Regenxbio to seek FDA's accelerated approval for Hunter Syndrome treatment, shares rise 8%

Aug. 03, 2022 10:02 AM ETREGENXBIO Inc. (RGNX)By: Anuron Mitra, SA News Editor2 Comments

Headquarters of US Food and Drug Administration (FDA)


  • Regenxbio (NASDAQ:RGNX) on Wednesday said it intends to file a biologics license application (BLA) in 2024 using the U.S. FDA's accelerated approval process for its RGX-121 treatment candidate for Mucopolysaccharidosis Type II (MPS II).
  • Shares of the clinical-stage biotech were 8% higher at $34.34 in early trading. The company is slated to report Q2 results after market close.
  • MPS II, also known as Hunter Syndrome, is a hereditary metabolic disease in which the body is missing or does not have enough of an enzyme needed to break down long chains of sugar molecules.
  • The FDA's accelerated approval program allows for the expedited development and review of drugs that treat serious conditions.
  • RGNX said its ongoing phase 1/2 trial of RGX-121 in children up to five years old had been expanded into an active pivotal phase 1/2/3 trial and was enrolling patients.
  • Preliminary results from the early-to-mid stage trial of RGX-121 in Feb. showed that it continued to be well-tolerated with dose-dependent reductions of glycosaminoglycans in the cerebrospinal fluid.

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