Donidalorsen is a 2'-O-methoxyethyl–modified antisense oligonucleotide (ASO) conjugated to a triantennary N-acetylgalactosamine (GalNAc3) moiety that causes ribonuclease H1 (RNase H1) mediated degradation of prekallikrein (PKK) mRNA through selective binding to PKK mRNA, which results in reduced production of PKK protein. PKK is a pro-enzyme for plasma kallikrein, which results in the release of bradykinin, a potent vasodilator causing inflammation and swelling in hereditary angioedema (HAE).
This medicinal substance has been classified in the anatomical therapeutic chemical (ATC) classification according to its main therapeutic use as follows:
| ATC Group | Classification | |
|---|---|---|
| B06AC09 | B Blood and blood forming organs → B06 Other hematological agents → B06A Other hematological agents → B06AC Drugs used in hereditary angioedema | |
Competent medicine agencies globally have authorized commercialization of this active ingredient according to these medication package inserts (MPIs):
| Document | Type | Information Source | |
|---|---|---|---|
| DAWNZERA Solution for injection | MPI, EU: SmPC | European Medicines Agency (EU) | |
| DAWNZERA Solution for injection | MPI, US: SPL/PLR | FDA, National Drug Code (US) |
Donidalorsen is an active ingredient of these brands:
Austria (AT)Estonia (EE)Italy (IT)Lithuania (LT)United Kingdom (UK)United States (US)Note the following: The list of brand names is continuously updated, and thus does not include the total of products circulating worldwide. |
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