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Donidalorsen: First Approval.

Yahiya Y Syed1

  • 1Springer Nature, Private Bag 65901, Mairangi Bay, Auckland, 0754, New Zealand. dru@adis.com.

Drugs
|November 12, 2025
PubMed
Summary
This summary is machine-generated.

Donidalorsen, an RNA-targeted antisense oligonucleotide, is now approved in the USA for preventing hereditary angioedema attacks. This marks a significant advancement in HAE prophylaxis for patients aged 12 and older.

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Area of Science:

  • Pharmacology
  • Genetics
  • Drug Development

Background:

  • Hereditary angioedema (HAE) is a rare genetic disorder characterized by recurrent swelling attacks.
  • Current HAE treatments focus on managing acute attacks, with a need for effective prophylactic options.
  • Donidalorsen represents a novel therapeutic approach targeting the underlying pathophysiology of HAE.

Purpose of the Study:

  • To summarize the key developmental milestones of donidalorsen.
  • To highlight the regulatory pathway leading to its first approval for HAE prophylaxis.
  • To provide an overview of donidalorsen as a first-in-class RNA-targeted therapy.

Main Methods:

  • Review of regulatory submissions and clinical trial data for donidalorsen.
  • Analysis of the mechanism of action of RNA-targeted antisense oligonucleotides (ASOs).

Related Experiment Videos

  • Documentation of the drug development timeline and key decision points.

    • Main Results:

    • Donidalorsen received its first US approval on August 21, 2025, for HAE prophylaxis in patients aged 12 years and older.
    • The drug is a first-in-class, RNA-targeted antisense oligonucleotide (ASO).
    • Donidalorsen is currently under regulatory review in the EU and in Phase III development globally.

    Conclusions:

    • Donidalorsen's approval signifies a major breakthrough in HAE management.
    • This RNA-targeted ASO offers a new prophylactic strategy for HAE patients.
    • The successful development highlights the potential of ASO technology in treating genetic disorders.