Alpha 1-Antitrypsin
Regulatory milestones approvals, filings & regulatory actions · 2 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed alpha 1-antitrypsin deficiency 1 milestone | |||||
| Filed | EU (EMA) | — | alpha 1-antitrypsin deficiency | 2016-03-28 | “Submitted a Marketing Authorization Application (MAA) with the European Medicines Agency (EMA) for its proprietary inhaled AAT therapy as a treatment for AAT deficiency (AATD)” kamada.com ↗ “upon the filing of a Marketing Authorization Application (MAA) with the European Medicines Agency (EMA) for inhaled alpha-1 antitrypsin (AAT)” kamada.com ↗ “an inhaled formulation of AAT for the treatment of AAT deficiency that its MAA was submitted to the EMA” takeda.com ↗ |
| Approved Indication not stated 1 milestone | |||||
| Approved | EU (EMA) | Respreeza | — | 2015-08-20 | europa.eu ↗ |
Trials 43
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 13 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| Phase 1 | NCT02087813 | Mar 2014 → Mar 2016 | neuromyelitis optica | Stanford University | Withdrawn | No outcome recorded |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 1/28 trials | ||||||
| Phase 1/2 | NCT04722887 | Dec 2021 → Aug 2025 | 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 214 trials · 2 met primary · 2 missed | ||||||
| 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 | NCT06582329 | Jun 2025 → Dec 2026 expected | alcoholic hepatitis | Medical University Innsbruck | Recruiting | No outcome recorded |
| Phase 2 | NCT04495101 | Jul 2020 → Apr 2021 | COVID-19 | Grifols | Completed | No outcome recorded |
| Phase 2 | NCT03459040 | Aug 2018 → Aug 2020 | graft versus host disease | John Levine | Completed | Missed primary |
| 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 | NCT02001688 | Apr 2014 → Feb 2016 | alpha 1-antitrypsin deficiency | Kamada, Ltd. | Completed | Met primary |
| Phase 2 | NCT02005848 | Apr 2014 → Feb 2017 | type 1 diabetes mellitus | Kamada, Ltd. | Completed | No outcome recorded |
| Phase 2 | NCT01700036 | Jul 2013 → Oct 2016 | graft versus host disease | University of Michigan Rogel Cancer Center | Completed | No outcome recorded |
| Phase 2 | NCT01661192 | Jan 2013 → Jan 2017 | type 1 diabetes mellitus | Rabin Medical Center | Completed | No outcome recorded |
| Phase 2 | NCT01394835 | Sep 2011 → Sep 2012 | bronchiolitis obliterans syndrome | Rabin Medical Center | Unknown | No outcome recorded |
| Phase 2 | NCT00499837 | Sep 2007 → Jul 2008 | cystic fibrosis | Kamada, Ltd. | Completed | No outcome recorded |
| Phase 2 | NCT00263887 | Dec 2003 → Jan 2007 | alpha 1-antitrypsin deficiency | Grifols Therapeutics LLC | Completed | Met primary |
| Phase 2 | NCT00486837 | Dec 2003 → Jun 2004 | cystic fibrosis | Grifols Therapeutics LLC | Completed | Missed primary |
| Phase 2/36 trials · 1 met primary · 1 missed | ||||||
| Phase 2/3 | NCT02956122 | Apr 2017 → May 2018 | acute graft versus host disease | Baxalta now part of Shire | Terminated | No outcome recorded Stop: Business |
| 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 | Missed primary |
| Phase 2/3 | NCT00460096 | Mar → Sep 2007 | alpha 1-antitrypsin deficiency | Kamada, Ltd. | Completed | Met primary |
| Phase 2/3 | NCT01370018 | Dec 2006 → Feb 2007 | HIV infectious disease | Institute for Human Genetics and Biochemistry | Completed | No outcome recorded |
| Phase 35 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 | NCT02722304 | Nov 2016 → Sep 2018 | alpha 1-antitrypsin deficiency, chronic obstructive pulmonary disease | Baxalta now part of Shire | Terminated | 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 | NCT00301366 | Jun 2006 → Mar 2007 | alpha 1-antitrypsin deficiency | Grifols Therapeutics LLC | Completed | No outcome recorded |
| Phase 46 trials · 2 met primary · 1 missed | ||||||
| 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 | Missed primary |
| Phase 4 | NCT00396006 | Oct 2006 → Dec 2007 | alpha 1-antitrypsin deficiency | Baxalta now part of Shire | Completed | Met primary |
| Phase 4 | NCT00313144 | Jun 2006 → Dec 2008 | alpha 1-antitrypsin deficiency | Baxalta now part of Shire | Terminated | Met primary |
