics.
[5] CINRYZE® (C1 esterase inhibitor [human]). Product
Characteristics.
[6] Farkas, H., Martinez–Saguer, I., Bork, K., Bowen, T.,
Craig, T., Frank, M., ... & Zuraw, B. (2017). International
consensus on the diagnosis and management of pediatric patients
with hereditary angioedema with C1 inhibitor
deficiency. Allergy, 72(2), 300-313.
[7] European Medicines Agency, TAKHZYRO Summary of Product
Characteristics. Available at:
https://www.ema.europa.eu/en/documents/product-information/takhzyro-epar-product-information_en.pdf.
Last Accessed: September 2023.
[8] Banerji, A., Busse, P., Christiansen, S. C., Li, H.,
Lumry, W., Davis-Lorton, M., Bernstein, J. A., Frank, M., Castaldo,
A., Long, J. F., Zuraw, B. L., & Riedl, M. (2015). Current
state of hereditary angioedema management: a patient survey.
Allergy and asthma proceedings, 36(3), 213–217.
https://doi.org/10.2500/aap.2015.36.3824
[9] Bork, K., Hardt, J., Schicketanz, K. H., & Ressel,
N. (2003). Clinical studies of sudden upper airway obstruction in
patients with hereditary angioedema due to C1 esterase inhibitor
deficiency. Archives of Internal Medicine, 163(10), 1229-1235.
[10] Maurer M, Lumry WR, Li H, et al. Efficacy and Safety
of Lanadelumab in Pediatric Patients Aged 2 to <12 years With
Hereditary Angioedema: Results From the Open-Label, Multicenter
Phase 3 SPRING Study. Abstract submitted to European Academy of
Allergy and Clinical Immunology Hybrid Congress 2022.
[11] TAKHZYRO®(lanadelumab-flyo) injection. U.S. Prescribing
Information.
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