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Fazirsiran

Phase 3

Alpha1-Antitrypsin Deficiency | Small molecule | Other |Takeda Pharmaceutical Company Limited|Last Updated: May 13, 2026

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
RandomizedDouble-BlindPLACEBO_CONTROLLEDDMCBiomarker
Total Trials3
Total Enrollment241
FDA Designations
No designations recorded
Clinical Trials (3)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT06165341Study to Learn About the Safety of Fazirsiran and if it Can Help People With Alpha-1 Antitrypsin Liver Disease With Mild Liver Scarring (Fibrosis)PHASE3 RECRUITING 50Mar 1, 2024Aug 26, 2028Mar 5, 202641 United States, Austria +11
NCT05899673An Extension Study to Learn About the Long-Term Safety of Fazirsiran and if Fazirsiran Can Help People With Alpha-1 Antitrypsin Liver DiseasePHASE3 ACTIVE NOT_RECRUITING 31Aug 8, 2023May 2, 2033Oct 14, 202510 United States, Austria +3
NCT05677971Study to Check the Safety of Fazirsiran and Learn if Fazirsiran Can Help People With Liver Disease and Scarring (Fibrosis) Due to an Abnormal Version of Alpha-1 Antitrypsin ProteinPHASE3 RECRUITING 160Mar 6, 2023Dec 12, 2031May 13, 202689 United States, Australia +17
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Study Endpoints
Primary Endpoints
Number of Participants With Adverse Events (AEs) and Serious AEs (SAEs)
From start of study drug administration up to End of study (EOS) (Week 124)

An AE is any untoward medical occurrence in a clinical study participant, temporally associated with the use of the study intervention, whether or not it is considered related to the study intervention. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of the study intervention. An SAE is defined as any untoward medical occurrence that results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, congenital anomaly/birth defect, suspected transmission of any infectious agent, an important medical event. AEs and SAEs including any pulmonary AEs or SAEs indicative of worsening pulmonary condition (example, pulmonary exacerbation, respiratory infection, significant pulmonary function test decline) will be reported.

Number of Participants With Clinically Significant Change From Baseline in Pulmonary Function Parameters
From start of study drug administration up to EOS (Week 124)

Standard pulmonary function parameters will be used to study lung function. Clinical significance of pulmonary function parameters will be determined at the investigator's discretion.

Change From Baseline in Whole Lung 15th Percentile Density as Measured by Computed Tomography (CT) Lung Densitometry
Baseline up to Week 100

Change from baseline in whole lung 15th percentile density as measured by CT lung densitometry will be assessed.

Number of Participants With Clinically Significant Changes in Vital Signs
From start of study drug administration up to EOS (Week 124)

Vital signs include body temperature, respiratory rate, blood pressure (systolic and diastolic), pulse (beats per minute) and pulse oximetry. Clinical significance of vital signs will be determined at the investigator's discretion.

Number of Participants with Clinically Significant Changes in Electrocardiogram (ECG) Parameters
From start of study drug administration up to EOS (Week 124)

12-lead ECG will be evaluated. Any clinically significant change in ECG assessments will be determined at the investigator's discretion.

Number of Participants With Clinically Significant Changes in Clinical Laboratory Parameters
From start of study drug administration up to EOS (Week 124)

Laboratory parameters assessments include hematology, biochemistry including liver tests, coagulation, and urinalysis. Clinical significance of laboratory parameters will be determined at the investigator's discretion.

Number of Participants With Clinically Significant Changes From Baseline in Pulmonary Function Parameters
Baseline (current study), up to EOS (current study up to 10 years])

Standard pulmonary function parameters measured will be used to study lung function. Clinical significance of pulmonary function parameters will be determined at the investigator's discretion.

Number of Participants With Clinically Significant Changes in Laboratory Parameters
From start of study drug administration (in current study) up to EOS (current study [up to 10 years])

Laboratory parameters include hematology, biochemistry including liver tests, coagulation, and urinalysis. Clinical significance of laboratory parameters will be determined at the investigator's discretion.

Reduction From Baseline of at Least 1 Stage of Histologic Fibrosis (METAVIR Staging) in the Centrally Read Liver Biopsy at Week 106 in AATD-LD With METAVIR Stage F2 and F3 Fibrosis
Baseline, Week 106

Reduction from baseline of at least 1 stage of histologic fibrosis METAVIR staging in the centrally read liver biopsy at Week 106 in AATD-LD with METAVIR stage F2 and F3 fibrosis will be assessed.

Secondary Endpoints
Change From Baseline in Serum Z-AAT Protein Over Time to Week 106
Baseline up to Week 106
Percent Change From Baseline in Intrahepatic Liver Z-AAT Protein at Week 106
Baseline up to Week 106
Change From Baseline in Intrahepatic Z-AAT Protein Polymer Burden Assessed by Periodic Acid Schiff Plus Diastase (PAS+D) Staining in Liver Biopsy at Week 106
Baseline up to Week 106
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Study Design & Arms
AllocationRANDOMIZED
MaskingDOUBLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Fazirsiran 200 mgEXPERIMENTALParticipants will receive fazirsiran 200 milligrams (mg), injection, subcutaneously on Day 1, at Week 4 and then every 12 weeks (Q12W) for up to Week 100.
PlaceboPLACEBO_COMPARATORParticipants will receive fazirsiran matching placebo injection, subcutaneously on Day 1, at Week 4 and Q12W for up to Week 100.
FazirsiranEXPERIMENTALParticipants will receive fazirsiran 200 milligram per milliliter (mg/ml) subcutaneous (SC) injection on Day 1, at Week 4, and then every 12 weeks (Q12 W) thereafter up to Week 196.
Interventions
NameTypeDescription
Fazirsiran InjectionDRUGFazirsiran will be injected subcutaneously.
PlaceboDRUGFazirsiran matching placebo.
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Eligibility Criteria
Age Range18 Years — 75 Years
SexALL
Healthy VolunteersNo
Study Sites41

Inclusion Criteria: * In the opinion of the investigator, the participant is capable of understanding and fully complying with the protocol requirements and adhering to the protocol schedule. * The participant is able to read, understand, and complete the study questionnaires electronically per the...

Countries:United StatesAustriaBelgiumCanadaFranceGermanyItalyPolandPortugalSpainSwedenSwitzerlandUnited KingdomAustraliaBrazilCzechiaDenmarkIrelandNetherlands
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT06165341primaryCompletionDate: changed
MEDIUMMay 26, 2026NCT05677971primaryCompletionDate: changed
LOWMay 26, 2026NCT05899673primaryCompletionDate: changed
LOWMay 24, 2026NCT06165341studyFirstPostDate: changed
LOWMay 24, 2026NCT05677971studyFirstPostDate: changed
LOWMay 24, 2026NCT05899673studyFirstPostDate: changed