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2w ago

FDA Grants Accelerated Approval to Forzinity, First Therapy for Barth Syndrome

The agency based its decision on gains in knee‑extensor strength judged reasonably likely to signal real‑world benefit.

Signage is seen outside of the Food and Drug Administration (FDA) headquarters in White Oak, Maryland, U.S., August 29, 2020. REUTERS/Andrew Kelly/File Photo
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vial and syringe for vaccine on white background closeup

Overview

  • Eligibility covers patients with Barth syndrome who weigh at least 30 kilograms, according to the FDA authorization.
  • Forzinity is a once‑daily subcutaneous injection that binds to the inner part of mitochondria to improve their structure and function.
  • The surrogate endpoint reflects improved strength in the muscle that straightens the knee, which the FDA says may translate into standing more easily or walking farther.
  • Stealth Biotherapeutics must run a post‑market confirmatory trial, and the FDA can seek withdrawal if effectiveness is not verified.
  • Barth syndrome is a rare mitochondrial disorder that primarily affects males, often causing severe heart failure in infancy, with an estimated 230 to 250 identified cases worldwide as of 2020.