Drugs / Alpha 1-Antitrypsin
last change Aug 2026 re-read 2 minutes ago

Alpha 1-Antitrypsin

Protein / enzyme biologic

Developed for
alpha 1-antitrypsin deficiency · type 1 diabetes mellitus · pulmonary emphysema · chronic obstructive pulmonary disease · COVID-19 · cystic fibrosis · graft versus host disease · acute graft versus host disease
+12 more · HIV infectious disease · acute myocardial infarction · alcoholic hepatitis · bronchiolitis obliterans syndrome · chronic graft versus host disease · chronic pancreatitis · eosinophilic esophagitis · neuromyelitis optica · transplant rejection · viral pneumonia · bronchiectasis · post-cardiac arrest syndrome
Investigated by
Grifols Therapeutics LLC · Kamada, Ltd. · CSL Behring · Baxalta now part of Shire · National Cancer Institute (NCI) · Arriva Pharmaceuticals, Inc.
+34 more · Grifols Japan K.K. · Immune Tolerance Network (ITN) · Institute for Human Genetics and Biochemistry · Juvenile Diabetes Research Foundation · National Heart, Lung, and Blood Institute (NHLBI) · National Institute of Allergy and Infectious Diseases (NIAID) · National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) · National Institutes of Health (NIH) · Rabin Medical Center · Shire · AIDS Community Research Initiative of America · Baxalta Innovations GmbH, now part of Shire · Blessing Corporate Services, Inc · Blood and Marrow Transplant Clinical Trials Network · Children's Hospital Medical Center, Cincinnati · Fondazione IRCCS Policlinico San Matteo di Pavia · Fred Hutchinson Cancer Center · Grifols Biologicals, LLC · Grifols, S.A. · Impatients N.V. trading as myTomorrows · John Levine · Massachusetts General Hospital · Medical University Innsbruck · Michael Campos, MD · Omni Bio Pharmaceutical, Inc. · Stanford University · Takeda Pharmaceuticals North America, Inc. · The Leukemia and Lymphoma Society · University of Alberta · University of Colorado, Denver · University of Iowa · University of Michigan Rogel Cancer Center · University of Minnesota · Virginia Commonwealth University

Regulatory milestones approvals, filings & regulatory actions · 1 recorded

MilestoneJurisdictionBrandIndicationDateSentence it was read from
Approved EU (EMA) Respreeza 2015-08-20 europa.eu