| Phase 4 | NCT00261833 | Mar 2006 → Sep 2012 | alpha 1-antitrypsin deficiency, pulmonary emphysema | CSL Behring | Completed | Mixed |
| 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 |
| Comparator or background therapy9 trials | ||||||
| Phase 1 | NCT00242385 Comparator | Dec 2005 → Jun 2006 | alpha 1-antitrypsin deficiency | Baxalta now part of Shire | Completed | No outcome recorded |
| Phase 1/2 | NCT02191839 Comparator | Jul 2014 → Jul 2015 | post-cardiac arrest syndrome | Soroka University Medical Center | Unknown | No outcome recorded |
| Phase 2 | NCT05856331 Comparator | Oct 2023 → Aug 2025 | alpha 1-antitrypsin deficiency, pulmonary emphysema | Sanofi | Completed | Met primary |
| 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 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 4 | NCT05582798 Comparator | Nov 2022 → Aug 2024 | bronchiectasis | University of Dundee | Completed | No outcome recorded |
News releases announcing trial results or a regulatory action · 67
| Date | Issuer | Release |
|---|---|---|
| 2026-05-18 | Sanofi | Results Press Release: ATS: phase 2 data demonstrate the superiority of efdoralprin alfa over a standard-of-care augmentation therapy in achieving higher fAAT levels in AATD sanofi.com ↗
In patients dosed Q3W, fAAT levels remained above the normal threshold (23.8 μM) for 100% of days during the 32-week study compared to 41% of days in patients on a standard-of-care augmentation therapy. |
| 2026-05-12 | AIRNA Corporation | Results AIRNA Presents New Preclinical Data on AATD Therapeutic Candidate AIR-001 and Cardiometabolic RNA-Editing Programs at ASGCT airna.com ↗
AIR-001 demonstrated best-in-class potential, achieving up to 59% precise RNA editing in vivo and restoring 40 µM functional M-AAT in a mouse model of AATD |
| 2026-04-16 | Frequency Therapeutics | Results Korro Announces Oral and Poster Presentations at Upcoming Scientific Conferences korrobio.com ↗
A GalNAc-conjugated RNA editing oligonucleotide achieves >90% editing of pathogenic SERPINA1 transcripts, restoring Alpha-1 Antitrypsin function in AATD mod |
| 2025-12-08 | Kamada, Ltd. | Results Kamada Announces Discontinuation of its Phase 3 Inhaled AAT Clinical Trial; Reiterates 2025 Full-Year Guidance and Projects Double-Digit Growth in Revenues and Profitability in 2026 kamada.com ↗
Based on the futility analysis outcome, the Company will discontinue the trial. |
| 2025-10-22 | Sanofi | Results Press Release: Sanofi’s efdoralprin alfa met all primary and key secondary endpoints in alpha-1 antitrypsin deficiency emphysema phase 2 study sanofi.com ↗
It demonstrated a statistically significant greater mean increase in functional AAT levels within normal range as measured by trough concentrations at steady state compared to those receiving weekly plasma-derived augmentation therapy at week 32 [p<0.0001]. |
| 2025-10-21 | Nanoform Finland Plc | Results Results of Nanoform Collaborative Study to be Presented at the 2025 Partnership Opportunities in Drug Delivery (PODD) Summit nanoform.com ↗
Nanoform Finland Plc (“Nanoform”), a leading nanoparticle medicine performance-enhancing company, today announced that key findings from two proof-of-concept studies conducted in collaboration between Nanoform and Takeda will be presented at the 2025 Partnership Opportunities in Drug Delivery (PODD) conference in Boston, Mass., which takes place on October 27-28, 2025. |
| 2025-10-01 | CRISPR Therapeutics | Results CRISPR Therapeutics to Present Preclinical Data on Alpha-1 Antitrypsin Deficiency (AATD) Utilizing Novel SyNTase Gene Editing Technology at the European Society of Gene and Cell Therapy (ESGCT) 2025 Annual Congress crisprtx.com ↗
The abstract describes that SyNTase editing produces high levels of editing (up to 95%) in SERPINA1-E342K human hepatocyte cell models without any detectable (<0.5%) off-target effects. |
| 2025-05-28 | Nanoform Finland Plc | Results Positive nanoform biologics respiratory data to be presented at drug delivery and formulation summit nanoform.com ↗
Comparison of other methods such as spray drying showed that the nanoformed particulate material could deposit significantly higher amounts of AAT within the alveolar system. |
