Ionis treatment for Angelman syndrome receives orphan drug and rare pediatric disease designations from U.S. FDA

CARLSBAD, Calif., June 13, 2022 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS), the leader in RNA-targeted therapeutics, announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation and rare pediatric disease designation to ION582, an investigational antisense medicine for the treatment of Angelman syndrome, a rare neurogenetic disorder caused by the loss of function of the maternally inherited Ubiquitin Protein Ligase E3A (UBE3A) gene. ION582 targets UBE3A.

The release as fetched from its publisher. ionis.com ↗