Trials 55 · a red edge is where a trial was stopped

PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 14 trials
Phase 1 NCT02087813 Mar 2014 → Mar 2016 neuromyelitis optica Stanford University Withdrawn No outcome recorded
Phase 1 NCT01347190 Apr → Aug 2011 cystic fibrosis CSL Behring Completed No outcome recorded
Phase 1 NCT01319331 Oct 2010 → Oct 2015 type 1 diabetes mellitus University of Colorado, Denver Completed No outcome recorded
Phase 1 NCT01183468 Oct 2010 → Jul 2013 type 1 diabetes mellitus National Institute of Allergy and Infectious Diseases (NIAID) Terminated No outcome recorded
Phase 1/29 trials
Phase 1/2 NCT04722887 Aug 2021 → Aug 2025 alpha 1-antitrypsin deficiency Grifols Therapeutics LLC Completed No outcome recorded
Phase 1/2 NCT02870309 Jul 2016 → Mar 2017 alpha 1-antitrypsin deficiency Grifols Therapeutics LLC Completed No outcome recorded
Phase 1/2 NCT02870348 Jul 2016 → Feb 2021 alpha 1-antitrypsin deficiency Grifols Therapeutics LLC Completed No outcome recorded
Phase 1/2 NCT02520076 Aug 2015 → Sep 2019 type 1 diabetes mellitus University of Alberta Completed No outcome recorded
Phase 1/2 NCT01936896 Dec 2013 → Jun 2014 acute myocardial infarction Virginia Commonwealth University Completed No outcome recorded
Phase 1/2 NCT01523821 Oct 2013 → Jan 2017 chronic graft versus host disease Fred Hutchinson Cancer Center Completed No outcome recorded
Phase 1/2 NCT01304537 Jun 2011 → Nov 2012 type 1 diabetes mellitus Kamada, Ltd. Completed No outcome recorded
Phase 1/2 NCT00157092 Mar → Oct 2004 alpha 1-antitrypsin deficiency Baxalta now part of Shire Completed No outcome recorded
Phase 1/2 NCT00161707 Jan → Oct 2003 alpha 1-antitrypsin deficiency Baxalta now part of Shire Completed No outcome recorded
Phase 219 trials
Phase 2 NCT07125664 Oct 2025 → Oct 2027 expected chronic obstructive pulmonary disease Fondazione IRCCS Policlinico San Matteo di Pavia Not yet recruiting No outcome recorded
Phase 2 NCT05485155 May 2025 → Oct 2026 expected eosinophilic esophagitis Children's Hospital Medical Center, Cincinnati Recruiting No outcome recorded
Phase 2 NCT06582329 Apr → Jul 2025 overdue alcoholic hepatitis Medical University Innsbruck Not yet recruiting No outcome recorded
Phase 2 NCT04547140 Jan → Dec 2021 COVID-19 Grifols Therapeutics LLC Terminated No outcome recorded
Phase 2 NCT04495101 Jul 2020 → Apr 2021 COVID-19 Grifols, S.A. Completed No outcome recorded
Phase 2 NCT03459040 Aug 2018 → Aug 2020 graft versus host disease John Levine Completed No outcome recorded
Phase 2 NCT02464878 Jan 2017 → Nov 2022 type 1 diabetes mellitus Massachusetts General Hospital Completed No outcome recorded
Phase 2 NCT02614872 Jul 2016 → Apr 2019 transplant rejection Kamada, Ltd. Completed No outcome recorded
Phase 2 NCT02005848 Apr 2014 → Feb 2017 type 1 diabetes mellitus Kamada, Ltd. Completed No outcome recorded
Phase 2 NCT02001688 Apr 2014 → Feb 2016 alpha 1-antitrypsin deficiency Kamada, Ltd. Completed No outcome recorded
Phase 2/36 trials
Phase 2/3 NCT02956122 Apr 2017 → May 2018 acute graft versus host disease Baxalta now part of Shire Terminated No outcome recorded
Phase 2/3 NCT02282527 Oct 2014 → Jan 2016 alpha 1-antitrypsin deficiency Grifols Therapeutics LLC Completed No outcome recorded
Phase 2/3 NCT01731691 Apr 2012 → Jul 2014 HIV infectious disease Institute for Human Genetics and Biochemistry Terminated No outcome recorded
Phase 2/3 NCT01217671 Dec 2009 → Nov 2014 pulmonary emphysema Kamada, Ltd. Completed No outcome recorded
Phase 2/3 NCT00460096 Mar → Sep 2007 alpha 1-antitrypsin deficiency Kamada, Ltd. Completed No outcome recorded
Phase 2/3 NCT01370018 Dec 2006 → Feb 2007 HIV infectious disease Institute for Human Genetics and Biochemistry Completed No outcome recorded
Phase 38 trials
