Stem definition | Drug id | CAS RN |
---|---|---|
small interfering RNA including siRNA, miRNA and piRNA | 5354 | 1639325-43-1 |
Molecule | Description |
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Synonyms:
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Givosiran is a double-stranded small interfering RNA that causes degradation of aminolevulinate synthase1 (ALAS1) mRNA in hepatocytes through RNA interference, reducing the elevated levels of liver ALAS1 mRNA. This leads to reduced circulating levels of neurotoxic intermediates aminolevulinic acid (ALA) and porphobilinogen (PBG), factors associated with attacks and other disease manifestations of AHP.
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Property | Value | Reference |
---|---|---|
S (Water solubility) | 357 mg/mL | Bocci G, Oprea TI, Benet LZ |
BDDCS (Biopharmaceutical Drug Disposition Classification System) | 1 | Bocci G, Oprea TI, Benet LZ |
Date | Agency | Company | Orphan |
---|---|---|---|
March 2, 2020 | EMA | Alnylam Netherlands B.V. | |
Nov. 20, 2019 | FDA | ALNYLAM PHARMS INC | |
June 23, 2021 | PMDA | ALNYLAM JAPAN K.K |
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Source | Code | Description |
---|---|---|
ATC | A16AX16 | ALIMENTARY TRACT AND METABOLISM OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS Various alimentary tract and metabolism products |
FDA CS | M0406100 | RNA, Small Interfering |
FDA PE | N0000008852 | Decreased RNA Integrity |
FDA EPC | N0000193848 | Small Interfering RNA |
FDA EPC | N0000194024 | Aminolevulinate Synthase 1-directed RNA Interaction |
Disease | Relation | SNOMED_ID | DOID |
---|---|---|---|
Hepatic porphyria | indication | 55056006 | DOID:3133 |
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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 189MG BASE/ML (EQ 189MG BASE/ML) | GIVLAARI | ALNYLAM PHARMS INC | N212194 | Nov. 20, 2019 | RX | SOLUTION | SUBCUTANEOUS | 9708610 | Jan. 1, 2024 | TREATMENT OF ACUTE HEPATIC PORPHYRIA |
EQ 189MG BASE/ML (EQ 189MG BASE/ML) | GIVLAARI | ALNYLAM PHARMS INC | N212194 | Nov. 20, 2019 | RX | SOLUTION | SUBCUTANEOUS | 10131907 | Aug. 24, 2028 | TREATMENT OF ACUTE HEPATIC PORPHYRIA |
EQ 189MG BASE/ML (EQ 189MG BASE/ML) | GIVLAARI | ALNYLAM PHARMS INC | N212194 | Nov. 20, 2019 | RX | SOLUTION | SUBCUTANEOUS | 8828956 | Dec. 4, 2028 | TREATMENT OF ACUTE HEPATIC PORPHYRIA |
EQ 189MG BASE/ML (EQ 189MG BASE/ML) | GIVLAARI | ALNYLAM PHARMS INC | N212194 | Nov. 20, 2019 | RX | SOLUTION | SUBCUTANEOUS | 8106022 | Dec. 12, 2029 | TREATMENT OF ACUTE HEPATIC PORPHYRIA |
EQ 189MG BASE/ML (EQ 189MG BASE/ML) | GIVLAARI | ALNYLAM PHARMS INC | N212194 | Nov. 20, 2019 | RX | SOLUTION | SUBCUTANEOUS | 10125364 | March 15, 2033 | TREATMENT OF ACUTE HEPATIC PORPHYRIA |
EQ 189MG BASE/ML (EQ 189MG BASE/ML) | GIVLAARI | ALNYLAM PHARMS INC | N212194 | Nov. 20, 2019 | RX | SOLUTION | SUBCUTANEOUS | 9631193 | March 15, 2033 | TREATMENT OF ACUTE HEPATIC PORPHYRIA |
EQ 189MG BASE/ML (EQ 189MG BASE/ML) | GIVLAARI | ALNYLAM PHARMS INC | N212194 | Nov. 20, 2019 | RX | SOLUTION | SUBCUTANEOUS | 9133461 | Nov. 30, 2033 | TREATMENT OF ACUTE HEPATIC PORPHYRIA |
EQ 189MG BASE/ML (EQ 189MG BASE/ML) | GIVLAARI | ALNYLAM PHARMS INC | N212194 | Nov. 20, 2019 | RX | SOLUTION | SUBCUTANEOUS | 10119143 | Oct. 3, 2034 | TREATMENT OF ACUTE HEPATIC PORPHYRIA |
EQ 189MG BASE/ML (EQ 189MG BASE/ML) | GIVLAARI | ALNYLAM PHARMS INC | N212194 | Nov. 20, 2019 | RX | SOLUTION | SUBCUTANEOUS | 11028392 | Oct. 3, 2034 | TREATMENT OF ACUTE HEPATIC PORPHYRIA |
Formulation strength | Trade name | Applicant | Application number | Approval date | Type | Dose form | Route | Exclusivity date | Description |
---|---|---|---|---|---|---|---|---|---|
EQ 189MG BASE/ML (EQ 189MG BASE/ML) | GIVLAARI | ALNYLAM PHARMS INC | N212194 | Nov. 20, 2019 | RX | SOLUTION | SUBCUTANEOUS | Nov. 20, 2024 | NEW CHEMICAL ENTITY |
EQ 189MG BASE/ML (EQ 189MG BASE/ML) | GIVLAARI | ALNYLAM PHARMS INC | N212194 | Nov. 20, 2019 | RX | SOLUTION | SUBCUTANEOUS | Nov. 20, 2026 | INDICATED FOR THE TREATMENT OF ADULTS WITH ACUTE HEPATIC PORPHYRIA (AHP) |
Target | Class | Pharos | UniProt | Action | Type | Activity value (-log[M]) | Mechanism action | Bioact source | MoA source |
---|---|---|---|---|---|---|---|---|---|
aminolevulinate synthase1 (ALAS1) mRNA | RNA | ANTISENSE INHIBITOR | UNKNOWN | DRUG LABEL |
ID | Source |
---|---|
ROV204583W | UNII |
C4688584 | UMLSCUI |
CHEMBL4297760 | ChEMBL_ID |
156323004 | PUBCHEM_CID |
DB15066 | DRUGBANK_ID |
CHEMBL4594265 | ChEMBL_ID |
D11702 | KEGG_DRUG |
10280 | INN_ID |
018210 | NDDF |
018211 | NDDF |
1217169003 | SNOMEDCT_US |
830100005 | SNOMEDCT_US |
830239005 | SNOMEDCT_US |
4038984 | VANDF |
2265712 | RXNORM |
327500 | MMSL |
37780 | MMSL |
d09456 | MMSL |
C000630124 | MESH_SUPPLEMENTAL_RECORD_UI |
Product | Category | Ingredients | NDC | Form | Quantity | Route | Marketing | Label |
---|---|---|---|---|---|---|---|---|
GIVLAARI | HUMAN PRESCRIPTION DRUG LABEL | 1 | 71336-1001 | INJECTION, SOLUTION | 189 mg | SUBCUTANEOUS | NDA | 28 sections |
GIVLAARI | HUMAN PRESCRIPTION DRUG LABEL | 1 | 71336-1001 | INJECTION, SOLUTION | 189 mg | SUBCUTANEOUS | NDA | 28 sections |
GIVLAARI | HUMAN PRESCRIPTION DRUG LABEL | 1 | 71336-1001 | INJECTION, SOLUTION | 189 mg | SUBCUTANEOUS | NDA | 28 sections |