Alpha1-antitrypsin Deficiency

8
Pipeline Programs
3
Companies
12
Clinical Trials
0
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
0
5
1
0
1
1
Early DiscoveryClinical DevelopmentMarket

Competitive Landscape

3 companies ranked by most advanced pipeline stage

Takeda
TakedaTOKYO, Japan
4 programs
2
1
1
Alpha1-proteinase inhibitorPhase 45 trials
Fazirsiran InjectionPhase 31 trial
Aerosolized, Recombinant Alpha 1-AntitrypsinPhase 1/21 trial
Aerosolized, Recombinant Alpha 1-AntitrypsinPhase 1/21 trial
Active Trials
NCT00157092Completed15Est. Oct 2004
NCT00161707Completed40Est. Oct 2003
NCT05899673Active Not Recruiting31Est. May 2033
+5 more trials
VP
1 program
1
VX-864Phase 21 trial
Active Trials
NCT04474197Completed44Est. May 2021
Grifols
GrifolsNEW YORK, NY
3 programs
3
Alpha-1 15%Phase 1/21 trial
Alpha-1 MPPhase 1/21 trial
Alpha-1 MPPhase 1/21 trial
Active Trials
NCT04722887CompletedEst. Aug 2025
NCT02870309CompletedEst. Mar 2017
NCT02870348CompletedEst. Feb 2021

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
2031
TakedaAlpha1-proteinase inhibitor
TakedaAlpha1-proteinase inhibitor
TakedaAlpha1-proteinase inhibitor
TakedaAlpha1-proteinase inhibitor
TakedaFazirsiran Injection
Vertex PharmaceuticalsVX-864
GrifolsAlpha-1 15%
GrifolsAlpha-1 MP
GrifolsAlpha-1 MP
TakedaAerosolized, Recombinant Alpha 1-Antitrypsin
TakedaAerosolized, Recombinant Alpha 1-Antitrypsin
TakedaAlpha1-proteinase inhibitor

Clinical Trials (12)

Total enrollment: 333 patients across 12 trials

NCT05466747TakedaAlpha1-proteinase inhibitor

A Study of RYMPHYSIA for Alpha1-Proteinase Inhibitor (A1PI) Therapy in Adults With A1PI Deficiency and Chronic Obstructive Pulmonary Disease (COPD)-Emphysema

Start: Jan 2024Est. completion: May 20290
Phase 4Withdrawn
NCT01651351TakedaAlpha1-proteinase inhibitor

GLASSIA Infusion Rate Study

Start: Jul 2012Est. completion: Jan 201330 patients
Phase 4Completed
NCT00396006TakedaAlpha1-proteinase inhibitor

Efficacy and Safety Study of Augmentation Therapy With ARALAST Fraction IV-1 (Human Alpha 1 - Proteinase Inhibitor)

Start: Oct 2006Est. completion: Dec 200721 patients
Phase 4Completed
NCT00313144TakedaAlpha1-proteinase inhibitor

Aralast alpha1-proteinase Inhibitor Surveillance Study

Start: Jun 2006Est. completion: May 2009127 patients
Phase 4Terminated
NCT05899673TakedaFazirsiran Injection

An Extension Study to Learn About the Long-Term Safety of Fazirsiran and if Fazirsiran Can Help People With Alpha-1 Antitrypsin Liver Disease

Start: Aug 2023Est. completion: May 203331 patients
Phase 3Active Not Recruiting

Evaluation of the Efficacy and Safety of VX-864 in Subjects With the PiZZ Genotype

Start: Jul 2020Est. completion: May 202144 patients
Phase 2Completed

A Study to Evaluate Safety, Tolerability and Pharmacokinetics of Two Different Doses of Alpha1-Proteinase Inhibitor Subcutaneous (Human) 15% in Participants With Alpha1-Antitrypsin Deficiency

Start: Aug 2021Est. completion: Aug 2025
Phase 1/2Completed

Safety and Pharmacokinetics of Alpha-1 MP (Alpha1-proteinase Inhibitor (Human), Modified Process) in Participants With Alpha1-Antitrypsin Deficiency

Start: Jul 2016Est. completion: Mar 2017
Phase 1/2Completed

Long-term Safety of Alpha1-Proteinase Inhibitor (Human) in Japanese Subjects With Alpha1 Antitrypsin Deficiency (GTI1401-OLE)

Start: Jul 2016Est. completion: Feb 2021
Phase 1/2Completed
NCT00157092TakedaAerosolized, Recombinant Alpha 1-Antitrypsin

Study of the Effect of Aerosolized, Recombinant Alpha 1-Antitrypsin on Epithelial Lining Fluid Analytes in Subjects With Alpha 1-Antitrypsin Deficiency

Start: Mar 2004Est. completion: Oct 200415 patients
Phase 1/2Completed
NCT00161707TakedaAerosolized, Recombinant Alpha 1-Antitrypsin

Safety Study of an Aerosolized, Recombinant Alpha 1-Antitrypsin in Subjects With Alpha 1-Antitrypsin Deficiency

Start: Jan 2003Est. completion: Oct 200340 patients
Phase 1/2Completed
NCT00242385TakedaAlpha1-proteinase inhibitor

Pharmacokinetic Study of ARALAST (Human Alpha1- PI)

Start: Dec 2005Est. completion: Jun 200625 patients
Phase 1Completed

Phase Legend

PreclinicalLab & animal studies
Phase 1Safety & dosing
Phase 2Efficacy testing
Phase 3Large-scale trials
On MarketApproved & available

Key Insights

1 late-stage (Phase 3) programs — potential near-term approvals
3 companies competing in this space