Phase 3 NCT07555483 May 2026 → Sep 2027 expected alpha 1-antitrypsin deficiency Grifols Therapeutics LLC Recruiting No outcome recorded
Phase 3 NCT04675086 Jan → Sep 2021 COVID-19, viral pneumonia Blessing Corporate Services, Inc Withdrawn No outcome recorded
Phase 3 NCT04167514 Jan 2020 → Sep 2023 graft versus host disease CSL Behring Completed No outcome recorded
Phase 3 NCT02722304 Nov 2016 → Sep 2018 alpha 1-antitrypsin deficiency, chronic obstructive pulmonary disease Baxalta now part of Shire Terminated No outcome recorded
Phase 3 NCT02796937 Jul 2016 → Sep 2028 expected pulmonary emphysema Grifols Therapeutics LLC Enrolling by invitation No outcome recorded
Phase 3 NCT02525861 Mar 2016 → Jul 2020 alpha 1-antitrypsin deficiency Baxalta now part of Shire Completed No outcome recorded
Phase 3 NCT01983241 Nov 2013 → Aug 2026 overdue pulmonary emphysema Grifols Therapeutics LLC Active not recruiting No outcome recorded
Phase 3 NCT00301366 Jun 2006 → Mar 2007 alpha 1-antitrypsin deficiency Grifols Therapeutics LLC Completed No outcome recorded
Phase 48 trials
Phase 4 NCT07326592 Jul 2026 → Sep 2033 expected alpha 1-antitrypsin deficiency, pulmonary emphysema CSL Behring Not yet recruiting No outcome recorded
Phase 4 NCT04440488 Mar 2021 → Sep 2025 alpha 1-antitrypsin deficiency, chronic obstructive pulmonary disease Shire Withdrawn No outcome recorded
Phase 4 NCT02713997 Dec 2016 → Jul 2023 chronic pancreatitis University of Minnesota Completed No outcome recorded
Phase 4 NCT01651351 Jul 2012 → Jan 2013 alpha 1-antitrypsin deficiency Baxalta now part of Shire Completed No outcome recorded
Phase 4 NCT00670007 Apr 2008 → Sep 2014 alpha 1-antitrypsin deficiency, pulmonary emphysema CSL Behring Completed No outcome recorded
Phase 4 NCT00396006 Oct 2006 → Dec 2007 alpha 1-antitrypsin deficiency Baxalta now part of Shire Completed No outcome recorded
Phase 4 NCT00313144 Jun 2006 → Dec 2008 alpha 1-antitrypsin deficiency Baxalta now part of Shire Terminated No outcome recorded
Phase 4 NCT00261833 Mar 2006 → Sep 2012 alpha 1-antitrypsin deficiency, pulmonary emphysema CSL Behring Completed No outcome recorded
Phase not stated1 trial
NCT03172455 acute graft versus host disease, alpha 1-antitrypsin deficiency Impatients N.V. trading as myTomorrows No longer available No outcome recorded
Also used as a comparator or background therapy in 10 trials
PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 11 trial
Phase 1 NCT00242385 Comparator Dec 2005 → Jun 2006 alpha 1-antitrypsin deficiency Baxalta now part of Shire Completed No outcome recorded
Phase 1/21 trial
Phase 1/2 NCT02191839 Comparator Jul 2014 → Jul 2015 post-cardiac arrest syndrome Soroka University Medical Center Unknown No outcome recorded
Phase 24 trials
Phase 2 NCT05856331 Comparator Oct 2023 → Aug 2025 alpha 1-antitrypsin deficiency, pulmonary emphysema Sanofi Completed No outcome recorded
Phase 2 NCT03385395 Comparator Jul 2018 → Dec 2019 alpha 1-antitrypsin deficiency Octapharma Withdrawn No outcome recorded
Phase 2 NCT01213043 Comparator Nov 2010 → Jan 2012 alpha 1-antitrypsin deficiency, pulmonary emphysema Grifols Therapeutics LLC Completed No outcome recorded
Phase 2 NCT02010411 Comparator Jun → Oct 2004 cystic fibrosis University of Sherbrooke Terminated No outcome recorded
Phase 32 trials
Phase 3 NCT04204252 Comparator Nov 2019 → Dec 2028 expected alpha 1-antitrypsin deficiency Kamada, Ltd. Recruiting No outcome recorded
Phase 3 NCT00295061 Comparator May 2006 → Feb 2007 alpha 1-antitrypsin deficiency Grifols Therapeutics LLC Completed No outcome recorded
Phase 42 trials
Phase 4 NCT05466747 Comparator Jan 2024 → Apr 2027 alpha 1-antitrypsin deficiency, chronic obstructive pulmonary disease Takeda Withdrawn No outcome recorded
Phase 4 NCT05582798 Comparator Nov 2022 → Aug 2024 bronchiectasis University of Dundee Completed No outcome recorded