| 2025-05-13 | AIRNA Corporation | Results AIRNA Presents Data Supporting RNA Editing Approach for Alpha-1 Antitrypsin Deficiency (AATD) at 28th ASGCT Annual Meeting airna.com ↗
AIRNA, a biotech company pioneering RNA editing therapeutics to transform the lives of patients with rare and common conditions, today announced preclinical proof-of-concept data supporting its best-in-class RNA editing strategy for alpha-1 antitrypsin deficiency (AATD). |
| 2024-05-13 | Targovax Oy | Results Circio presents pre-clinical proof-of-concept data for its circVec gene therapy platform at the ASGCT 2024 meeting circio.com ↗
By using circular RNA-based AAT expression, Circio is uniquely able to separate the two species for mutant-specific knockdown, thereby solving two problems with one single product. |
| 2023-05-16 | Kamada, Ltd. | Regulatory Kamada Announces Receipt of Marketing Authorization for Glassia® for Treatment of Alpha-1 Antitrypsin Deficiency in Switzerland kamada.com ↗
today announced that Swissmedic has granted marketing authorization for Glassia® [Alpha-1 Proteinase Inhibitor (Human)] in Switzerland for chronic augmentation and maintenance therapy in adults with clinically evident emphysema due to severe hereditary Alpha-1 antitrypsin deficiency (AATD). |
| 2022-11-07 | Kamada, Ltd. | Results Kamada Provides Update on Recent Progress Achieved in Ongoing Pivotal Phase 3 Clinical Trial of Inhaled AAT kamada.com ↗
Importantly, none of these patients discontinued treatment prematurely and no drug-related serious adverse events were reported. |
| 2022-05-02 | Beam Therapeutics Inc. | Results Beam Therapeutics to Present New Preclinical Data from Base Editing Pipeline at ASGCT 25th Annual Meeting beamtx.com ↗ |
| 2021-10-20 | Intellia Therapeutics | Results Intellia Therapeutics Presents Preclinical Data Demonstrating Advancements in its Broad Genome Editing Capabilities at the 2021 European Society of Gene & Cell Therapy Annual Congress intelliatx.com ↗
Achieved durable production of normal human alpha-1 antitrypsin protein levels and reduction of endogenous disease-associated protein in non-human primates for the treatment of liver and/or lung manifestations of alpha-1 antitrypsin deficiency (AATD) |
| 2021-10-12 | Intellia Therapeutics | Results Intellia Therapeutics to Present New Preclinical Data Highlighting In Vivo and Ex Vivo Genome Editing Advances at 2021 European Society of Gene & Cell Therapy Annual Congress intelliatx.com ↗
Additionally, we will be presenting non-human primate data from our in vivo platform where we have demonstrated our ability to both insert a functional gene and inactivate a gene to treat AATD. |
| 2021-01-22 | Grifols Therapeutics LLC | Regulatory Japan’s regulator approves Grifols’ Lynspad™ [alpha-1 proteinase inhibitor] for the treatment of alpha-1 antitrypsin deficiency grifols.com ↗
today announced that Japan’s Ministry of Health, Labour and Welfare (MHLW) has granted manufacturing and marketing approval for Lynspad™ (intravenous infusion 1000 mg), an alpha-1 proteinase inhibitor to treat severe alpha-1 antitrypsin deficiency (AATD) in Japan. |
| 2020-12-12 | Intellia Therapeutics | Results Intellia Therapeutics Achieves Normal Human Alpha-1 Antitrypsin Protein Levels in Non-Human Primates Through Targeted Gene Insertion for the Treatment of AAT Deficiency intelliatx.com ↗
Intellia Therapeutics, Inc. (NASDAQ:NTLA), is presenting the first demonstration of physiological protein levels of human alpha-1 antitrypsin (AAT) in non-human primates (NHPs) following a single administration. |
| 2020-11-09 | Arrowhead Pharmaceuticals | Results Arrowhead Pharmaceuticals to Participate in Upcoming Conferences arrowheadpharma.com ↗
ARO-AAT Reduces Serum and Intra-hepatic Z-AAT Protein in PIZZ Alpha-1 Antitrypsin Deficient Patients with Liver Disease Leading to Improvements in Clinically Relevant Liver Biomarkers |
| 2020-05-12 | Beam Therapeutics Inc. | Results Beam Therapeutics Reports Additional Data at ASGCT Annual Meeting and First Quarter 2020 Financial Results beamtx.com ↗
Alpha-1 Antitrypsin Deficiency Program Demonstrates More than Four-Fold Increase in Circulating Levels of Functional Protein Following Durable Direct Correction In Vivo |
