Berotralstat

Plasma Kallikrein Inhibitor — BLOOD AND BLOOD FORMING ORGANS

Key facts

Berotralstat is an onp drug in the Plasma Kallikrein Inhibitor class catalogued in DrugCentral. It was first approved by the FDA in 2020. Its mechanism of action is classified as Cytochrome P450 2D6 Inhibitors, Cytochrome P450 3A4 Inhibitors and Kallikrein Inhibitors. DrugCentral lists 2 molecular targets, including KLKB1 and PLG. Recorded indications include Hereditary angioneurotic edema.

• Approval status: onp (first approved 2020 by the FDA).
• Drug class: Plasma Kallikrein Inhibitor.
• Mechanism of action: Cytochrome P450 2D6 Inhibitors; Cytochrome P450 3A4 Inhibitors; Kallikrein Inhibitors.
• Molecular targets: KLKB1 and PLG (2 listed in DrugCentral).
• Indications: Hereditary angioneurotic edema.
• ATC code: B06AC06 (Drugs used in hereditary angioedema).
• Molecular formula: C30H26F4N6O; molecular weight 562.57 g/mol.

Indications & contraindications

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

Indications (1)

• Hereditary angioneurotic edema

Mechanism & targets

• Mechanism of action (MoA): Cytochrome P450 2D6 Inhibitors; Cytochrome P450 3A4 Inhibitors; Kallikrein Inhibitors; P-Glycoprotein Inhibitors
• Established pharmacologic class: Plasma Kallikrein Inhibitor

Molecular targets (2)

Target	Gene	Action	Activity
Plasma kallikrein	KLKB1	inhibitor	IC50 7.80
Plasminogen	PLG	unknown	IC50 5.40

Regulatory approvals (3)

Date	Agency	Marketed as	Applicant	Orphan
2021-04-30	EMA	—	BioCryst Ireland Limited	Yes
2021-01-22	PMDA	—	ORPHAN PACIFIC, Inc	Yes
2020-12-03	FDA	—	BIOCRYST	Yes

ATC classification

ATC code	Therapeutic hierarchy
B06AC06	BLOOD AND BLOOD FORMING ORGANS › OTHER HEMATOLOGICAL AGENTS › OTHER HEMATOLOGICAL AGENTS › Drugs used in hereditary angioedema

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
Hereditary angioedema	330.0

Chemical structure

• Molecular formula: C₃₀H₂₆F₄N₆O
• Molecular weight: 562.57 g/mol
• CAS number: —
• InChIKey: UXNXMBYCBRBRFD-MUUNZHRXSA-N
• PubChem CID: 137528262

C1CC1CN[C@@H](C2=CC(=C(C=C2)F)NC(=O)C3=CC(=NN3C4=CC=CC(=C4)CN)C(F)(F)F)C5=CC=CC(=C5)C#N

Frequently asked questions

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

What is Berotralstat used for?

DrugCentral lists the following indications for Berotralstat: Hereditary angioneurotic edema.

How does Berotralstat work?

Berotralstat is classified as Cytochrome P450 2D6 Inhibitors, Cytochrome P450 3A4 Inhibitors and Kallikrein Inhibitors; its molecular targets in DrugCentral include KLKB1 (inhibitor) and PLG.

When was Berotralstat first approved?

Berotralstat was first approved by the FDA in 2020.

What drug class does Berotralstat belong to?

Berotralstat is in the Plasma Kallikrein Inhibitor class.

What is the chemical formula of Berotralstat?

Berotralstat has the molecular formula C30H26F4N6O and a molecular weight of 562.57 g/mol.

Sources

• DrugCentral drug card 5430 — structure, indications, targets, approvals, pharmacologic classes (CC BY-SA 4.0)
• ChEMBL CHEMBL5189739 — compound record and bioactivity
• PubChem CID 137528262 — chemical identifiers
• DrugBank DB15982 — drug record
• FDA UNII XZA0KB1BDQ — substance identity
• DailyMed: FDA labels for Berotralstat — current prescribing information (label search)
• UniProt P03952 (KLKB1) — target protein
• UniProt P00747 (PLG) — target protein
• FDA Adverse Event Reporting System (openFDA FAERS) — adverse-event signals, as aggregated by DrugCentral

How this page is built: methodology · editorial policy & corrections.