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| Stem definition | Drug id | CAS RN |
|---|---|---|
| antisense oligonucleotides | 5796 | None |
| Molecule | Description |
|---|---|
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Synonyms:
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Donidalorsen is an ASO-GalNAc conjugate that causes ribonuclease H1 (RNase H1)-mediated degradation of PKK mRNA through 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 swelling and pain in HAE. In patients with HAE, C1-inhibitor (C1-INH) deficiency or dysfunction leads to excessive plasma kallikrein activity, bradykinin generation, and angioedema attacks. Donidalorsen lowers PKK concentration, preventing excessive bradykinin production in patients with HAE.
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| Date | Agency | Company | Orphan |
|---|---|---|---|
| Aug. 21, 2025 | FDA | Ionis Pharmaceuticals, Inc. | |
| Jan. 19, 2026 | EMA | Otsuka Pharmaceutical Netherlands B.V. |
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| Source | Code | Description |
|---|---|---|
| ATC | B06AC09 | BLOOD AND BLOOD FORMING ORGANS OTHER HEMATOLOGICAL AGENTS OTHER HEMATOLOGICAL AGENTS Drugs used in hereditary angioedema |
| CHEBI has role | CHEBI:22587 | antiviral agent |
| Disease | Relation | SNOMED_ID | DOID |
|---|---|---|---|
| Hereditary angioneurotic edema | indication | 82966003 | DOID:14735 |
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| Formulation strength | Trade name | Applicant | Application number | Approval date | Type | Dose form | Route | Patent number | Patent expiration date | Patent use |
|---|---|---|---|---|---|---|---|---|---|---|
| EQ 80MG BASE/0.8ML (EQ 80MG BASE/0.8ML) | DAWNZERA (AUTOINJECTOR) | IONIS PHARMS INC | N219407 | Aug. 21, 2025 | RX | SOLUTION | SUBCUTANEOUS | 9315811 | Oct. 21, 2032 | PROPHYLAXIS TO PREVENT ATTACKS OF HEREDITARY ANGIOEDEMA (HAE) IN ADULT AND PEDIATRIC PATIENTS 12 YEARS OF AGE AND OLDER |
| EQ 80MG BASE/0.8ML (EQ 80MG BASE/0.8ML) | DAWNZERA (AUTOINJECTOR) | IONIS PHARMS INC | N219407 | Aug. 21, 2025 | RX | SOLUTION | SUBCUTANEOUS | 10294477 | May 1, 2035 | PROPHYLAXIS TO PREVENT ATTACKS OF HEREDITARY ANGIOEDEMA (HAE) IN ADULT AND PEDIATRIC PATIENTS 12 YEARS OF AGE AND OLDER |
| Formulation strength | Trade name | Applicant | Application number | Approval date | Type | Dose form | Route | Exclusivity date | Description |
|---|---|---|---|---|---|---|---|---|---|
| EQ 80MG BASE/0.8ML (EQ 80MG BASE/0.8ML) | DAWNZERA (AUTOINJECTOR) | IONIS PHARMS INC | N219407 | Aug. 21, 2025 | RX | SOLUTION | SUBCUTANEOUS | Aug. 21, 2032 | PROPHYLAXIS TO PREVENT ATTACKS OF HEREDITARY ANGIOEDEMA (HAE) IN ADULT AND PEDIATRIC PATIENTS 12 YEARS OF AGE AND OLDER |
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| ID | Source |
|---|---|
| 2304692-48-4 | SECONDARY_CAS_RN |
| CHEMBL4650235 | ChEMBL_ID |
| C000723381 | MESH_SUPPLEMENTAL_RECORD_UI |
| 4044242 | VANDF |
| C5447584 | UMLSCUI |
| CHEBI:758141 | CHEBI |
| CHEMBL4650236 | ChEMBL_ID |
| 11653 | INN_ID |
| 2304701-45-7 | SECONDARY_CAS_RN |
| 44351 | MMSL |
| d10307 | MMSL |
| 020204 | NDDF |
| 020205 | NDDF |
| 13610 | IUPHAR_LIGAND_ID |
| ZD4D8M32TL | UNII |
| 2724361 | RXNORM |
| C000711268 | MESH_SUPPLEMENTAL_RECORD_UI |
| D13154 | KEGG_DRUG |
| Product | Category | Ingredients | NDC | Form | Quantity | Route | Marketing | Label |
|---|---|---|---|---|---|---|---|---|
| DAWNZERA | HUMAN PRESCRIPTION DRUG LABEL | 1 | 71860-103 | INJECTION, SOLUTION | 80 mg | SUBCUTANEOUS | NDA | 28 sections |