| 2020-04-28 | Beam Therapeutics Inc. | Results Beam Therapeutics to Present First Data Highlighting Base Editing Program for Alpha-1 Antitrypsin Deficiency at 23rd ASGCT Annual Meeting beamtx.com ↗
Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced preclinical data showcasing the potential of its novel base editing approach for the treatment of alpha-1 antitrypsin deficiency (Alpha-1) liver and lung diseases. |
| 2019-10-24 | Intellia Therapeutics | Results Intellia Therapeutics Presents In Vivo and Ex Vivo Data at the 2019 Annual Congress of the European Society of Gene and Cell Therapy (ESGCT) intelliatx.com ↗
First reported consecutive in vivo gene knockout and insertion achieves therapeutically relevant results in an alpha-1 antitrypsin deficiency mouse model |
| 2019-10-19 | Vertex Pharmaceuticals Incorporated | Regulatory Vertex Announces Reimbursement of Cystic Fibrosis Medicines SYMDEKO® (tezacaftor/ivacaftor and ivacaftor) for Eligible Patients Ages 12 and Older, and ORKAMBI® (lumacaftor/ivacaftor) in Children Ages 2 to 5, With Certain CFTR Mutations in Australia vrtx.com ↗
In addition, ORKAMBI® (lumacaftor/ivacaftor) is now also reimbursed for the treatment of children with CF ages 2 to 5 who have two copies of the F508del mutation in the CFTR gene. |
| 2019-10-16 | Intellia Therapeutics | Results Intellia Therapeutics Announces Presentations at the 2019 Annual Congress of the European Society of Gene and Cell Therapy (ESGCT) intelliatx.com ↗
In Vivo Gene Knockout Followed by Targeted Gene Insertion Results in Simultaneous Reduced Mutant Protein Levels and Durable Transgene Expression |
| 2019-04-25 | CSL Behring | Regulatory CSL Behring Delivers on its Continued Commitment to Alpha 1 Patients with FDA Approval of Convenient Single-Vial Dosing for ZEMAIRA® [Alpha1-Proteinase Inhibitor (Human)] csl.com ↗
Global biotherapeutics leader CSL Behring today announced that the US Food and Drug Administration (FDA) has approved 4- and 5-gram vial sizes for ZEMAIRA® [Alpha1-Proteinase Inhibitor (Human)], its therapy for treating Alpha 1 Antitrypsin Deficiency (Alpha 1). |
| 2019-04-16 | Kamada, Ltd. | Regulatory Kamada announces FDA acceptance of Inhaled AAT program path forward kamada.com ↗ |
| 2019-02-07 | Kamada, Ltd. | Results Kamada Announces Additional Interim Results from Phase 2 Proof of Concept Clinical Trial of Intravenous Alpha-1 Antitrypsin Treatment for Prevention of Lung Transplant Rejection kamada.com ↗
Rehovot, Israel, February 6, 2019 – Kamada Ltd. (NASDAQ & TASE: KMDA), a plasma-derived protein therapeutics company focused on orphan indications, today announced interim results from the Company’s Phase 2 trial of intravenous Alpha-1 Antitrypsin (IV-AAT) for the prevention of lung transplant rejection following one year of treatment for all patients. |
| 2018-07-10 | Kamada, Ltd. | Regulatory Kamada Receives Positive Scientific Advice from European Medicines Agency on a New Phase 3 Study Design for Inhaled AAT kamada.com ↗
Kamada Ltd. (the “Company”) (NASDAQ & TASE: KMDA), a plasma-derived protein therapeutics company, today announced that it has received positive scientific advice from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) related to the development plan for its proposed pivotal Phase 3 study for its proprietary Inhaled Alpha-1 Antitrypsin therapy (Inhaled AAT) for the treatment of alpha-1 antitrypsin deficiency (AATD). |
| 2018-01-08 | Kamada, Ltd. | Results Kamada Announces Interim Results from Phase 2 Clinical Trial of Intravenous Alpha-1 Antitrypsin Treatment for Prevention of Lung Transplant Rejection kamada.com ↗
Kamada Ltd. (NASDAQ & TASE: KMDA), a plasmaderived protein therapeutics company focused on orphan indications, today announced interim results from the Company's Phase 2 trial of intravenous Alpha-1 Antitrypsin (IV AAT) for the prevention of lung transplant rejection. |
| 2017-11-01 | Kamada, Ltd. | Results Kamada Announces Top-line Results of Phase 2 Trial of Alpha-1 Antitrypsin in Newly Diagnosed Type-1 Diabetes Patients kamada.com ↗
“Given this study was not powered to show efficacy, the results are very encouraging,” |
| 2017-09-07 | CSL Behring | Results CSL Behring Presents New Alpha 1 Antitrypsin Deficiency Treatment Data at Global ERS Congress csl.com ↗
