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Biomarker and diagnostic research for Glanzmann thrombasthenia has been reported in the published literature.
No approved treatments are currently available for Glanzmann thrombasthenia. An additional 1 compound holds orphan drug designation.
While no drugs are FDA-approved specifically for Glanzmann thrombasthenia, some of the following designated compounds may be used off-label in clinical practice. Treatment decisions should be made in consultation with a specialist familiar with this condition.
The following drugs have received orphan drug designation from the FDA for Glanzmann thrombasthenia. Orphan designation reflects regulatory interest and does not indicate approval for treatment.
Brand Name | Generic Name | Sponsor |
|---|
Estimated prevalence: Unknown (Unknown prevalence).
6 clinical trials registered, 4 recruiting. Interventions under study include other interventions, drug therapy, medical devices, and biologic therapy. Pipeline includes 1 PHASE2, 1 PHASE1, 1 NA. Research is sponsored by a mix of industry and academic institutions.
NCT ID | Title | Phase | Sponsor | Status |
|---|---|---|---|---|
[NCT06211634](https://clinicaltrials.gov/study/NCT06211634) |
Data assembled from 5 of 12 sources · Last updated Sep 19, 2026, 1:59 AM UTC
European rare disease database
Genetic and Rare Diseases Info Center
Common questions about Glanzmann thrombasthenia
Designated
Exclusivity End |
|---|
Designation Status |
|---|
human IgG4 bispecific antibody recognizing factor VIIa and TLT-1 | human IgG4 bispecific antibody recognizing factor VIIa and TLT-1 | Hemab ApS | 2024 | — | Designated |
Gene therapy approaches for Glanzmann thrombasthenia have been reported in the published literature.
6 trials found
A Phase 1/2 Study to Investigate Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of HMB-001 in Glanzmann Thrombasthenia |
PHASE1 |
Hemab ApS |
ACTIVE_NOT_RECRUITING |
[NCT00230165](https://clinicaltrials.gov/study/NCT00230165) | The Genetics and Functional Basis of Inherited Platelet, White Blood Cell, Red Blood Cell, and Blood Clotting Disorders. | — | Rockefeller University | RECRUITING |
[NCT07136857](https://clinicaltrials.gov/study/NCT07136857) | Eptacog Beta in Glanzmann's (HeT_LFB-Strength-Study_FID531) | PHASE2 | Emory University | RECRUITING |
[NCT06204042](https://clinicaltrials.gov/study/NCT06204042) | Multinational Glanzmann Study | — | UMC Utrecht | NOT_YET_RECRUITING |
[NCT04119908](https://clinicaltrials.gov/study/NCT04119908) | Videomicroscopy for the Prediction of Bleeding in Constitutional Haemorrhagic Diseases | NA | University Hospital, Lille | RECRUITING |
83 publications have been identified in PubMed for Glanzmann thrombasthenia. Research spans Case Report / Case Series (35%), Epidemiology / Natural History (20%), and Basic Science / Preclinical (17%).
Research Type | Count | % of Total |
|---|---|---|
Patient case studies | 29 | 35% |
Disease patterns and progression | 17 | 20% |
Laboratory research | 14 | 17% |
Testing and diagnosis research | 8 | 10% |
Research summaries | 8 | 10% |
New treatment approaches | 4 | 5% |
Clinical study results | 3 | 4% |
Alasmari BG (2026). [PMID: 41798562](https://pubmed.ncbi.nlm.nih.gov/41798562/). *Cureus*. [Case Report / Case Series]
Rutten KHG (2026). [PMID: 41614558](https://pubmed.ncbi.nlm.nih.gov/41614558/). *Platelets*. [Basic Science / Preclinical]
Rutten KHG (2026). [PMID: 41403382](https://pubmed.ncbi.nlm.nih.gov/41403382/). *Haemophilia : the official journal of the World Federation of Hemophilia*. [Gene Therapy / Novel Therapeutics]
Ramaekers K (2026). [PMID: 42213640](https://pubmed.ncbi.nlm.nih.gov/42213640/). *Blood*. [Basic Science / Preclinical]
Glonnegger H (2026). [PMID: 42082146](https://pubmed.ncbi.nlm.nih.gov/42082146/). *Hamostaseologie*. [Diagnostic / Biomarker]
Casini A (2026). [PMID: 41988957](https://pubmed.ncbi.nlm.nih.gov/41988957/). *Haemophilia*. [Review / Meta-Analysis]
Mohammad Alizade T (2026). [PMID: 41591555](https://pubmed.ncbi.nlm.nih.gov/41591555/). *Annals of hematology*. [Case Report / Case Series]
Citla-Sridhar D (2026). [PMID: 42154520](https://pubmed.ncbi.nlm.nih.gov/42154520/). *J Pediatr Hematol Oncol*. [Epidemiology / Natural History]
Touré SA (2026). [PMID: 41630424](https://pubmed.ncbi.nlm.nih.gov/41630424/). *Blood coagulation & fibrinolysis : an international journal in haemostasis and thrombosis*. [Epidemiology / Natural History]
El-Sayed HA (2026). [PMID: 41728181](https://pubmed.ncbi.nlm.nih.gov/41728181/). *Indian journal of hematology & blood transfusion : an official journal of Indian Society of Hematology and Blood Transfusion*. [Diagnostic / Biomarker]
AI-curated news mentioning Glanzmann thrombasthenia
Updated Apr 10, 2026
Hemab Therapeutics has filed for a $100 million IPO to advance its development of antibody-based therapies for blood coagulation disorders. The company is Phase 3-ready for its lead candidate, sutacimig (HMB-001), targeting Glanzmann thrombasthenia and Factor VII deficiency.