Press releases naming this drug 27 releases

DateIssuerRelease
2025-12-17 Sanofi Press Release: Sanofi’s efdoralprin alfa earns orphan designation in the EU for alpha-1 antitrypsin deficiency related emphysema sanofi.com
2025-12-01 Regeneron Pharmaceuticals Regeneron and Tessera Therapeutics to Jointly Develop TSRA-196, an Investigational Gene Editing Therapy for Alpha-1 Antitrypsin Deficiency (AATD) regeneron.com
2025-10-22 Sanofi Press Release: Sanofi’s efdoralprin alfa met all primary and key secondary endpoints in alpha-1 antitrypsin deficiency emphysema phase 2 study sanofi.com
2024-01-23 Sanofi Press Release: Sanofi to acquire Inhibrx, Inc., adding potential best-in- class rare disease asset for Alpha-1 Antitrypsin Deficiency to pipeline sanofi.com
2024-01-02 CSL Behring CSL Behring Demonstrates Continued Commitment to Alpha-1 Community with Addition of ZEMAIRA® [Alpha1-Proteinase Inhibitor (Human)] 4- and 5-Gram Vials csl.com
2023-01-09 Takeda Arrowhead and Takeda Announce Topline Results from SEQUOIA Phase 2 Study of Fazirsiran in Patients with Alpha-1 Antitrypsin Deficiency-Associated Liver Disease takeda.com
2022-10-11 Vertex Pharmaceuticals Incorporated Vertex Advances Program Targeting Alpha-1 Antitrypsin Deficiency vrtx.com
2022-06-27 Takeda Results from Phase 2 Study of Fazirsiran in Patients with Alpha-1 Antitrypsin Deficiency Published in New England Journal of Medicine takeda.com
2021-06-10 Vertex Pharmaceuticals Incorporated Vertex Announces Primary Endpoint Achieved in Phase 2 Study of VX-864 in Alpha-1 Antitrypsin Deficiency vrtx.com
2020-10-14 Vertex Pharmaceuticals Incorporated Vertex Provides Update on its Clinical Programs Targeting Alpha-1 Antitrypsin Deficiency vrtx.com
2020-10-08 Takeda Takeda and Arrowhead Collaborate to Co-Develop and Co-Commercialize ARO-AAT for Alpha-1 Antitrypsin-Associated Liver Disease takeda.com
2020-04-06 Alnylam Pharmaceuticals Alnylam and Dicerna Form RNAi Therapeutics Collaboration on Alpha-1 Antitrypsin Deficiency-Associated Liver Disease and Complete Cross-License Agreement for Primary Hyperoxaluria Programs alnylam.com

Evidence & citations 44 cited values

Every value below carries the sentence it was read from. 80 sources stand behind the page.

FieldValueCited text
Known as alpha-1 antitrypsin “Intravenous augmentation therapy with purified preparations of AAT (Alpha1-antitrypsin) derived from human plasma is a well consolidated specific therapeutic option to treat...” NCT07125664
30

“Phase II, Double-Blind, Placebo-Controlled Study to Explore the ELF and Plasma Concentration as Well as Safety of Inhaled Alpha 1 Antitrypsin in Alpha-1 Antitrypsin Deficiency Subjects” NCT02001688

“Alpha1-Proteinase Inhibitor (Human), AralastÔ, is a sterile, stable, lyophilized preparation of purified human alpha1-proteinase inhibitor (a1-PI), also known as alpha1-antitrypsin.” NCT04675086

“Augmentation therapy with intravenous AAT (alpha-1 antitrypsin) is the only specific therapy for individuals with pulmonary disease from AAT deficiency (AATD).” PMID 30965011 Aug 2019

“Safety and Efficacy of Inhaled Alpha-1 Antitrypsin in Preventing Bronchiolitis Obliterable Syndrome in Lung Transplant Recipients” NCT01394835

Alpha-1 Antitrypsin (A1AT) acts as an anti-inflammatory protein by inhibiting the generation of pro-inflammatory cytokines.” NCT06582329

“This clinical trial will study the safety and efficacy of using the drug Zemaira, an Alpha 1-Antitrypsin (AAT) medication” NCT01700036

“plasma-derived alpha-1 antitrypsin (AAT) inhibits the inflammatory injury in myocardial ischemia and reperfusion.” PMID 29634656 Jun 2018

“Alpha-1 MP is a stable, sterile, lyophilized preparation of human alpha1-PI, also known as alpha1-antitrypsin.” NCT02870348

