Ionis Pharmaceuticals' Dawnzera has received FDA approval for HAE treatment in adults and children, offering a competitive edge with its patient-friendly auto-injector and flexible dosing schedule. A switch study demonstrated a substantial reduction in HAE attack rates and strong patient preference for Dawnzera over existing treatments, supporting a Buy rating by William Blair analyst Myles Minter.
Ionis Pharmaceuticals' Dawnzera (donidalorsen) has received FDA approval for the prophylaxis of hereditary angioedema (HAE) in both adults and children aged 12 and above. This approval marks a significant milestone in the treatment of HAE, a rare and potentially life-threatening genetic condition characterized by recurrent attacks of severe swelling.
Dawnzera is the first 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 drug is administered via a self-injectable auto-injector, offering patients a convenient and flexible dosing schedule every four or eight weeks.
The approval of Dawnzera comes on the heels of promising clinical trial results. The Phase 3 OASIS-HAE study demonstrated that Dawnzera significantly reduced monthly HAE attack rates by 81% compared to placebo over 24 weeks. Additionally, a switch study showed that patients previously treated with other prophylactic therapies experienced a 62% reduction in HAE attack rates after switching to Dawnzera.
The approval of Dawnzera is particularly notable as it enters a crowded market with 11 other FDA-approved treatments for HAE. However, Dawnzera's unique RNA-targeted approach and patient-friendly auto-injector set it apart from existing treatments. The drug's long dosing interval and strong efficacy have led William Blair analyst Myles Minter to issue a Buy rating for Ionis Pharmaceuticals.
Ionis Pharmaceuticals has a history of innovation in RNA-targeted medicines, and the approval of Dawnzera further solidifies its position as a leader in this field. The company has also committed to providing comprehensive support services to help patients access the medicine and manage their treatment.
For more information, visit Ionis Pharmaceuticals' website at [Ionis.com](https://ionis.com).
References:
[1] https://finance.yahoo.com/news/dawnzera-donidalorsen-approved-u-first-154500495.html
[2] https://www.fiercepharma.com/pharma/crowded-hae-market-steps-ionis-after-fda-nod-dawnzera
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