MedPath

FDA Accepts Elamipretide NDA for Barth Syndrome Treatment

• The FDA has accepted Stealth BioTherapeutics' NDA for elamipretide in treating Barth syndrome, a rare genetic condition with no approved therapies. • The application is supported by Phase 3 data and additional efficacy/safety data, with the FDA planning an advisory committee meeting for review. • Elamipretide, a mitochondrial-targeted therapeutic, has received Fast Track, Orphan Drug, and Rare Pediatric Disease designations for Barth syndrome. • If approved, elamipretide would be the first marketing authorization for this drug and offer a new treatment option for Barth syndrome patients.

Stealth BioTherapeutics has announced that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for elamipretide for the treatment of Barth syndrome. This decision marks a significant step forward for a condition with no currently approved therapies.
The NDA is supported by positive data from the SPIBA-001 Phase 3 Natural History Control Study, along with additional efficacy and safety data from the TAZPOWER Part 2 baseline-controlled trial. Elamipretide has previously been granted Fast Track Designation in 2017, Orphan Drug Designation in 2018, and Rare Pediatric Disease Designation in 2020.

Advisory Committee Meeting Planned

The FDA has indicated plans to hold an advisory committee meeting to discuss the application, a decision welcomed by Stealth BioTherapeutics. Reenie McCarthy, Chief Executive Officer, stated the company looks forward to the committee's input on the seriousness of Barth syndrome and the urgency of the unmet need.

Elamipretide: A Potential First-in-Class Therapy

Elamipretide is a first-in-class mitochondrial-targeted therapeutic. If approved, it would be the first marketing authorization for this drug. It is also in development for primary mitochondrial myopathy, with pivotal data from the fully-enrolled Phase 3 NuPOWER trial expected in late 2024.

About Barth Syndrome

Barth syndrome is an ultra-rare genetic condition primarily affecting males, estimated to impact one in 1,000,000 males worldwide, or about 150 individuals in the United States. It is characterized by cardiac abnormalities, muscle weakness, debilitating fatigue, heart failure, recurrent infections, and delayed growth. The disease is associated with reduced life expectancy, with 85% of early deaths occurring by age 5. Currently, there are no FDA- or EMA-approved therapies for Barth syndrome.
Subscribe Icon

Stay Updated with Our Daily Newsletter

Get the latest pharmaceutical insights, research highlights, and industry updates delivered to your inbox every day.

Related Topics

FDA Expands Setmelanotide Approval to Include Children Aged 2-5 with Rare Obesity Forms

The FDA has expanded the approval of setmelanotide (Imcivree) to include children aged 2 to 5 years with rare genetic forms of obesity.
The expanded indication is based on the VENTURE phase 3 trial, which demonstrated significant BMI reduction in young children with POMC, LEPR deficiencies, and Bardet-Biedl syndrome.

Solid Biosciences' SGT-212 Receives FDA Fast Track Designation for Friedreich's Ataxia Treatment

Solid Biosciences' SGT-212, a dual-route gene therapy for Friedreich's ataxia (FA), has received Fast Track designation from the FDA, expediting its development and review process.
SGT-212 delivers the frataxin gene via intravenous and direct intradentate nucleus infusions, targeting both neurological and cardiac manifestations of FA.

FDA Grants Fast Track and Rare Pediatric Disease Designations to Gildeuretinol for Stargardt Disease

The FDA has granted rare pediatric disease and fast track designations to gildeuretinol, an oral therapy being developed by Alkeus Pharmaceuticals for Stargardt disease.
Clinical data from the TEASE-1 study showed gildeuretinol slowed the growth rate of atrophic retinal lesions by 21.6% compared to placebo over 24 months.

FDA Raises Doubts About Stealth BioTherapeutics' Elamipretide for Barth Syndrome

The FDA has expressed significant concerns regarding the effectiveness of Stealth BioTherapeutics' elamipretide for treating Barth syndrome.
According to the FDA, the drug failed to meet its primary endpoints in the pivotal TAZPOWER trial, raising questions about its efficacy.

FDA Advisory Committee Backs Elamipretide for Barth Syndrome Despite Data Concerns

The FDA's Cardiovascular and Renal Drugs Advisory Committee voted 10-6 in favor of elamipretide for treating Barth syndrome, a rare genetic disorder.
The decision was made despite concerns about the strength and design of the clinical trial data presented by Stealth BioTherapeutics.

FDA Advisory Committee to Review Elamipretide for Barth Syndrome

The FDA's Cardiovascular and Renal Drugs Advisory Committee will review elamipretide for Barth syndrome on October 10, 2024.
Elamipretide, a first-in-class mitochondria-targeted therapeutic, is under review as a potential treatment for this ultra-rare genetic condition.

FDA Grants Priority Review for Setmelanotide sNDA in Young Children with Rare Genetic Obesity

The FDA has accepted Rhythm Pharmaceuticals' sNDA for setmelanotide, seeking approval for children aged 2-6 with obesity due to Bardet-Biedl syndrome or POMC/LEPR deficiency.
The sNDA is supported by Phase 3 trial data showing a 3.04 mean reduction in BMI-Z score and an 18.4% mean reduction in BMI with setmelanotide treatment.

KalVista Submits NDA to FDA for Oral Sebetralstat in Hereditary Angioedema

KalVista Pharmaceuticals has submitted a New Drug Application (NDA) to the FDA for sebetralstat, an oral plasma kallikrein inhibitor, for on-demand treatment of hereditary angioedema (HAE).
The NDA is supported by Phase 3 KONFIDENT trial data, which demonstrated sebetralstat's ability to achieve significantly faster symptom relief compared to placebo in HAE patients.

FDA Accepts Stealth BioTherapeutics' NDA for Elamipretide in Barth Syndrome

The FDA has accepted Stealth BioTherapeutics' NDA for elamipretide, a potential first-in-class therapy for Barth syndrome, a rare genetic condition.
The NDA is supported by positive data from the SPIBA-001 Phase 3 study and the TAZPOWER Part 2 trial, highlighting elamipretide's efficacy and safety.

FDA Accepts Milestone's Cardamyst NDA for PSVT Treatment

The FDA has accepted Milestone Pharmaceuticals' NDA for Cardamyst (etripamil) nasal spray, a potential treatment for paroxysmal supraventricular tachycardia (PSVT).
Cardamyst is designed for patient self-administration, offering a rapid-response therapy to manage PSVT episodes without immediate medical oversight.

Reference News

[1]
Stealth Biotherapeutics Announces FDA Acceptance of New Drug Application for Elamipretide for the Treatment of Barth Syndrome
drugs.com · Apr 8, 2024

Stealth BioTherapeutics announces FDA acceptance of New Drug Application for elamipretide, a potential treatment for Bar...

© Copyright 2025. All Rights Reserved by MedPath
HomeTerms & ConditionsPrivacy Policy