CLINICAL TRIALS PROFILE FOR TAKHZYRO

All Clinical Trials for TAKHZYRO

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT04687137 ↗	Expanded Access Program With Lanadelumab for Japanese People With Hereditary Angioedema	Recruiting	Takeda	Phase 3	2021-02-10	The expanded access program allows people to gain access to an unlicensed treatment on compassionate grounds. Lanadelumab, also known as TAK-743, is a medicine to help prevent hereditary angioedema attacks. Lanadelumab is not yet licensed for use in Japan. The main aim of this study is to allow Japanese teenagers and adults with type I or type II hereditary angioedema to be treated with lanadelumab, through the expanded access program in Japan. Participants can either have taken part in the previous study SHP643-302 or can be new participants. Participants just completing study SHP643-302 who reach the criteria can automatically take part in this study. However, for new participants, the study doctor will check who can take part at the first study visit. For those who can take part, new participants will receive injections of lanadelumab just under the skin. Eventually, after training, some of these will be able to inject themselves with lanadelumab in the same way. Participants who injected themselves with lanadelumab in study SHP643-302 can continue to do so during this study. The study doctors will decide if each participant will be treated with lanadelumab every 2 weeks or every 4 weeks. Treatment with lanadelumab will continue until lanadelumab is commercially available in Japan or the sponsor (Takeda) stops the study. Participants can visit the clinic during treatment if needed. If treatment continues after 6 months, participants will visit the clinic every 12 weeks for a check-up. This will include noting any hereditary angioedema attacks and side effects from the treatment. After 7 months of treatment, the study staff will check-up with each participant every 2 weeks by telephone. After treatment has finished, participants will visit the clinic for a final-check-up 4 weeks later.
NCT04848272 ↗	Inhibition of Plasma Kallikrein as a New Therapy for Lung Injury	Not yet recruiting	St Vincent's Institute of Medical Research	Phase 1	2021-04-01	Phase 1 study investigating safety of lanadelumab administration to patients with lung injury
NCT05460325 ↗	A Study of Lanadelumab (SHP643) in Chinese Participants With Hereditary Angioedema (HAE)	Recruiting	Takeda	Phase 3	2022-06-22	The main aim of this study is to evaluate the safety of lanadelumab in Chinese participants with HAE. Participants will be treated with a subcutaneous injection of lanadelumab every two weeks for 26 weeks. During the study, each participant will make 16 visits to study clinics.

Clinical Trial Conditions for TAKHZYRO

Condition Name

Condition Name for TAKHZYRO
Intervention	Trials
Hereditary Angioedema	1
Hereditary Angioedema (HAE)	1
Lung Injury	1

Condition MeSH

Condition MeSH for TAKHZYRO
Intervention	Trials
Angioedemas, Hereditary	2
Angioedema	2
Wounds and Injuries	1
Lung Injury	1

Clinical Trial Locations for TAKHZYRO

Trials by Country

Trials by Country for TAKHZYRO
Location	Trials
Japan	8
China	4
Australia	1

Clinical Trial Progress for TAKHZYRO

Clinical Trial Phase

Clinical Trial Phase for TAKHZYRO
Clinical Trial Phase	Trials
Phase 3	2
Phase 1	1

Clinical Trial Status

Clinical Trial Status for TAKHZYRO
Clinical Trial Phase	Trials
Recruiting	2
Not yet recruiting	1

Clinical Trial Sponsors for TAKHZYRO

Sponsor Name

Sponsor Name for TAKHZYRO
Sponsor	Trials
Takeda	2
St Vincent's Institute of Medical Research	1

Sponsor Type

Sponsor Type for TAKHZYRO
Sponsor	Trials
Industry	2
Other	1

TAKHZYRO Market Analysis and Financial Projection

Last updated: February 9, 2026

What is the current status of TAKHZYRO in clinical trials?

TAKHZYRO (lanadelumab) is a monoclonal antibody developed by Dyax Corp, now part of AbbVie, for preventing hereditary angioedema (HAE) attacks. Its approval by regulatory authorities in multiple regions has marked a significant milestone. The drug's clinical development has focused on preventing HAE episodes through subcutaneous administration.

Clinical Trial Phases and Completion

• Phase 3 Trials: TAKHZYRO completed pivotal Phase 3 studies, including the HELP (Hereditary Angioedema Long-term Prophylaxis Study), which involved over 125 patients. Results showed a notable reduction in attack frequency, with a 76-88% decrease versus placebo.
• Regulatory Approvals: The FDA approved TAKHZYRO in August 2018 for HAE prophylaxis in adults. The European Medicines Agency followed with approval in July 2019, and other markets have approved the drug based on pivotal trial data.
• Ongoing Evaluations: Post-approval, trials continue to explore long-term safety, dosing schedules, and efficacy in pediatric populations.