A further post hoc analysis now shows that treatment is beneficial in patients with both early and late stage disease. |
| 2017-07-20 | Kamada, Ltd. | Regulatory Kamada Submits Proposed Phase 3 Protocol to FDA for Inhaled Alpha-1-Antitrypsin for Treatment of Alpha-1 Antitrypsin Deficiency Disease kamada.com ↗ |
| 2017-06-27 | Kamada, Ltd. | Regulatory Kamada Announces Withdrawal of European Marketing Authorization Application for Inhaled Alpha-1-Antitrypsin for Treatment of Alpha-1 Antitrypsin Deficiency Disease kamada.com ↗
today announced that the Company has withdrawn the Marketing Authorization Application (MAA) for its proprietary inhaled Alpha-1 Antitrypsin (AAT) therapy for the treatment of Alpha-1 Antitrypsin Deficiency (AATD) with the European Medicines Agency (EMA). |
| 2017-05-24 | Kamada, Ltd. | Results Kamada Presents Updated Data from Phase 2 Clinical Trial of Inhaled Alpha-1-Antitrypsin for Treatment of Alpha-1 Antitrypsin Deficiency at 2017 American Thoracic Society International Conference kamada.com ↗
Previously announced top-line data from this trial indicated that patients treated with Kamada's inhaled AAT demonstrated a significant increase in endothelial lining fluid (ELF), AAT antigenic and ANEC levels |
| 2017-01-23 | Kamada, Ltd. | Regulatory Kamada Announces Positive Scientific Advice Response from the European Medicines Agency Focused on Alpha-1 Antitrypsin IV for Treatment of Acute Graft-Versus-Host Disease kamada.com ↗ |
| 2016-12-02 | CSL Behring | Results The Lancet Respiratory Medicine Publishes RAPID Extension Study Data Confirming Disease-modifying Effect of Respreeza® [Alpha1-Proteinase Inhibitor (Human)] in Patients with Alpha-1 Antitrypsin Deficiency (AATD) csl.com ↗
02 Dec 2016 Global biotherapeutics leader CSL Behring announced today that data from the RAPID Open Label Extension study, conducted in patients with alpha-1 antitrypsin deficiency (AATD), demonstrates that the use of Alpha1-Proteinase Inhibitor (A1-PI) therapy slows the progressive and irreversible loss of lung tissue, confirming that early intervention is beneficial. |
| 2016-08-30 | Kamada, Ltd. | Results Kamada Meets Primary Endpoint of U.S. Phase 2 Study of Inhaled Alpha-1 Antitrypsin for the Treatment of Alpha-1 Antitrypsin Deficiency kamada.com ↗
Kamada Ltd. (Nasdaq: KMDA) (TASE: KMDA.TA), a plasma-derived protein therapeutics company focused on orphan indications, today announced positive top-line results, meeting the primary endpoint of the Company's U.S. Phase 2 clinical trial of its proprietary inhaled Alpha-1 Antitrypsin (AAT) therapy for the treatment of Alpha-1 Antitrypsin Deficiency (AATD). |
| 2016-06-15 | Takeda | Regulatory Shire and Kamada announce FDA approval of expanded label for self-infusion of Glassia for the treatment of emphysema due to severe AAT deficiency takeda.com ↗ |
| 2016-06-15 | Kamada, Ltd. | Regulatory Shire and Kamada Announce FDA Approval of Expanded Label for Self-Infusion of Glassia for the Treatment of Emphysema Due to Severe AAT Deficiency kamada.com ↗ |
| 2016-03-28 | Kamada, Ltd. | Regulatory Kamada Submits Marketing Authorization Application with the European Medicines Agency for its Proprietary Inhaled Alpha-1 Antitrypsin to Treat Alpha-1 Antitrypsin Deficiency kamada.com ↗
The filing was validated by the EMA. |
| 2016-01-06 | Kamada, Ltd. | Results Kamada Reports Further Positive Interim Data from Phase 1/2 Study of its Alpha-1 Antitrypsin to Treat Graft Versus Host Disease kamada.com ↗
The interim results showed that plasma AAT levels increased in both cohorts and remained stable for the duration of treatment. |
| 2015-09-28 | CSL Behring | Results New Clinical Data on Treatment to Slow the Progression of Emphysema in Patients with Alpha-1 Antitrypsin Deficiency Presented at European Respiratory Society International Congress in Amsterdam csl.com ↗
Our findings, for the first time in a single randomized-controlled trial, provided additional evidence that treatment with an alpha-1 proteinase inhibitor provides a clinical benefit by slowing the accelerated loss of lung tissue. |
| 2015-07-07 | Kamada, Ltd. | Results Kamada Announces Publication in Pediatric Diabetes kamada.com ↗
