Ionis Pharmaceuticals Inc (Nasdaq: IONS) announced on Monday that the FDA has accepted its New Drug Application (NDA) for donidalorsen, an RNA-targeted therapy designed to prevent hereditary angioedema (HAE) attacks in patients aged 12 and older. The Prescription Drug User Fee Act (PDUFA) target decision date is set for 21 August 2025.

Assuming approval, donidalorsen would be the first RNA-targeted prophylactic treatment for HAE, and Ionis' second independent commercial launch. The FDA's decision follows positive results from the Phase 3 OASIS-HAE trial and ongoing Phase 2 and Phase 3 open-label studies, which demonstrated a 96% sustained reduction in HAE attack rates over three years.

Donidalorsen works by targeting prekallikrein (PKK), a key enzyme in the inflammatory pathway associated with HAE attacks. With Orphan Drug Designation granted by the FDA in 2023, Ionis and its partner Otsuka are preparing a Marketing Authorization Application for European regulatory submission.

Ionis, a leader in RNA-targeted medicine, has pioneered innovations across multiple therapeutic areas and currently markets five drugs, with a robust pipeline aimed at meeting high unmet needs in neurology, cardiology and other critical fields.