Forzinity (elamipretide) / Stealth BioTherapeutics - LARVOL DELTA

Mitochondrial Quality Control in Inherited Mitochondrial Cardiomyopathy: Convergent Pathobiology and a Testable Therapeutic Framework. (PubMed, Int J Mol Sci) - "We propose testable predictions addressing progression, selective rescue, and treatment timing, together with outcomes that would challenge the hypothesis. This framework supports genotype- and stage-specific investigation without assuming that enhanced mitophagy will benefit every mitochondrial cardiomyopathy."

Journal • Review • Ataxia • Cardiomyopathy • Cardiovascular • Friedreich ataxia • Gene Therapies • Heart Failure • Movement Disorders

Elamipretide: a mitochondria-targeting drug for Barth syndrome. (PubMed, Trends Pharmacol Sci) - No abstract available

Journal

Clinical Trial in Patients With Barth Syndrome- 4TAZPower (clinicaltrials.gov) - P4 | N=48 | Recruiting | Sponsor: Stealth BioTherapeutics Inc. | Not yet recruiting ➔ Recruiting

Enrollment open • TAFAZZIN

Elamipretide in pediatric Barth syndrome: from heart failure to school return. (PubMed, Orphanet J Rare Dis) - "This case suggests that elamipretide may improve cardiac function, exercise tolerance, nutrition, and HRQoL in preschool-aged pediatric BTHS patients, potentially delaying the need for transplantation. Broader pediatric evaluation is warranted. Further strategies, such as youth-tailored hybrid cardiac rehabilitation programs, could complement pharmacological therapy and further optimize outcomes in this population."

Journal • Cardiomyopathy • Cardiovascular • Congestive Heart Failure • Heart Failure • Hematological Disorders • Metabolic Disorders • Myositis • Neutropenia • Pain • Pediatrics • Transplantation

Clinical development in primary mitochondrial diseases at a translational inflection point: Lessons, mechanisms, and emerging therapeutic strategies. (PubMed, Biomed Pharmacother) - "Four therapies have achieved regulatory authorization, including idebenone for Leber hereditary optic neuropathy, taurine for MELAS, and recent FDA approvals of doxecitine and doxribtimine (Kygevvi) for thymidine kinase 2 deficiency and elamipretide (FORZINITY) for Barth syndrome. Emerging strategies include NAD⁺ augmentation, soluble guanylate cyclase stimulation, mTOR modulation, gene therapies, and heteroplasmy-targeting approaches. Collectively, these advances mark an emerging inflection point and suggest a path toward greater regulatory success in the coming decade."

Journal • Review • Gene Therapies • Inherited Retinal Dystrophy • Leber Hereditary Optic Neuropathy • Metabolic Disorders • Ocular Inflammation • Ophthalmology • Optic Neuritis

AMCP Market Insights: Managed care considerations in Barth syndrome. (PubMed, J Manag Care Spec Pharm) - "Additionally, there are numerous elements of care to which patients with Barth syndrome and their caregivers need equitable access, which is complicated by involving multiple specialists and fragmentation. Suggested payer practices involve education, care delivery, and coverage and benefit design."

Journal • Review • CNS Disorders

Is an emerging pharmacotherapeutic era for rare mitochondrial diseases here? (PubMed, Cell Metab) - "Two FDA approvals emerged in 1 year, elamipretide (Forzinity) for Barth syndrome and deoxynucleoside therapy (Kygevvi) for TK2 deficiency, with another under review. Zink et al.1 suggest sildenafil (Viagra) could treat Leigh syndrome, highlighting drug repurposing for severe pediatric mitochondrial disease."

Journal • Metabolic Disorders • Pediatrics

4TAZPower: Ph3b/4 Trial in Patients With Barth Syndrome (clinicaltrials.gov) - P4 | N=48 | Not yet recruiting | Sponsor: Stealth BioTherapeutics Inc.

New P4 trial • TAFAZZIN

CARDIAC RECOVERY, FUNCTIONAL IMPROVEMENT, AND SCHOOL REINTEGRATION WITH ELAMIPRETIDE IN BARTH SYNDROME (ACC 2026) - "Subcutaneous elamipretide (0.5 mg/kg/day) was initiated alongside maximal conventional heart failure therapy... Elamipretide may improve cardiac function, exercise tolerance, nutrition, and HRQoL in preschool-aged pediatric BTHS patients, potentially delaying the need for transplantation. Broader pediatric evaluation is warranted. Further strategies, such as youth-tailored hybrid cardiac rehabilitation programs, could complement pharmacological therapy and further optimize outcomes in this population."