No serious adverse events, diabetic ketoacidosis (DKA), or severe hypoglycemic episodes were reported. |
| 2015-06-29 | CSL Behring | Regulatory CSL Behring Receives Positive CHMP Opinion for Respreeza® as Maintenance Treatment for Severe Alpha-1 Antitrypsin Deficiency Patients in Europe csl.com ↗
The CHMP positive opinion will be transmitted to the European Commission (EC) to start the EC decision-making process. |
| 2015-06-01 | CSL Behring | Results The Lancet Publishes CSL Behring’s Alpha-1 Antitrypsin Deficiency Study Data csl.com ↗
Findings from CSL Behring’s RAPID study, the largest placebo-controlled trial ever conducted in patients with alpha-1 antitrypsin deficiency (AATD), demonstrate that the use of Alpha1-Proteinase Inhibitor therapy may slow the progressive loss of lung tissue experienced by these critically ill patients. |
| 2015-05-20 | Kamada, Ltd. | Results Kamada Reports Updated Data from European Phase 2/3 Clinical Study of Inhaled Alpha-1 Antitrypsin to Treat AAT Deficiency kamada.com ↗
In addition to previously announced results that focused on the statistically significant lung function beneficial effect, additional data on the nature of symptoms of the first exacerbation, a clinically important measurement, were reported during the panel session. |
| 2015-05-17 | Alnylam Pharmaceuticals | Regulatory Alnylam Files Clinical Trial Application (CTA) for ALN-AAT, an Investigational RNAi Therapeutic for the Treatment of Alpha-1 Antitrypsin Deficiency-Associated Liver Disease (Alpha-1 Liver Disease) alnylam.com ↗ |
| 2015-04-21 | Kamada, Ltd. | Results Kamada Reports Additional Data from European Phase 2/3 Clinical Study of Inhaled Alpha-1 Antitrypsin to Treat AAT Deficiency kamada.com ↗
Kamada Ltd. (Nasdaq and TASE: KMDA), a plasma-derived protein therapeutics company focused on orphan indications, announces additional results from its European Phase 2/3 clinical trial of the Company's inhaled alpha-1 antitrypsin (AAT) to treat AAT deficiency (AATD). |
| 2015-03-23 | Kamada, Ltd. | Regulatory Kamada Awarded European Orphan Drug Designation for its Alpha-1 Antitrypsin to Treat Graft-versus-Host Disease kamada.com ↗
Receipt of European Orphan Drug designation for our AAT to treat GvHD is a key milestone that supports our global regulatory and development strategy |
| 2015-02-02 | Kamada, Ltd. | Results Kamada Reports Encouraging Interim Data from its Phase 1/2 Extension Study: AAT in Pediatric Patients with Type 1 Diabetes kamada.com ↗
This interim report from the extension study is presented following 6 additional AAT infusions for subjects in the AAT treatment arm. |
| 2014-12-11 | Kamada, Ltd. | Results Kamada Announces Positive Interim Results from Phase I/II Clinical Study of its human Alpha-1 Antitrypsin to Treat Graft-versus-Host Disease kamada.com ↗ |
| 2014-11-10 | Arcturus Therapeutics, Inc. | Results Arcturus Therapeutics Presents Preclinical Messenger RNA Data in Non-Human Primates at the 2nd International mRNA Health Conference arcturusrx.com ↗
Delivery of chemically modified messenger RNA in LUNAR™ resulted in rapid expression of protein within 2 hours and lasting for greater than 24 hours. |
| 2014-10-29 | Kamada, Ltd. | Regulatory FDA Grants Kamada Orphan Drug Designation for the Treatment of Graft versus Host Disease kamada.com ↗
Kamada Ltd. (Nasdaq and TASE: KMDA) , a plasma-derived protein therapeutics company focused on orphan indications, announces that the U.S. Food and Drug Administration’s (FDA) Office of Orphan Products Development has granted orphan drug designation for Glassia®, the Company’s proprietary human Alpha-1 Antitrypsin (AAT), to treat Graft-versus-host-disease (GVHD). |
| 2014-10-21 | Kamada, Ltd. | Results Kamada Announces Study in Support of Alpha-1 Antitrypsin for the Treatment of Type-1 Diabetes Published in the Journal of Diabetes Science and Technology kamada.com ↗
Kamada Ltd. (Nasdaq and TASE: KMDA) , a plasma-derived protein therapeutics company focused on orphan indications, announces that a comprehensive review of the data available in the literature in support of the mechanism of action of Alpha-1 Antitrypsin (AAT) for the treatment of Type-1 Diabetes, was published in the August 2014 edition of the peer-reviewed, Journal of Diabetes Science and Technology. |
