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 […]
In a significant development for the treatment of chronic hepatitis B (CHB), GSK plc (LSE/NYSE: GSK) has announced that the US Food and Drug Administration (FDA) has granted Fast Track designation to bepirovirsen, an investigational antisense oligonucleotide (ASO). This milestone is a testament to the potential of bepirovirsen in addressing the unmet medical needs of […]
In a major development for hereditary transthyretin-mediated amyloidosis (ATTRv-PN or hATTR-PN) patients, the US Food and Drug Administration (FDA) has approved Wainua (eplontersen), developed by AstraZeneca PLC and Ionis Pharmaceuticals Inc. This approval is particularly noteworthy as Wainua is the first and only FDA-approved medication for ATTRv-PN that can be self-administered using an auto-injector. The […]
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 […]
Ionis Pharmaceuticals is set to take full ownership of Akcea Therapeutics by acquiring the remaining stake of around 24% in the latter for about $500 million in an all-cash deal. The California-based biotech company, which is engaged in RNA-targeted drug discovery and development, will buy all the shares of it didn’t own before in Akcea […]