Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) has announced that the U.S. Food and Drug Administration (FDA) has approved Dawnzera™ (donidalorsen) for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years of age and older. This approval marks a significant advancement in the treatment of HAE, a rare and potentially life-threatening genetic condition affecting an estimated 7,000 people in the U.S.
Dawnzera is the first and only RNA-targeted medicine approved for HAE, designed to target plasma prekallikrein (pkk), a key protein that activates inflammatory mediators associated with acute attacks of HAE. The medication offers the longest dosing option for HAE, with dosing every four (q4w) or eight weeks (q8w), and is self-administered via a subcutaneous autoinjector.
The approval of Dawnzera was based on positive results from the phase 3 global, multicenter, randomized, double-blind, placebo-controlled Oasis-HAE study. The study demonstrated that Dawnzera q4w significantly reduced monthly HAE attack rate by 81% compared to placebo over 24 weeks, with a mean attack rate reduction increasing to 87% when measured from the second dose. Additionally, Dawnzera q4w reduced moderate-to-severe HAE attacks by approximately 90% over 24 weeks when measured from the second dose.
Furthermore, the ongoing Oasisplus open-label extension (OLE) study showed that Dawnzera q8w had a similar effect as q4w over time, demonstrating a 94% total mean attack rate reduction from baseline across both dosing groups after one year in the OLE. The switch cohort evaluating Dawnzera q4w in patients previously treated with other prophylactic treatments also yielded positive results, with a 62% reduction in mean HAE attack rate from prior prophylactic treatment over 16 weeks, and 84% of patients surveyed preferring Dawnzera over their prior prophylactic treatment.
In terms of safety and tolerability, Dawnzera demonstrated a favorable profile, with the most common adverse reactions being injection site reactions, upper respiratory tract infection, urinary tract infection, and abdominal discomfort.
The approval of Dawnzera has been well-received by key figures in the HAE community, with Anthony J. Castaldo, CEO & Chairman of the Board, U.S. Hereditary Angioedema Association (HAEA) and Hereditary Angioedema International (HAEi), stating that the approval gives people living with HAE and their physicians another important choice for aligning treatment with individual needs.
The medication is expected to be available in the U.S. in the coming days, and Ionis has expressed its commitment to supporting the HAE community through Ionis Every Step™, offering a suite of services designed to meet the unique needs of HAE patients and healthcare providers.
This approval marks Ionis' second independent launch in less than nine months, following the successful launch of Tryngolza® for Familial Chylomicronemia Syndrome (FCS), and the company is poised for potential additional launches next year. As a result of these announcements, the company's shares have moved 0.36% on the market, and are now trading at a price of $42.9651. For the full picture, make sure to review IONIS PHARMACEUTICALS INC's 8-K report.