| 2014-09-04 | Kamada, Ltd. | Results Kamada Reports Final Results from Phase 2/3 Clinical Trial of Inhaled Alpha-1 Antitrypsin to Treat Alpha-1 Antitrypsin Deficiency kamada.com ↗
The Company also reports that the study did not show statistically significant differences between inhaled AAT and placebo in the secondary exacerbation endpoints measured in the ITT population. |
| 2014-05-16 | Kamada, Ltd. | Results Kamada Announces Preliminary Results from Phase II/III Pivotal Trial in Europe and Canada of Inhaled AAT to Treat Alpha-1 Antitrypsin Deficiency kamada.com ↗
A preliminary analysis of the results indicates clinically meaningful signs for inhaled AAT efficacy as well as additional positive signs in specific study populations. |
| 2014-04-24 | Kamada, Ltd. | Regulatory Kamada Announces Significantly Improved Infusion Rate for Glassia® kamada.com ↗ |
| 2014-04-05 | CSL Behring | Results CSL Behring Presents Data on RAPID Trial Extension and Supports Alpha-1 International Research and Patient Conference csl.com ↗ |
| 2013-11-02 | Alnylam Pharmaceuticals | Results Alnylam Presents New Pre-clinical Data on ALN-AAT, an RNAi Therapeutic Targeting Alpha-1-Antitrypsin (AAT) for the Treatment of AAT Deficiency Liver Disease alnylam.com ↗
New Results in Mouse Model of AAT Deficiency Demonstrate that a Subcutaneously Administered GalNAc-siRNA Conjugate Targeting AAT Reduces Levels of Mutant AAT by Greater than 90%, Attenuates Fibrosis, and Arrests Development of Liver Tumors |
| 2013-10-31 | Kamada, Ltd. | Results Kamada Reports New Data from an Extension Study of its Phase I/II Trial with Glassia in Pediatric Patients with Type 1 Diabetes kamada.com ↗ |
| 2013-07-10 | Kamada, Ltd. | Results Study demonstrating pancreatic islet xenograft survival with kamada’s AAT published in peer-reviewed publication kamada.com ↗ |
| 2013-04-03 | Kamada, Ltd. | Results Kamada published an additional interim report for its pivotal clinical trial for treatment of alpha-1 deficiency by inhalation kamada.com ↗ |
| 2012-11-17 | Kamada, Ltd. | Results Kamada reports type-1 diabetes success kamada.com ↗
Kamada presents positive results for efficacy and safety in a clinical study involving Type-1 diabetes pediatric and young adult patients treated with its AAT product |
| 2012-11-14 | Alnylam Pharmaceuticals | Results Alnylam Presents New Pre-clinical Data on RNAi Therapeutics for the Treatment of Alpha-1 Antitrypsin (AAT) Deficiency alnylam.com ↗ |
| 2012-11-04 | Kamada, Ltd. | Results Kamada Announces New Data on its Glassia Drug Supporting its Diabetes Activities and Treatment of Additional Therapeutic Indications kamada.com ↗
Based on the results, Kamada reports that its AAT protein is modulating the activity of type B lymphocytes and by that enables the avoidance of organ transplant rejection and raises the likelihood for the transplant success. |
| 2012-10-14 | Kamada, Ltd. | Regulatory Kamada receives FDA Approval to its IND for Phase II Clinical Trial of Inhaled Alpha-1 Antitrypsin Deficiency (AATD-IH) in the US kamada.com ↗ |
| 2012-04-15 | Kamada, Ltd. | Results Kamada Reports Interim Positive Results in Phase I/II Clinical Trial for D1-AAT Drug for Treatment of Juvenile Diabetes (Diabetes Type 1) kamada.com ↗
The trial found stabilization and even improvement in diabetes measures of the patients, and a high safety profile in the 20 children and adolescents tested. |
| 2012-03-10 | Kamada, Ltd. | Regulatory Kamada Receives FDA Approval for Submission of IND for Clinical Trial of AAT by Inhalation kamada.com ↗
Kamada Ltd. (TASE: KMDA) has obtained Food and Drug Administration (FDA) approval to conduct a clinical trial in the US for the company&'s next generation drug – an inhalable version of AAT (Alpha-1 Antitrypsin deficiency) for the treatment of cystic fibrosis. |
All press releases naming this drug 185 releases
| Date | Issuer | Release |
|---|---|---|
| Show all 185 releases opens the full record — a long page | ||
Evidence & citations 31 cited values
Every value below carries the sentence it was read from. 64 sources stand behind the page.