Additional Studies and Future Trials

• AbbVie has initiated trials to examine TAKHZYRO's efficacy as an on-demand therapy.
• Trials expanding indications include prophylaxis in pediatric patients and adolescent groups.
• Long-term extension studies continue to monitor safety and attack prevention durability.

What is the current market landscape for TAKHZYRO?

The global hereditary angioedema market has experienced rapid growth driven by increased diagnosis rates and the adoption of targeted biologics. TAKHZYRO's entry has leveraged its profile as an effective subcutaneous option with a favorable dosing schedule.

Market Size and Growth

• Market Value: The HAE treatment market was valued at approximately $600 million in 2022.
• Growth Rate: Expected compound annual growth rate (CAGR) of 9.5% from 2023 to 2028.
• Key Players: Other biologics include Berinert (CSL Behring), Cinryze (Takeda), and Ruconest (Svizera Pharmaceuticals).

Regional Market Penetration

• United States: Dominates market share, accounting for roughly 50% of revenues.
• Europe: Represents around 30%; approvals lag slightly behind the US timeline.
• Asia-Pacific: Growing adoption, with estimated 10% market share as awareness and diagnosis improve.

Competitive Advantages

• Dosing: Once every two weeks, simplifying adherence.
• Efficacy: Significant reduction in attack frequency, supported by Phase 3 trial data.
• Administration: Subcutaneous injection suitable for outpatient settings.

What are the market projections for TAKHZYRO over the next five years?

Forecasts anticipate continued growth driven by new indications and geographic expansion.

Year	Projected Market Size	Expected Takphyros Revenue	Key Drivers	Assumptions
2023	$650 million	$150 million	US market growth, brand recognition	Stable approval status, no new competitors
2024	$720 million	$180 million	Expanded pediatric approvals	Moderate market penetration increases
2025	$800 million	$220 million	Entry into additional markets	Successful global expansion
2026	$880 million	$250 million	New trial approvals	Broadened indication scope
2027	$970 million	$290 million	Off-label use and increased awareness	Higher adoption rates

Market shares projection

By 2027, TAKHZYRO is expected to hold roughly 30% of the HAE prophylaxis market, competing mainly with Cinryze and BCX7353, a small-molecule oral agent currently under development.

What are the key factors influencing TAKHZYRO's market performance?

• Pricing and Reimbursement: Pricing varies by region, influencing adoption. In the US, the list price was approximately $600,000 annually as of 2022.
• Patient Access: Insurance coverage, particularly for specialty biologics, affects market penetration.
• Regulatory Environment: Expanding approvals in pediatric groups and new indications will drive growth.
• Competitive Dynamics: Introduction of oral therapies could influence TAKHZYRO's market share after 2024.

What are the main challenges faced by TAKHZYRO?

• High Cost: Affordability remains an obstacle despite efficacy.
• Patient Compliance: Need for regular injections may impact long-term adherence.
• Emerging Therapies: Oral options and gene therapies could cannibalize market share.

What is the outlook for ongoing and future pipelines?

• AbbVie is evaluating TAKHZYRO for on-demand treatment, which could double its market opportunity.
• The drug's manufacturing process, based on recombinant DNA technology, is scalable, supporting broader launches.
• Long-term safety data will further bolster confidence among prescribers and payers.

Key Takeaways

• TAKHZYRO has established itself as a leading HAE prophylactic biologic with high clinical efficacy.
• The global HAE market is projected to grow at a CAGR of approximately 9.5%, driven by increased diagnosis and expanding indications.
• Revenue projections suggest significant growth over the next five years, with TAKHZYRO capturing a sizable market share.
• Cost and competitive factors pose challenges but can be mitigated through strategic pricing and expanding approvals.
• Exploring additional indications and routes of administration will be critical for sustained growth.

FAQs

1. What regulatory approvals does TAKHZYRO have globally?
TAKHZYRO was approved by the FDA in 2018, EMA in 2019, with other regions following based on regional approval processes.

2. How does TAKHZYRO compare to other HAE therapies?
It offers less frequent dosing and subcutaneous administration, which improve patient convenience compared to IV-based therapies like Cinryze.

3. What are the main adverse effects associated with TAKHZYRO?
Common adverse effects are injection site pain, headache, and rash. Long-term safety remains favorable based on current data.

4. Is there potential for TAKHZYRO in pediatric populations?
Yes. Trials are ongoing, and regulatory submissions are anticipated as safety and efficacy data mature.

5. How will the competitive landscape evolve in the next five years?
Oral therapies and gene editing approaches are under development, potentially disrupting the biologics market segment.

---

Citations

1. [1] FDA approval of TAKHZYRO, August 2018.
2. [2] EMA approval, July 2019.
3. [3] Market analysis reports, 2022.
4. [4] Clinical trial registries, 2022.
5. [5] Pricing and reimbursement data, 2022.