U.S. FDA Approves Takeda’s TAKHZYRO® (lanadelumab-flyo) to Prevent Hereditary Angioedema (HAE) Attacks in Children 2 Years of Age and Older

Rhea-AI Summary

Takeda has received FDA approval for the expanded use of TAKHZYRO (lanadelumab-flyo) as the first prophylaxis treatment for children aged 2 to <6 years with hereditary angioedema (HAE). This approval is based on efficacy data from the Phase 3 HELP and SPRING studies, showing a reduction in HAE attacks by 94.8% in pediatric patients. Previously, children 2 to <6 years lacked approved prophylactic options. TAKHZYRO is already available in over 60 countries and aims to address the needs of HAE patients through ongoing research and clinical programs.

Positive

• TAKHZYRO is the first approved prophylaxis treatment for children aged 2 to <6 years with HAE.
• Efficacy data show a 94.8% reduction in HAE attacks in pediatric patients during the study.
• TAKHZYRO is already available in over 60 countries, indicating a strong global presence.

Negative

• Further confirmatory studies are required to substantiate efficacy results since the SPRING study was not designed for statistical hypothesis testing.

• TAKHZYRO is the First and Only Prophylaxis Treatment Approved in the U.S. for Children 2 to <6 Years of Age With HAE^1-4
• Approval Supported by Extrapolation of Efficacy Data From the Phase 3 HELP Study With Additional Data From the Phase 3 SPRING Study in Pediatric Patients 2 to <12 Years of Age¹
• HAE is a Rare, Debilitating and Potentially Life-Threatening Condition That Causes Unpredictable and Serious Angioedema Attacks That May Occur Early in Childhood^5,6

OSAKA, Japan & CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Takeda (TSE:4502/NYSE:TAK) today announced that the U.S. Food and Drug Administration (FDA) has 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 pediatric patients 2 to <12 years of age.¹ Prior to today’s approval, the only approved routine prophylaxis treatment options for children 6 to <12 years of age required dosing every three to four days, and children with HAE 2 to <6 years of age had no approved prophylaxis treatment, making TAKHZYRO the first prophylaxis treatment for this age group.^1-5 The recommended dose is 150 mg/1 mL solution in a single-dose prefilled syringe every four weeks in patients 2 to <6 years of age and every two weeks in patients 6 to <12 years of age.¹

HAE attacks can involve serious and debilitating swelling in the abdomen, face, feet, genitals, hands and throat.^5,7 Potentially fatal upper airway angioedema has been reported in patients as young as 3 years of age.⁶ In a survey from 2017 (N=445), the average HAE diagnosis took an average of 8.4 years after symptom onset.⁸ In this study of patients with HAE, 50% experienced anxiety, 34% had difficulty with social activity and 58% reported symptoms negatively affected career advancement.⁸

“Today’s approval for TAKHZYRO in pediatric patients as young as 2 years of age brings a welcome and important addition to treatment options available for children living with HAE,” said Anthony Castaldo, president and CEO of the U.S. Hereditary Angioedema Association (HAEA).

The sBLA approval was supported by extrapolation of efficacy data from the HELP Study, a Phase 3 trial that included patients 12 to <18 years of age, and additional pharmacokinetic analyses showing similar drug exposures between adults and pediatric patients, as well as safety and pharmacodynamic data from the SPRING Study, an open-label Phase 3 trial in 21 HAE patients 2 to <12 years of age.¹ The primary objectives of the SPRING Study were the safety and pharmacokinetics of TAKHZYRO.⁹ The most common treatment-related treatment emergent adverse events in the study were injection site pain (29%), injection site erythema (14%), injection site swelling (5%), administration site pain (5%) and injection site reaction (5%).⁹ The prevention of HAE attacks was measured as a secondary objective.⁹ TAKHZYRO reduced the rate of HAE attacks in pediatric patients by a mean of 94.8% compared to baseline, from 1.84 attacks per month to 0.08 attacks during the 52-week treatment period (N=21).⁹ The majority of patients (76.2%, n=16) were attack-free with an average of 99.5% attack-free days.⁹ These efficacy results are from an open-label, non-controlled trial, and the study was not designed for statistical hypothesis testing. Further confirmatory studies are required to draw any conclusions from these data.

“Today’s approval of the expanded indication of TAKHZYRO represents a significant step forward for the HAE community as it helps some of its youngest patients who are living with the disease to have a long-term prophylaxis treatment available to them,” said Julie Kim, president, U.S. Business Unit and U.S. country head at Takeda. “Takeda is a committed leader in the rare disease space, and today’s approval underscores our confidence in TAKHZYRO, as well as our dedication to addressing the needs of HAE patients through continued research, clinical programs and real-world data collection.”

