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Hypoplastic left heart syndrome (HLHS) refers to the abnormal development of the left-sided cardiac structures, resulting in obstruction to blood flow from the left ventricular outflow tract. In addition, the syndrome includes underdevelopment of the left ventricle, aorta, and aortic arch, as well as mitral atresia or stenosis.
Biomarker and diagnostic research for hypoplastic left heart syndrome has been reported in the published literature.
No approved treatments are currently available for hypoplastic left heart syndrome. An additional 2 compounds hold orphan drug designation.
While no drugs are FDA-approved specifically for hypoplastic left heart syndrome, 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 hypoplastic left heart syndrome. Orphan designation reflects regulatory interest and does not indicate approval for treatment.
Brand Name | Generic Name | Sponsor |
|---|
Estimated prevalence: Unknown (Unknown prevalence).
12 clinical trials registered, 4 recruiting. Interventions under study include procedural interventions, other interventions, drug therapy, and medical devices. Pipeline includes 1 PHASE3, 3 PHASE2, 1 PHASE1. Research is sponsored by a mix of industry and academic institutions.
NCT ID | Title | Phase | Sponsor | Status |
|---|---|---|---|---|
[NCT04467671](https://clinicaltrials.gov/study/NCT04467671) |
Data assembled from 5 of 12 sources · Last updated Sep 19, 2026, 6:55 PM UTC
European rare disease database
Genetic and Rare Diseases Info Center
Designated
Exclusivity End |
|---|
Designation Status |
|---|
rexlemestrocel-L | rexlemestrocel-L | Mesoblast, Inc. | 2024 | — | Designated |
Allogeneic bone marrow-derived mesenchymal stromal cells (MSCs) | Allogeneic bone marrow-derived mesenchymal stromal cells (MSCs) | Longeveron, Inc. | 2021 | — | Designated |
Gene therapy approaches for hypoplastic left heart syndrome have been reported in the published literature.
12 trials found
Two-Year Study of the Safety and Efficacy of the Second-Generation Tissue Engineered Vascular Grafts |
PHASE2 |
Nationwide Children's Hospital |
RECRUITING |
[NCT06768008](https://clinicaltrials.gov/study/NCT06768008) | An Integrated Prenatal and Postnatal Treatment Model for the Treatment of Newborns With Critical Congenital Heart Disease | — | Beijing Anzhen Hospital | RECRUITING |
[NCT06711666](https://clinicaltrials.gov/study/NCT06711666) | Prenatal Maternal Mental Health and Neurodevelopment in Congenital Heart Disease | — | Institut National de la Santé Et de la Recherche Médicale, France | RECRUITING |
[NCT04106479](https://clinicaltrials.gov/study/NCT04106479) | NIRS in Congenital Heart Defects - Correlation With Echocardiography | — | McGill University Health Centre/Research Institute of the McGill University Health Centre | RECRUITING |
175 publications have been identified in PubMed for hypoplastic left heart syndrome. Research spans Review / Meta-Analysis (25%), Epidemiology / Natural History (21%), and Clinical Trial Publication (16%).
Research Type | Count | % of Total |
|---|---|---|
Research summaries | 35 | 25% |
Disease patterns and progression | 30 | 21% |
Clinical study results | 23 | 16% |
Laboratory research | 14 | 10% |
Testing and diagnosis research | 13 | 9% |
Patient case studies | 10 | 7% |
Other research | 9 | 6% |
New treatment approaches | 6 | 4% |
Kritzmire SM (2026). [PMID: 32119463](https://pubmed.ncbi.nlm.nih.gov/32119463/). *Unknown Journal*. [Gene Therapy / Novel Therapeutics]
Bandlamudi M (2026). [PMID: 32491753](https://pubmed.ncbi.nlm.nih.gov/32491753/). *Unknown Journal*. [Clinical Trial Publication]
Vargas A (2026). [PMID: 40624190](https://pubmed.ncbi.nlm.nih.gov/40624190/). *Pediatr Cardiol*. [Diagnostic / Biomarker]
Miyagi C (2026). [PMID: 41776366](https://pubmed.ncbi.nlm.nih.gov/41776366/). *Pediatr Res*. [Review / Meta-Analysis]
Hoenig SM (2026). [PMID: 40835214](https://pubmed.ncbi.nlm.nih.gov/40835214/). *Semin Thorac Cardiovasc Surg*. [Review / Meta-Analysis]
Sana MK (2026). [PMID: 32644554](https://pubmed.ncbi.nlm.nih.gov/32644554/). *Unknown Journal*. [Epidemiology / Natural History]
Balaji A (2026). [PMID: 41649542](https://pubmed.ncbi.nlm.nih.gov/41649542/). *Pediatr Cardiol*. [Review / Meta-Analysis]
Czosek RJ (2026). [PMID: 41404994](https://pubmed.ncbi.nlm.nih.gov/41404994/). *Pacing Clin Electrophysiol*. [Review / Meta-Analysis]
Kelly B (2026). [PMID: 40404979](https://pubmed.ncbi.nlm.nih.gov/40404979/). *Pediatr Cardiol*. [Clinical Trial Publication]
Dannesbo S (2026). [PMID: 41185129](https://pubmed.ncbi.nlm.nih.gov/41185129/). *Prenat Diagn*. [Clinical Trial Publication]
AI-curated news mentioning hypoplastic left heart syndrome
Updated Sep 8, 2026
A new study identifies genetic determinants linked to early heart failure in patients with hypoplastic left heart syndrome. This research could inform future therapeutic strategies and genetic screening approaches.
A case report highlights protein-losing enteropathy in a child with hypoplastic left heart syndrome following Fontan palliation. This condition may provide insights into post-operative complications in pediatric cardiac patients.
The clinical trial enrolled 40 pediatric patients at twelve premiere infant and children’s treatment institutions across the country. ELPIS II is being conducted in collaboration with the National Heart, Lung, and Blood Institute (NHLBI) through grants from the NIH. The laromestorcel HLHS program has received three FDA designations: Orphan Drug designation, Fast Track designation and Rare Pediatric Disease ... The clinical trial enrolled 40 pediatric patients at twelve premiere infant and children’s treatment institutions across the country. ELPIS II is being conducted in collaboration with the National Heart, Lung, and Blood Institute (NHLBI) through grants from the NIH. The laromestorcel HLHS program has received three FDA designations: Orphan Drug designation, Fast Track designation and Rare Pediatric Disease designation. Longeveron announced that a constructive Type C meeting with the U.S. FDA was held to discuss the ongoing development of laromestrocel in HLHS.... (NASDAQ: LGVN), a clinical stage regenerative medicine biotechnology company developing cellular therapies for life-threatening and chronic aging-related conditions, today announced that a constructive Type C meeting with the U.S. Food and Drug Administration (FDA or Agency) was held in late March, with the FDA providing their meeting summary in late April, to discuss the ongoing development of laromestrocel (LOMECEL-B®), a proprietary, scalable, allogeneic, investigational cellular therapy currently being evaluated in a Phase 2b clinical trial (ELPIS II) for hypoplastic left heart syndrome ( Laromestrocel development programs have received five distinct and important FDA designations: for the HLHS program - Orphan Drug designation, Fast Track designation, and Rare Pediatric Disease designation; and, for the AD program - Regenerative Medicine Advanced Therapy (RMAT) designation and Fast Track designation.