U.S. Food and Drug Administration Accepts Takeda’s Supplemental Biologics License Application for Use of TAKHZYRO® (lanadelumab-flyo) to Prevent Hereditary Angioedema (HAE) Attacks in Children 2 Years of Age and Older

Rhea-AI Summary

Takeda has announced that the FDA has accepted a supplemental Biologics License Application (sBLA) for the expanded use of TAKHZYRO (lanadelumab-flyo) to prevent hereditary angioedema (HAE) attacks in pediatric patients aged 2 to <12 years old. Currently, no approved prophylactic treatments exist for children under 6. If approved, TAKHZYRO would be the first treatment available for this demographic. The FDA has granted priority review, with a decision expected in the first half of 2023.

Positive

• Acceptance of sBLA by FDA for TAKHZYRO's expanded use.
• Potential to become the first prophylactic treatment for children under 6 years with HAE.
• Priority review granted by FDA, emphasizing the urgency for this treatment.

Negative

• None.

− If Approved, TAKHZYRO Would Be the First and Only Prophylaxis Treatment Available for HAE Patients Younger Than 6 Years of Age ^1-3

− Filing is Based on Data from Phase 3 SPRING Study in Pediatric Patients 2 to <12 Years of Age

− HAE is a Rare, Debilitating and Potentially Life-Threatening Condition that Causes Unpredictable and Serious Attacks that May Occur Very Early in Childhood ^4,5

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Takeda (TSE:4502/NYSE:TAK) today announced that the U.S. Food & Drug Administration (FDA) has accepted a supplemental Biologics License Application (sBLA) for the potential expanded use of TAKHZYRO^® (lanadelumab-flyo) for prophylaxis to prevent attacks of hereditary angioedema (HAE) in pediatric patients 2 to <12 years of age. Currently, children with HAE under the age of 6 have no approved prophylaxis treatment. If approved, TAKHZYRO could potentially become the first treatment of its kind for this population.^1-3 The FDA has granted priority review of the application and indicated a decision is expected in the first half of 2023.

"Unpredictable, debilitating and potentially life-threatening HAE swelling attacks can cause a physical and emotional toll on those living with this rare disorder; this is burdensome for young children and their caregivers,” said Cheryl Schwartz, Senior Vice President, U.S. Rare Disease Business Unit at Takeda. “If TAKHZYRO is approved for this expanded use, children as young as 2 years old would have a treatment option which has shown proven effective prevention of HAE attacks in those over 12 years of age.”

The sBLA is based on data from the SPRING study, the first and only open-label Phase 3 trial for HAE patients under the age of 12. If approved, children in the U.S. would have access to treatment for the prevention of HAE attacks, which can involve serious and severely debilitating swelling in the abdomen, face, feet, genitals, hands and throat.^4,6 Potentially fatal upper airway angioedema has been reported in patients as young as 3 years old.⁵

Currently, TAKHZYRO is approved and available in more than 30 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.

About TAKHZYRO^® (lanadelumab-flyo) Injection

TAKHZYRO is a prescription medicine used to prevent attacks of hereditary angioedema (HAE) in people 12 years of age and older. It is not known if TAKHZYRO is safe and effective in children under 12 years of age. TAKHZYRO is intended for self-administration or administration by a caregiver with subcutaneous injection of 300 mg/2 mL (150 mg/1 mL) solution in a single-dose prefilled syringe or in a single-dose vial every 2 weeks. Dosing every 4 weeks may be considered in some patients. The patient or caregiver should be trained by a healthcare professional.⁷

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, and headache.

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 the 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.^4,6 Attacks that obstruct the airways can cause asphyxiation and are potentially life-threatening.⁶ HAE affects an estimated 1 in 50,000 people worldwide.⁴ It is often under-recognized, under-diagnosed and under-treated.⁸

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). 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.

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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. HAEGARDA^® (C1 Esterase Inhibitor Subcutaneous [Human]). Product Characteristics.
2. CINRYZE^® (C1 esterase inhibitor [human]). Prescribing Information.
3. 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;72(2):300-313. doi:10.1111/all.13001.
4. 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.
5. 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;163(10):1229–35. doi: 10.1001/archinte.163.10.1229.
6. Banerji A. Hereditary angioedema: Classification, pathogenesis, and diagnosis. Allergy Asthma Proc. 2011;32:403–407.
7. TAKHZYRO^® (lanadelumab-flyo) injection. Prescribing Information.
8. Longhurst HJ, Bork K. Hereditary angioedema: an update on causes, manifestations, and treatment. Br J Hosp Med. 2019;80(7):391-398.

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Media Contacts:

U.S. Media

Erin-Marie Beals

erin-marie.beals@takeda.com

+1 781-336-9417



Ex-U.S. Media

JC Molina

juan.molina@takeda.com

Source: Takeda Pharmaceutical Company Limited

FAQ

What is the status of TAKHZYRO's FDA approval?

The FDA has accepted a supplemental Biologics License Application for TAKHZYRO, with a decision expected in the first half of 2023.

What age group is TAKHZYRO intended for after the FDA approval?

If approved, TAKHZYRO will be available for pediatric patients aged 2 to less than 12 years.

What condition does TAKHZYRO aim to treat?

TAKHZYRO is designed to prevent attacks of hereditary angioedema (HAE) in pediatric patients.

Why is the approval of TAKHZYRO significant?

It would be the first and only prophylaxis treatment available for HAE patients younger than 6 years of age.

What data supports the FDA's review of TAKHZYRO?

The sBLA is based on data from the Phase 3 SPRING study in pediatric patients aged 2 to <12 years.