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TAK-743

Phase 3

Hereditary Angioedema | Small molecule | Other |Takeda Pharmaceutical Company Limited|Last Updated: Jun 26, 2023

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
UNCONTROLLEDBiomarker
Total Trials1
Total Enrollment12
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT04687137Expanded Access Program With Lanadelumab for Japanese People With Hereditary AngioedemaPHASE3 COMPLETED 12Feb 10, 2021Jun 18, 2022Jun 26, 20239 Japan
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Study Endpoints
Primary Endpoints
Number of Participants With Treatment Emergent Adverse Events (TEAEs) Including Serious TEAEs and Adverse Events of Special Interest (AESI)
From start of study drug administration (in current study) up to end of study (EOS) (Day 294)

TEAEs were defined as adverse events (AEs) with onset at the time of or following the first exposure to lanadelumab in this study, or medical conditions present prior to the start of treatment but increasing in severity or relationship at the time of or following the start of treatment. Serious TEAEs were defined as any untoward clinical manifestation of signs, symptoms, outcomes (related to IP or not) that at any dose: resulted in death, was life-threatening, required inpatient/prolongation of hospitalization, resulted in persistent/significant disability/incapacity, congenital abnormality/birth defect, important medical event. AESI were defined as investigator-reported hypersensitivity reactions, events of disordered coagulation as bleeding/hypercoagulable. Relatedness to study drug was based on Investigator's discretion. TEAEs were classified and reported as Hereditary Angioedema (HAE) attack and non-HAE attack AEs in this outcome measure.

Number of Participants With Clinically Significant Abnormalities in Clinical Laboratory Tests
From start of study drug administration (in current study) up to EOS (Day 294)

Clinical laboratory testing included clinical serum chemistry, hematology, coagulation and urinalysis. Any change in clinical laboratory abnormalities which were deemed clinically significant by the investigator were recorded as TEAEs.

Number of Participants With Clinically Significant Abnormalities in Vital Signs
From start of study drug administration (in current study) up to EOS (Day 294)

Vital signs parameters included blood pressure, heart rate, body temperature and respiratory rate. Any change in vital sign abnormalities which were deemed clinically significant by the investigator were recorded as TEAEs.

Secondary Endpoints
Time to First HAE Attack After Day 0 for the Efficacy Evaluation Period for Non-Rollover Participants
Day 0 (in current study) through Day 182
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
TAK-743EXPERIMENTAL -
Interventions
NameTypeDescription
TAK-743 300 mgDRUGTAK-743 300 mg, subcutaneous injection every 2 or 4 weeks
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Eligibility Criteria
Age Range12 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites9

Inclusion Criteria: 1. In the opinion of the Investigator, the participant is capable of understanding and complying with protocol requirements. 2. Be of Japanese descent, defined as born in Japan and having Japanese parents and Japanese maternal and paternal grandparents. 3. Male and female HAE pa...

Countries:Japan
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