Elamipretide

Overview

Elamipretide has been used in trials studying the treatment of Leber's Hereditary Optic Neuropathy.

Indication

No indication information available.

Associated Conditions

No associated conditions information available.

Clinical Trials

Title	Posted	Study ID	Phase	Status	Sponsor
ReNEW:Phase 3 Study of Efficacy, Safety & Pharmacokinetics of Subcutaneous Injections of Elamipretide in Subjects With Dry Age-Related Macular Degeneration (Dry AMD)	2024/04/18	NCT06373731	Phase 3	Recruiting	Stealth BioTherapeutics Inc.
FRDA Investigator Initiated Study (IIS) With Elamipretide	2021/12/23	NCT05168774	Phase 1	Completed	Children's Hospital of Philadelphia
Study to Evaluate Efficacy and Safety of Elamipretide in Subjects With Primary Mitochondrial Disease From Nuclear DNA Mutations (nPMD)	2021/12/17	NCT05162768	Phase 3	Active, not recruiting	Stealth BioTherapeutics Inc.
An Intermediate Size Expanded Access Protocol of Elamipretide	2020/12/30	NCT04689360	N/A	AVAILABLE	Stealth BioTherapeutics Inc.
A Trial to Evaluate Safety, Tolerability and Efficacy of Elamipretide in Subjects With Barth Syndrome	2017/04/04	NCT03098797	Phase 2	Completed	Stealth BioTherapeutics Inc.
Open-Label Extension Trial to Characterize the Long-term Safety and Tolerability of Elamipretide in Subjects With Genetically Confirmed Primary Mitochondrial Myopathy (PMM)	2016/11/29	NCT02976038	Phase 2	Terminated	Stealth BioTherapeutics Inc.
A Phase 2 Study to Evaluate the Cardiac and Renal Effects of Short Term Treatment With Elamipretide in Patients Hospitalized With Congestion Due to Heart Failure	2016/09/26	NCT02914665	Phase 2	Completed	Stealth BioTherapeutics Inc.
An Open-Label, Phase 1 Clinical Study to Evaluate the Safety and Tolerability of Subcutaneous Elamipretide in Subjects With Intermediate Age-Related Macular Degeneration	2016/07/28	NCT02848313	Phase 1	Completed	Stealth BioTherapeutics Inc.
A Study to Evaluate the Effects of 4 Weeks Treatment With Subcutaneous Elamipretide on Left Ventricular Function in Subjects With Stable Heart Failure With Preserved Ejection Fraction	2016/06/27	NCT02814097	Phase 2	Completed	Stealth BioTherapeutics Inc.
Safety, Tolerability, Efficacy of MTP-131 for Treatment of Mitochondrial Disease in Subjects From the MMPOWER Study	2016/06/20	NCT02805790	Phase 2	Completed	Stealth BioTherapeutics Inc.

FDA Drug Approvals

Approved Product	Manufacturer	NDC Code	Route	Strength	Effective Date
No FDA approvals found for this drug.

EMA Drug Approvals

Approved Product	Authorization Holder	Status	Issued Date
No EMA approvals found for this drug.

HSA Drug Approvals

Approved Product	Manufacturer	Approval Number	Dosage Form	Strength	Approval Date
No HSA approvals found for this drug.

NMPA Drug Approvals

Approved Product	Company	Approval Number	Drug Type	Dosage Form	Approval Date
No NMPA approvals found for this drug.

PPB Drug Approvals

Approved Product	Registration No.	Company	Licence No.	Strength	Registration Date
No PPB approvals found for this drug.

TGA Drug Approvals

Approved Product	ARTG ID	Sponsor	Registration Type	Status	Registration Date
No TGA approvals found for this drug.

Related News

FDA Rejects Elamipretide for Rare Mitochondrial Disease Despite Patient Improvements

a month ago

The FDA's Division of Cardiology and Nephrology rejected elamipretide's new drug application on May 29, 2025, despite reported improvements in patients with rare mitochondrial diseases.

FDA Misses Review Deadline for GSK's Nucala COPD Expansion Amid Regulatory Delays

2 months ago

• The FDA has missed its May 7 target decision date for GSK's application to expand Nucala (IL-5 blocker) for use in chronic obstructive pulmonary disease. • GSK confirmed the delay but remains optimistic, stating they are "working closely" with the FDA and "continue to expect approval" based on recent discussions with regulators. • This incident adds to a pattern of FDA regulatory delays, including missed deadlines for Novavax's COVID-19 vaccine and Stealth BioTherapeutics' elamipretide, amid reports of significant staff reductions at the agency.

FDA Budget Cuts Linked to Drug Approval Delays as Small Biotech Faces PDUFA Postponement

3 months ago

• Recent government efficiency cuts at the FDA appear to be causing delays in drug approval timelines, with 66% of healthcare professionals surveyed expressing concern about potential PDUFA date postponements. • Stealth BioTherapeutics' treatment for rare Barth syndrome, elamipretide, has experienced an unexplained decision delay, with no typical safety or manufacturing concerns cited as reasons for the postponement. • Industry experts warn that while widespread disruption isn't yet evident, continued FDA resource constraints could significantly impact clinical trial oversight and create approval backlogs lasting years.

FDA Misses Deadline for Stealth Biotherapeutics' Rare Barth Syndrome Drug Elamipretide