Alpha-1 antitrypsin (A1AT) reduces inflammatory cytokines” NCT02713997

“Human, Alpha-1 Antitrypsin (AAT) [GLASSIA®] Treatment” NCT02614872

“a drug called alpha-1-antitrypsin (AAT)” NCT03459040

“Alpha-1 anti-trypsin (AAT)” NCT01936896

“Drug: Alpha -1 Antitrypsin” NCT01394835

Alpha-1 Antitrypsin (AAT)” NCT01217671

NCT00157092

NCT01304537

NCT02713997

NCT02464878

NCT02191839

NCT00499837

NCT01700036

NCT03459040

NCT02087813

NCT02520076

NCT00161707

NCT01661192

NCT01936896

NCT04204252

NCT02005848

NCT01319331

Known as A1AT “Alpha-1 Antitrypsin (A1AT) acts as an anti-inflammatory protein by inhibiting the generation of pro-inflammatory cytokines.” NCT06582329
1

“Alpha-1 antitrypsin (A1AT) reduces inflammatory cytokines” NCT02713997

Known as A1PI “INTRODUCTION: Augmentation therapy with plasma-derived α1-proteinase inhibitor (A1PI) products is currently the only approved disease-specific therapy for α1-antitrypsin...” PMID 36001294 Aug 2022
4

“Patients who had received either A1PI treatment (Zemaira or Respreeza; early-start group) or placebo (delayed-start group) in the RAPID-RCT trial were included in this 2-year...” PMID 27916480 Dec 2016

“Alpha1-Proteinase Inhibitor (Human), AralastÔ, is a sterile, stable, lyophilized preparation of purified human alpha1-proteinase inhibitor (a1-PI), also known as alpha1-antitrypsin.” NCT04675086

“GLASSIA (human alpha-1 proteinase inhibitor (A1PI), also known as human alpha-1 antitrypsin, Kamada-AAT or Kamada-API) is a, liquid, ready-to-use preparation of human A1PI.” NCT03172455

“α1 proteinase inhibitor (A1PI) augmentation treatment” PMID 26026936 May 2015

Known as A1PI-GLASSIA “In this study, a new, high-purity (≥90% A1PI in monomeric form), ready-to-use, liquid formulation of A1PI-GLASSIA (Kamada, Ness Ziona, Israel) was compared to PROLASTINÆ...” PMID 23822603 Jul 2013
Known as AAT “Intravenous augmentation therapy with purified preparations of AAT (Alpha1-antitrypsin) derived from human plasma is a well consolidated specific therapeutic option to treat...” NCT07125664
10

“Augmentation therapy with intravenous AAT (alpha-1 antitrypsin) is the only specific therapy for individuals with pulmonary disease from AAT deficiency (AATD).” PMID 30965011 Aug 2019

“This clinical trial will study the safety and efficacy of using the drug Zemaira, an Alpha 1-Antitrypsin (AAT) medication” NCT01700036

“plasma-derived alpha-1 antitrypsin (AAT) inhibits the inflammatory injury in myocardial ischemia and reperfusion.” PMID 29634656 Jun 2018

“Phase II Study of the Safety and Efficacy of Inhaled Alpha-1 Antitrypsin (AAT ) in Cystic Fibrosis Patients” NCT00499837

“Human, Alpha-1 Antitrypsin (AAT) [GLASSIA®] Treatment” NCT02614872

“Alpha-1 Antitrypsin 40mg (AAT, Glassia®)” NCT01304537

“low AAT (Alpha 1 Antitrypsin)” NCT06582329

AAT( Alpha 1 Antitrypsin)” NCT01661192

“Alpha-1 Antitrypsin (AAT)” NCT01217671

“Drug: AATNCT01394835

Known as Alfa1 Antitrypsin ClinicalTrials.gov intervention name — accepted as the source's own label NCT06582329
Known as Alpha-1 “the abundant blood protein α1proteinase inhibitor (α1PI, α1-antitrypsin, Alpha-1)” PMID 31824943 Nov 2019
Known as Alpha-1 antitrypsin (AAT) ClinicalTrials.gov intervention name — accepted as the source's own label NCT04167514
Known as Alpha-1 Antitrypsin (human) ClinicalTrials.gov intervention name — accepted as the source's own label NCT01669421
Known as ALPHA-1 ANTITRYSIN “Intervenous ALPHA-1 ANTITRYSIN (AAT)Glasia™ in Type 1 Diabetes Mellitus” NCT01661192
Known as Alpha-1 MP “This is a multi-center, open-label study to evaluate the long-term safety of weekly intravenous (IV) infusions of 60 mg/kg alpha1-PI (human), modified process (Alpha-1 MP) in...” NCT02870348
4

