Patisiran

Small Interfering RNA — NERVOUS SYSTEM

Key facts

Patisiran is a drug in the Small Interfering RNA class catalogued in DrugCentral. It was first approved by the EMA in 2018. DrugCentral lists 1 molecular target, including TTR. Recorded indications include Polyneuropathy in amyloidosis and Transthyretin familial amyloid polyneuropathy.

• Drug class: Small Interfering RNA; Transthyretin-directed RNA Interaction.
• Molecular targets: TTR (1 listed in DrugCentral).
• Indications: Polyneuropathy in amyloidosis and Transthyretin familial amyloid polyneuropathy.
• ATC code: N07XX12 (Other nervous system drugs).

Indications & contraindications

Drug–disease relationships curated by DrugCentral (OMOP / SNOMED concepts).

Indications (2)

• Polyneuropathy in amyloidosis
• Transthyretin familial amyloid polyneuropathy

Mechanism & targets

• Established pharmacologic class: Small Interfering RNA; Transthyretin-directed RNA Interaction

Molecular targets (1)

Target	Gene	Action	Activity
transthyretin (TTR) mRNA	TTR	other	—

Regulatory approvals (3)

Date	Agency	Marketed as	Applicant	Orphan
2019-06-18	PMDA	—	ALNYLAM JAPAN K.K.	Yes
2018-10-08	FDA	—	ALNYLAM PHARMS INC	Yes
2018-08-27	EMA	—	ALNYLAM NETHERLANDS B.V.	Yes

ATC classification

ATC code	Therapeutic hierarchy
N07XX12	NERVOUS SYSTEM › OTHER NERVOUS SYSTEM DRUGS › OTHER NERVOUS SYSTEM DRUGS › Other nervous system drugs

Pharmacogenomics

Drug-label and guideline annotations from PharmGKB and CPIC.

• EMA label: TTR · Testing Required
• FDA label: TTR · Testing Required
• HCSC label: TTR · Testing Required

Reported adverse-event signals

Strongest FDA FAERS disproportionality signals (log-likelihood ratio). A signal is a statistical association in spontaneous reports, not proof that the drug causes the event.

MedDRA term	LLR
Intentional dose omission	246.3
Product dose omission issue	163.8
Death	145.6
Cardiac failure	103.9
Infusion related reaction	101.2
Vitamin A decreased	94.2
Infusion site extravasation	80.6
Hospitalisation	75.0
Product preparation error	73.6
Therapy interrupted	72.8
No adverse event	72.5
Cardiac pacemaker insertion	65.5

Chemical structure

• Molecular formula: —
• Molecular weight: —
• CAS number: 1420706-45-1
• InChIKey: —
• PubChem CID: —

Frequently asked questions

Answers are generated from the structured data on this page, not written by hand.

What is Patisiran used for?

DrugCentral lists the following indications for Patisiran: Polyneuropathy in amyloidosis and Transthyretin familial amyloid polyneuropathy.

How does Patisiran work?

Its molecular targets in DrugCentral include TTR.

When was Patisiran first approved?

Patisiran was first approved by the EMA in 2018.

What drug class does Patisiran belong to?

Patisiran is in the Small Interfering RNA class.

Sources

• DrugCentral drug card 5313 — structure, indications, targets, approvals, pharmacologic classes (CC BY-SA 4.0)
• ChEMBL CHEMBL3989987 — compound record and bioactivity
• DrugBank DB14582 — drug record
• FDA UNII 50FKX8CB2Y — substance identity
• DailyMed: FDA labels for Patisiran — current prescribing information (label search)
• UniProt P02766 (TTR) — target protein
• FDA Adverse Event Reporting System (openFDA FAERS) — adverse-event signals, as aggregated by DrugCentral
• PharmGKB / CPIC — pharmacogenomic label annotations

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