Meeting Scheduled with Cardiovascular and
Renal Drugs Advisory Committee on October
10, 2024
NEEDHAM,
Mass., Sept. 9, 2024 /PRNewswire/ --
Stealth BioTherapeutics Inc. (the "Company" or "Stealth"), a
clinical-stage biotechnology company focused on the discovery,
development and commercialization of novel therapies for diseases
involving mitochondrial dysfunction, announced today that the U.S.
Food and Drug Administration (FDA) will hold an advisory committee
meeting with the Cardiovascular and Renal Drugs Advisory Committee
(CRDAC) on October 10, 2024 to
evaluate the New Drug Application (NDA) for elamipretide, the first
potential therapy for Barth syndrome. This ultra-rare, progressive
disease affects approximately 150 individuals in the United States, causing severe cardiac and
skeletal muscle manifestations that significantly reduce life
expectancy. The NDA has been granted priority review, with a
Prescription Drug User Fee Act (PDUFA) action date of January 29, 2025.
"We are grateful to the patients, families, and physicians who
participated in the studies that supported this new drug
application, as well as to the patient and medical community
members who requested a review of elamipretide as the first
potential therapy for Barth syndrome," said Reenie McCarthy, Chief Executive Officer. "We
look forward to meeting with the Cardiovascular and Renal Drugs
Advisory Committee to discuss elamipretide's potential to improve
the lives of individuals living with Barth syndrome, a devastating
disease with a significant and urgent unmet medical need."
If approved, this would be the first marketing authorization for
elamipretide, a first-in-class mitochondria-targeted therapeutic.
In addition to Barth syndrome, elamipretide is in Phase 3 trials
for primary mitochondrial myopathy, with pivotal data expected by
the end of 2024, and for dry age-related macular degeneration.
About Barth Syndrome
Barth syndrome is an ultra-rare genetic condition characterized by
cardiac abnormalities leading to exercise intolerance, 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. Barth syndrome occurs primarily in males and is estimated to
affect one in 1,000,000 males worldwide or around 150 individuals
in the United States. There are
currently no FDA- or EMA-approved therapies for patients with Barth
syndrome. Elamipretide has Orphan Drug, Fast Track and Rare
Pediatric Designation from the FDA and Orphan Drug Designation from
the EMA for the treatment of Barth syndrome.
About Stealth BioTherapeutics
Our mission is to develop novel therapies to improve the lives of
patients living with diseases of mitochondrial dysfunction. Our
lead product candidate, elamipretide, is under review for Barth
syndrome and in late-stage development for primary mitochondrial
myopathy and dry age-related macular degeneration. We are also
evaluating a topical ophthalmic formulation of our
second-generation clinical-stage candidate, bevemipretide
(SBT-272), for dry age-related macular degeneration, and have a
deep pipeline of novel compounds under evaluation for rare
neurological and cardiac disease indications.
Media Contact
Anna Stallmann Communications
Anna Stallmann
anna@annacomms.com
Investor Contact
Austin Murtagh
Precision AQ
Austin.Murtagh@precisionaq.com
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SOURCE Stealth BioTherapeutics Inc.