Cardiomyopathy • Cardiovascular • Congestive Heart Failure • Heart Failure • Hematological Disorders • Metabolic Disorders • Myositis • Neutropenia • Rare Diseases

2025 FDA TIDES (Peptides and Oligonucleotides) Harvest. (PubMed, Pharmaceuticals (Basel)) - "Fitusiran and donidalorsen are the first oligonucleotide therapies approved for antithrombin deficiency and hereditary angioedema, respectively, while plozasiran represents the second approved therapy for familial chylomicronemia syndrome...In 2025, elamipretide further expanded this paradigm by becoming the first disease-specific treatment approved for Barth syndrome. This review provides an overview of TIDES approved in 2025, with emphasis on their chemical structures, medical targets, modes of action, routes of administration, and associated adverse effects."

Journal • Review • Cardiovascular • Complement-mediated Rare Disorders • Familial Chylomicronemia Syndrome • Hereditary Angioedema

Elamipretide—The First Approved Treatment for Barth Syndrome: Genetic Basis, Mechanistic Rationale, and Phase 4 Confirmatory Trial (ACMG 2026) - " SPIBA-401 is a post-marketing, phase 4, randomized, double-blind, placebo-controlled trial evaluating efficacy, safety, and pharmacokinetics of once-daily subcutaneous (SC) elamipretide... Elamipretide is the first approved treatment for BTHS, addressing the fundamental mitochondrial dysfunction underlying multi-system dysfunction. By stabilizing mitochondrial membrane structure and improving bioenergetics, elamipretide has demonstrated clinically meaningful improvements in exercise capacity, muscle strength, and patient-reported fatigue. The ongoing SPIBA-401 Phase 4 confirmatory trial is designed to validate prior findings in a global cohort and define the extent of durable benefit and safety."

P4 data • Cardiomyopathy • Cardiovascular • Hematological Disorders • Metabolic Disorders • Myositis • Neutropenia

SHAPE: Study of Healthy Aging and Physical Function With Elamipretide (clinicaltrials.gov) - P2 | N=30 | Recruiting | Sponsor: David Marcinek

New P2 trial

Elamipretide: First Approval. (PubMed, Drugs) - "Elamipretide (Forzinity™) is a mitochondrial cardiolipin binder being developed by Stealth BioTherapeutics for the treatment of a range of disorders featuring mitochondrial dysfunction...Elamipretide is also under phase III clinical development for use in the treatment of dry age-related macular degeneration and mitochondrial myopathies. This article summarizes the milestones in the development of elamipretide leading to this first approval for Barth syndrome."

Journal • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Genetic Disorders • Macular Degeneration • Metabolic Disorders • Myositis • Ophthalmology • Pediatrics • Retinal Disorders

Real-world disease burden and health care resource utilization for patients with Barth syndrome. (PubMed, J Med Econ) - "With a noteworthy absence of pharmacoeconomic evidence, utilization of combined clinical case report data from the medical literature, along with registry and claims datasets, demonstrate that BTHS is a costly disease associated with high disease burden and excessive HCRU. Forthcoming treatments (e.g. elamipretide) have the potential to reduce the high disease burden/HCRU."

HEOR • Journal • Real-world evidence • Cardiomyopathy • Cardiovascular • Congestive Heart Failure • Genetic Disorders • Heart Failure • Rare Diseases • Transplantation

Elamipretide: The first cardiolipin-directed mitochondrial therapeutic for Barth syndrome approved under accelerated approval. (PubMed, Drug Discov Ther) - "As a condition of accelerated approval, a confirmatory trial is required. Elamipretide represents a promising therapy addressing an unmet medical need in BTHS and provides a foundation for future mitochondria-targeted treatments."

Clinical • Journal • Cardiomyopathy • Cardiovascular • Fatigue • Hematological Disorders • Metabolic Disorders • Myositis • Neutropenia • TAFAZZIN

FORZINITY (Elamipritide). (PubMed, Clin Ther) - No abstract available

Journal

NuPower: Study to Evaluate Efficacy and Safety of Elamipretide in Subjects With Primary Mitochondrial Disease From Nuclear DNA Mutations (nPMD) (clinicaltrials.gov) - P3 | N=102 | Completed | Sponsor: Stealth BioTherapeutics Inc. | Active, not recruiting ➔ Completed

