Company is preparing for Advisory Committee
Meeting with the Cardiovascular and Renal Drugs Advisory
Committee
NEEDHAM,
Mass., July 29, 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, today announced completion of
the mid-cycle review meeting with the U.S. Food and Drug
Administration ("FDA") regarding its New Drug Application ("NDA")
for elamipretide for the treatment of Barth syndrome. If approved,
elamipretide would be the first therapy for Barth syndrome, an
ultra-rare, progressive, life-shortening, cardioskeletal disease
that affects approximately 150 individuals in the United States. The FDA has confirmed its
intent to hold an advisory committee meeting, which the Company
expects will occur this fall. The NDA for elamipretide was granted
priority review and has been assigned a Prescription Drug User Fee
Act ("PDUFA") action date of January
29, 2025.
"We look forward to sharing the evidence supporting
elamipretide's potential to improve the lives of patients living
with Barth syndrome with the advisory committee," said Reenie McCarthy, Chief Executive Officer. "We
thank the Center for Drug Evaluation and Research and the Office of
Cardiology, Hematology, Endocrinology and Nephrology for their
leadership in charting pathways for devastating ultrarare diseases
like Barth syndrome. This community is in dire need of a treatment
option, and we believe the evidence supports elamipretide as
the first therapy to address this unmet need."
If approved, this would be the first marketing authorization for
elamipretide, a first-in-class mitochondrial targeted therapeutic.
Elamipretide is also in Phase 3 development for primary
mitochondrial myopathy, with pivotal data expected in late 2024,
and 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
five. Barth syndrome occurs almost exclusively 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
Stern Investor Relations
Austin.Murtagh@sternir.com
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SOURCE Stealth BioTherapeutics Inc.