“Sequential, blinded treatment periods of Alpha-1 MP (experimental), then crossed-over to Prolastin (active comparator), followed by open-label Alpha-1 MP” NCT00295061

NCT02796937

NCT02870309

NCT01983241

Known as Alpha-proteinase inhibitor ClinicalTrials.gov intervention name — accepted as the source's own label NCT05485155
Known as alpha1 -PI [human] “Because alpha1 -PI [human] (alpha1 -PI[h]) therapy can inhibit pro-inflammatory mediators associated with β-cell destruction and reduced insulin production” PMID 33244872 Dec 2020
Known as alpha1 -PI[h] “Because alpha1 -PI [human] (alpha1 -PI[h]) therapy can inhibit pro-inflammatory mediators associated with β-cell destruction and reduced insulin production” PMID 33244872 Dec 2020
Known as Alpha1-PI ClinicalTrials.gov intervention name — accepted as the source's own label NCT02093221
Known as alpha1-proteinase inhibitor “single dose ARALAST alpha1-proteinase inhibitor 60 mg/kg” NCT00242385
9

Alpha1-Proteinase Inhibitor Subcutaneous (Human), 15%” NCT07555483

“ARALAST Alpha1-Proteinase InhibitorNCT00313144

NCT04675086

NCT00261833

NCT05582798

NCT00396006

NCT01370018

NCT07125664

NCT01651351

Known as alpha1-proteinase inhibitor (human) “A Multi-center, Randomized, Double-blind, Crossover Study to Assess the Safety and Pharmacokinetics of Liquid Alpha₁-Proteinase Inhibitor (Human) Compared to Prolastin®-C in...” NCT02282527
11

Alpha1-Proteinase Inhibitor (Human), AralastÔ, is a sterile, stable, lyophilized preparation of purified human alpha1-proteinase inhibitor (a1-PI), also known as alpha1-antitrypsin.” NCT04675086

“Biochemical efficacy and safety of a new, ready-to-use, liquid alpha-1-proteinase inhibitor, GLASSIA (alpha1-proteinase inhibitor (human), intravenous).” PMID 23822603 Jul 2013

Alpha1-Proteinase Inhibitor (Human), 15%, Subcutaneous infusion” NCT04722887

“Alpha1 Proteinase Inhibitor (Human) Inhalation Solution (CR002)” NCT01347190

“Alpha 1-Proteinase Inhibitor Human” NCT01183455

NCT01523821

NCT00263887

NCT00486837

NCT00301366

NCT00295061

NCT00670007

Known as Alpha1-proteinase Inhibitor (Human), Modified Process “Alpha-1 MP (Alpha1-proteinase Inhibitor (Human), Modified Process)” NCT02870309
Known as Alpha1-Proteinase Inhibitor, Modified Process Alpha1-Proteinase Inhibitor, Modified Process (Alpha-1 MP) is used for augmentation therapy” PMID 30416054 Nov 2018
Known as Another Available A1PI ClinicalTrials.gov intervention name — accepted as the source's own label NCT05466747
Known as Aralast “The Effect of Augmentation Therapy With ARALAST Fraction IV-1 (ARALAST) Alpha1-Proteinase Inhibitor (α1-PI) on the Level of α1-PI and Other Analytes in the Bronchoalveolar...” NCT00396006
2

“single dose ARALAST alpha1-proteinase inhibitor 60 mg/kg” NCT00242385

NCT00313144

Known as ARALAST Fr. IV-1 “ARALAST Fraction IV-1 Alpha1-Proteinase Inhibitor (ARALAST Fr. IV-1)” NCT00242385
Known as ARALAST NP ClinicalTrials.gov intervention name — accepted as the source's own label NCT02722304
3

NCT04440488

NCT01183468

NCT01183455

Known as AralastÔ “Alpha1-Proteinase Inhibitor (Human), AralastÔ, is a sterile, stable, lyophilized preparation of purified human alpha1-proteinase inhibitor (a1-PI), also known as alpha1-antitrypsin.” NCT04675086
Known as CE1226 ClinicalTrials.gov intervention name — accepted as the source's own label NCT07326592