TAKHZYRO was originally approved in the U.S. in 2018 for prophylaxis to prevent attacks of HAE in adult and pediatric patients 12 years and older.¹ It is currently available in more than 60 countries around the world and is supported by a robust clinical development program, which includes one of the largest prevention studies in HAE with the longest active treatment duration.^10,11

About TAKHZYRO^® (lanadelumab-flyo) Injection

TAKHZYRO is a fully human monoclonal antibody that specifically binds and decreases plasma kallikrein activity and is indicated for prophylaxis to prevent attacks of HAE in patients 2 years of age and older. TAKHZYRO is intended for self-administration for patients 12 years of age and older, or administration by a caregiver with subcutaneous injection of solution in a single-dose prefilled syringe or in a single-dose vial. The patient or caregiver should be trained by a healthcare professional. For pediatric patients 2 to <12 years of age, TAKHZYRO should be administered by a healthcare provider or caregiver with a subcutaneous injection of solution in a single-dose prefilled syringe. Please see full prescribing information for recommended doses for each patient age group. It is not known if TAKHZYRO is safe and effective in children under 2 years of age.¹

TAKHZYRO Safety Information for United States

TAKHZYRO may cause serious side effects, including allergic reactions. Call your healthcare provider or get emergency help right away if you have any of the following symptoms:

• wheezing
• difficulty breathing
• chest tightness
• fast heartbeat
• faintness
• rash
• hives

The most common side effects seen with TAKHZYRO were injection site reactions (pain, redness, and bruising), upper respiratory infection, headache, rash, dizziness, diarrhea, and muscle aches.

These are not all the possible side effects of TAKHZYRO. For more information, ask your healthcare provider or pharmacist. You may report side effects to FDA at 1-800-FDA-1088.

TAKHZYRO has not been studied in pregnant or breastfeeding women. Talk to your healthcare provider about the risk of taking TAKHZYRO if you are pregnant, plan to be pregnant, are breastfeeding, or plan to breastfeed.

Please see full Prescribing Information, including information for patients.

About Hereditary Angioedema

Hereditary angioedema (HAE) is a rare genetic disorder that results in recurring attacks of edema – swelling – in various parts of the body, including the abdomen, face, feet, genitals, hands and throat. The swelling can be debilitating and painful.^5,7 Attacks that obstruct the airways can cause asphyxiation and are potentially life-threatening.⁷ HAE affects an estimated 1 in 50,000 people worldwide.¹² It is often under-recognized, under-diagnosed and under-treated.¹²

About Takeda

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), with expertise in immune and inflammatory diseases. 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. For more information, visit https://www.takeda.com.

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 briefing 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. No shares or other securities are being offered to the public by means of this press release. No offering of securities shall be made in the United States except pursuant to registration under the U.S. Securities Act of 1933, as amended, or an exemption therefrom. This press release is being given (together with any further information which may be provided to the recipient) on the condition that it is for use by the recipient for information purposes only (and not for the evaluation of any investment, acquisition, disposal or any other transaction). Any failure to comply with these restrictions may constitute a violation of applicable securities laws. The companies in which Takeda directly and indirectly owns investments are separate entities. In this press release, “Takeda” is sometimes used for convenience where references are made to Takeda and its subsidiaries in general. Likewise, the words “we”, “us” and “our” are also used to refer to subsidiaries in general or to those who work for them. These expressions are also used where no useful purpose is served by identifying the particular company or companies.

Forward-Looking Statements

This press release and any materials distributed in connection with this press release may contain forward-looking statements, beliefs or opinions regarding Takeda’s future business, future position and results of operations, including estimates, forecasts, targets and plans for Takeda. Without limitation, forward-looking statements often include words such as “targets”, “plans”, “believes”, “hopes”, “continues”, “expects”, “aims”, “intends”, “ensures”, “will”, “may”, “should”, “would”, “could” “anticipates”, “estimates”, “projects” or similar expressions or the negative thereof. These forward-looking statements are based on assumptions about many important factors, including the following, which could cause actual results to differ materially from those expressed or implied by the forward-looking statements: the economic circumstances surrounding Takeda’s global business, including general economic conditions in Japan and the United States; competitive pressures and developments; changes to applicable laws and regulations, including global health care reforms; challenges inherent in new product development, including uncertainty of clinical success and decisions of regulatory authorities and the timing thereof; uncertainty of commercial success for new and existing products; manufacturing difficulties or delays; fluctuations in interest and currency exchange rates; claims or concerns regarding the safety or efficacy of marketed products or product candidates; the impact of health crises, like the novel coronavirus pandemic, on Takeda and its customers and suppliers, including foreign governments in countries in which Takeda operates, or on other facets of its business; the timing and impact of post-merger integration efforts with acquired companies; the ability to divest assets that are not core to Takeda’s operations and the timing of any such divestment(s); and other factors identified in Takeda’s most recent Annual Report on Form 20-F and Takeda’s other reports filed with the U.S. Securities and Exchange Commission, available on Takeda’s website at: https://www.takeda.com/investors/sec-filings/ or at www.sec.gov. Takeda does not undertake to update any of the forward-looking statements contained in this press release or any other forward-looking statements it may make, except as required by law or stock exchange rule. Past performance is not an indicator of future results and the results or statements of Takeda in this press release may not be indicative of, and are not an estimate, forecast, guarantee or projection of Takeda’s future results.

