In a significant development for hereditary angioedema (HAE) patients, Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for donidalorsen, an investigational RNA-targeted therapy designed to prevent HAE attacks in individuals aged 12 and older. The FDA has set a Prescription Drug User […]
Ionis Pharmaceuticals, a US-based developer of RNA-targeted therapies, has launched the OASIS-HAE phase 3 clinical trial for donidalorsen (formerly IONIS-PKK-LRx) in patients having hereditary angioedema (HAE). Donidalorsen, which is an investigational antisense drug, is designed to lower the production of prekallikrein, which has a major role in triggering inflammatory mediators pertaining to acute attacks of […]