| Field | Value | Cited 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 ↗29“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 ↗ “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 ↗ |
| Known as | a1-PI | “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 20224“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 | 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-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 ↗9“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: AAT” NCT01394835 ↗ |
| 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 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 ↗ |
| 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 Inhibitor” NCT00313144 ↗ |
| 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 ↗10“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 ↗ |
| 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 ↗ |
| 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 | CR002 | “Alpha1 Proteinase Inhibitor (Human) Inhalation Solution (CR002)” NCT01347190 ↗ |
| Known as | Glasia | “Intervenous ALPHA-1 ANTITRYSIN (AAT)Glasia™ in Type 1 Diabetes Mellitus” NCT01661192 ↗ |
| Known as | Glassia | “A Randomized, Double-blind, Parallel-group, Multicenter, Pharmacokinetic Study Comparing Weekly Intravenous Administration of OctaAlpha1 (Octapharma) With a Marketed...” NCT03385395 ↗10“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 ↗ “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 “Alpha 1-Proteinase Inhibitor, Human 1 MG [Glassia]” NCT01523821 ↗ “Human, Alpha-1 Antitrypsin (AAT) [Glassia®]” NCT02005848 ↗ “Alpha-1 Antitrypsin 40mg (AAT, Glassia®)” NCT01304537 ↗ |
| Known as | human alpha-1 antitrypsin | “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 ↗ |
| Known as | human alpha-1 proteinase inhibitor | “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 ↗ |
| Known as | Kamada-AAT | “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 ↗ |
| Known as | Kamada-AAT for Inhalation | “This is a randomised , placebo controlled, double blind , multicentre, Phase II/III study evaluating the safety and efficacy of Kamada AAT for inhalation in patients with...” NCT01217671 ↗1“Daily inhalation of 80 mg/day "Kamada-AAT for Inhalation" for 104 weeks” NCT04204252 ↗ |
| Known as | Kamada-API | “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 ↗ |
| Known as | Prolastin | “Augmentation therapy with Prolastin® (Alpha₁-Proteinase Inhibitor [Human]) to increase the levels of alpha₁-PI has been used to treat individuals with AAT deficiency for over 20 years.” PMID 20920295 ↗ Sep 20103“alpha1-proteinase inhibitor, produced from the plasma of human donors (Prolastin 1000 mg, powder and solvent for solution for infusion)” NCT07125664 ↗ |
| Known as | Prolastin-C | “Patients will be randomly assigned to receive Prolastin-C 120mg/kg (n=10 patients) by weekly intravenous infusions, Prolastin-C 180mg/kg (n=10 patients) by weekly intravenous...” NCT05582798 ↗3“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 ↗ |
| Known as | rAAT | “The purpose of this randomized, double-blind, placebo-controlled study is to evaluate the short-term safety of inhaled recombinant alpha 1-antitrypsin (rAAT) in subjects with...” NCT00161707 ↗ |
| Known as | Zemaira | ClinicalTrials.gov intervention name — accepted as the source's own label NCT05856331 ↗ |
| Known as | α1-Antitrypsin | “α-1 antitrypsin (AAT) has been shown to reduce inflammatory markers, promote a semimature LPS-induced DC phenotype, facilitate Treg expansion, and protect pancreatic islets...” PMID 22634621 ↗ May 20127“α1Proteinase inhibitor (α1PI or α1antitrypsin) therapy has been the standard treatment for people with insufficient α1PI blood levels.” NCT01731691 ↗ “Efficacy and safety of inhaled α1-antitrypsin in patients with severe α1-antitrypsin deficiency and frequent exacerbations of COPD.” PMID 31467115 ↗ Nov 2019 “We conducted a multicenter proof-of-concept "preemptive" treatment trial of α-1-antitrypsin (AAT), a serine protease inhibitor” PMID 33351103 ↗ Dec 2020 “Similar to aprotinin, α1-Antitrypsin (AAT) is a 52-kDa circulating serine protease inhibitor classified as a SERPIN protein.” NCT02191839 ↗ “Long-term safety of α-1 antitrypsin therapy in children and adolescents with Type 1 diabetes.” PMID 30236025 ↗ Sep 2018 “the abundant blood protein α1proteinase inhibitor (α1PI, α1-antitrypsin, Alpha-1)” PMID 31824943 ↗ Nov 2019 “α1-Antitrypsin (AAT), a naturally abundant serine protease inhibitor” PMID 29437593 ↗ Feb 2018 |
| Known as | α1PI | “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 ↗1“the abundant blood protein α1proteinase inhibitor (α1PI, α1-antitrypsin, Alpha-1)” PMID 31824943 ↗ Nov 2019 |
| Known as | α1proteinase inhibitor | “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 20224“BACKGROUND: Purified α1 proteinase inhibitor (A1PI) slowed emphysema progression in patients with severe α1 antitrypsin deficiency in a randomised controlled trial (RAPID-RCT),...” PMID 27916480 ↗ Dec 2016 “α1Proteinase inhibitor (α1PI or α1antitrypsin) therapy has been the standard treatment for people with insufficient α1PI blood levels.” NCT01731691 ↗ “the abundant blood protein α1proteinase inhibitor (α1PI, α1-antitrypsin, Alpha-1)” PMID 31824943 ↗ Nov 2019 “α1 proteinase inhibitor (A1PI) augmentation treatment” PMID 26026936 ↗ May 2015 |
| Action | Restore | “Inhalation of AAT may restore protective levels in the lungs.” PMID 19251783 ↗ Feb 2009 |
| Modality | Protein / enzyme biologic | “Recombinant Alpha 1-Antitrypsin” NCT00157092 ↗ |
| Route | Inhaled | “administered via nebulization” NCT00157092 ↗ |