Trial completion • Metabolic Disorders • Myositis • DNA2 • MGME1 • RRM2B

ReNEW:Phase 3 Study of Efficacy, Safety & Pharmacokinetics of Subcutaneous Injections of Elamipretide in Subjects With Dry Age-Related Macular Degeneration (Dry AMD) (clinicaltrials.gov) - P3 | N=313 | Active, not recruiting | Sponsor: Stealth BioTherapeutics Inc. | Recruiting ➔ Active, not recruiting | Trial primary completion date: Aug 2026 ➔ Aug 2027

Enrollment closed • Trial primary completion date • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders

Stealth BioTherapeutics Announces FDA Accelerated Approval of FORZINITY (elamipretide HCl), the First Therapy for Progressive and Life-limiting Ultra-rare Genetic Disease Barth Syndrome (PRNewswire) - "The approval of FORZINITY is supported by the efficacy and safety data from the TAZPOWER clinical trial."

Accelerated approval • Genetic Disorders

STEALTH BIOTHERAPEUTICS RESUBMITS NEW DRUG APPLICATION FOR ELAMIPRETIDE FOR THE TREATMENT OF BARTH SYNDROME (PRNewswire) - "At FDA's request, the resubmission also contains additional post-marketing commitments regarding a proposed post-marketing trial to confirm the clinical benefit of elamipretide for the treatment of Barth syndrome."

FDA filing • Genetic Disorders

Elamipretide in the Management of Barth Syndrome: Current Evidence and a Case Report. (PubMed, Mol Genet Metab) - "Here we describe a case of prenatally identified Barth syndrome-related severe left ventricle (LV) non-compaction cardiomyopathy, where ELAM was initiated shortly after birth for clinical heart failure and was associated with significant and sustained clinical improvement leading to an inactive status on the heart transplant list with eventual anticipated delisting. We provide a review of the current literature including the pathophysiology of Barth syndrome, the mechanism of action of ELAM, and its clinical applications."

Journal • Review • Cardiomyopathy • Cardiovascular • Congestive Heart Failure • Heart Failure • Hematological Disorders • Metabolic Disorders • Myositis • Neutropenia • Transplantation

Cardiac Pathology in a Patient with a Novel Pathogenic Variant c.703del (p.Ile235SerfsTer4) of the TAFAZZIN Gene. (PubMed, Cardiovasc Pathol) - "We describe a case of Barth syndrome harboring a novel pathogenic variant of the TAFAZZIN gene exhibiting dilated cardiomyopathy, hypertrabeculation, endocardial fibroelastosis, and prominent mitochondrial abnormality. Elamipretide was well tolerated."

Journal • Cardiomyopathy • Cardiovascular • Congestive Heart Failure • Heart Failure • Metabolic Disorders • Transplantation • TAFAZZIN

Contemporary insights into elamipretide's mitochondrial mechanism of action and therapeutic effects. (PubMed, Biomed Pharmacother) - "Elamipretide continues to show promise as a potential therapy for mitochondrial disorders. New basic science advances have improved understanding of elamipretide's MOA, enabling a better understanding of the molecular consequences of elamipretide-cardiolipin interactions."

Journal • Review • Age-related Macular Degeneration • Macular Degeneration • Metabolic Disorders • Myositis • Ophthalmology • Retinal Disorders

Initial Psychometric Evaluation of the Barth Syndrome Symptom Assessment (BTHS-SA) for Adolescents and Adults in a Phase 2 Clinical Study. (PubMed, Orphanet J Rare Dis) - P2/3 | "Though the small sample size limits strong conclusions, this analysis suggests the BTHS-SA can produce reliable scores upon which valid inferences may be drawn. The BTHS-SA may be a useful tool to evaluate treatment benefits in this underserved population."

Clinical • Journal • P2 data • Fatigue • Myositis • TAFAZZIN

Significantly Improved Cardiac Function in Neonate with Barth Syndrome on Investigational Drug Elamipretide (ISHLT 2025) - "He was discharged home on oral heart failure medications and subcutaneous elamipretide...Barth syndrome is an X-linked recessive mitochondrial disorder arising from pathogenic variants in the TAFAZZIN gene, encoding a transacylase in the inner mitochondrial that catalyzes final processing and maturation of cardiolipin, which is important for high energy-requiring tissues such as cardiac muscle. Elamipretide is an investigational drug that stabilizes cardiolipin, resulting in mitochondrial membrane stabilization.Summary Our neonate with Barth syndrome associated LVNC cardiomyopathy showed remarkable improvement in cardiac function on elamipretide, demonstrating the potential of this medication for patients with this rare syndrome."

Cardiomyopathy • Cardiovascular • Congestive Heart Failure • Heart Failure • Metabolic Disorders • TAFAZZIN