Medical information

This press release contains information about products that may not be available in all countries, or may be available under different trademarks, for different indications, in different dosages, or in different strengths. Nothing contained herein should be considered a solicitation, promotion or advertisement for any prescription drugs including the ones under development.

References

1. TAKHZYRO^® (lanadelumab-flyo) injection. U.S. Prescribing Information.
2. HAEGARDA^® (C1 Esterase Inhibitor Subcutaneous [Human]). U.S. Prescribing Information.
3. CINRYZE^® (C1 esterase inhibitor [human]). U.S. Prescribing Information.
4. Farkas H, Martinez-Saguer I, Bork K, et al. International consensus on the diagnosis and management of pediatric patients with hereditary angioedema with C1 inhibitor deficiency. Allergy. 2017 Feb;72(2):300-313. doi:10.1111/all.13001.
5. Busse PJ, Christiansen SC, Riedl MA, et al. US HAEA Medical Advisory Board 2020 guidelines for the management of hereditary angioedema. J Allergy Clin Immunol Pract. 2021 Jan;9(1):132-150.e3. doi: 10.1016/j.jaip.2020.08.046.
6. Bork K, Hardt J, Schicketanz KH, Ressel N. Clinical studies of sudden upper airway obstruction in patients with hereditary angioedema due to C1 esterase inhibitor deficiency. Arch Intern Med. 2003 May;163(10):1229–35. doi:10.1001/archinte.163.10.1229.
7. Banerji A. Hereditary angioedema: Classification, pathogenesis, and diagnosis. Allergy Asthma Proc. 2011 Nov-Dec;32(6):403–407. doi:10.2500/aap.2011.32.3492.
8. Banerji A, Davis KH, Brown TM, et al. Patient-reported burden of hereditary angioedema: findings from a patient survey in the United States. Ann Allergy Asthma Immunol. 2020 Jun;124(6):600-607. doi:10.1016/j.anai.2020.02.018.
9. Maurer M, Lumry WR, Li HH, 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. Presented July 1-3, 2022 Prague, Czech Republic at European Academy of Allergy and Clinical Immunology Hybrid Congress 2022.
10. Takeda Pharmaceuticals. Data on File.
11. Banerji A, Bernstein JA, Johnston DT, et al; for HELP OLE Investigators. Long-term prevention of hereditary angioedema attacks with lanadelumab: the HELP OLE Study. Allergy. 2022 Mar;77(3):979-990. doi:10.1111/all.15011.
12. Longhurst HJ, Bork K. Hereditary angioedema: an update on causes, manifestations, and treatment. Br J Hosp Med. 2019 Jul;80(7):391-398. doi:10.12968/hmed.2019.80.7.391.

 

View source version on businesswire.com: https://www.businesswire.com/news/home/20230203005431/en/

U.S. Media

Erin-Marie Beals

erin-marie.beals@takeda.com

+1 781-336-9417



International Media

JC Molina

jc.molina@takeda.com



Japanese Media

Jun Saito

jun.saito@takeda.com

+81 (0) 3-3278-2325

Source: Takeda Pharmaceutical Company Limited

FAQ

What is TAKHZYRO and its approved age group?

TAKHZYRO is a prophylaxis treatment approved for preventing HAE attacks in children aged 2 to <6 years.

How effective is TAKHZYRO in reducing HAE attacks?

In clinical studies, TAKHZYRO reduced the rate of HAE attacks by 94.8% in pediatric patients.

What studies supported the approval of TAKHZYRO?

Approval was based on data from the Phase 3 HELP study and additional data from the SPRING study.

Is TAKHZYRO available in other countries?

Yes, TAKHZYRO is currently available in over 60 countries worldwide.

What are the common side effects of TAKHZYRO?

Common side effects include injection site reactions, upper respiratory infections, headache, and rash.