Ataluren

MUSCULO-SKELETAL SYSTEM

Key facts

Ataluren is a drug catalogued in DrugCentral. It was first approved by the EMA in 2014. DrugCentral lists 2 molecular targets, including BLVRB and HDAC6. Recorded indications include Duchenne muscular dystrophy.

• Molecular targets: BLVRB and HDAC6 (2 listed in DrugCentral).
• Indications: Duchenne muscular dystrophy.
• ATC code: M09AX03 (Other drugs for disorders of the musculo-skeletal system).
• Molecular formula: C15H9FN2O3; molecular weight 284.25 g/mol.

Indications & contraindications

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

Indications (1)

• Duchenne muscular dystrophy

Mechanism & targets

Molecular targets (2)

Target	Gene	Action	Activity
Flavin reductase (NADPH)	BLVRB	unknown	Kd 6.70
Histone deacetylase 6	HDAC6	unknown	IC50 6.52

Regulatory approvals (1)

Date	Agency	Marketed as	Applicant	Orphan
2014-07-31	EMA	—	PTC Therapeutics International Limited	Yes

ATC classification

ATC code	Therapeutic hierarchy
M09AX03	MUSCULO-SKELETAL SYSTEM › OTHER DRUGS FOR DISORDERS OF THE MUSCULO-SKELETAL SYSTEM › OTHER DRUGS FOR DISORDERS OF THE MUSCULO-SKELETAL SYSTEM › Other drugs for disorders of the musculo-skeletal system

Chemical structure

Full physicochemical, ADMET and 3D-structure analysis of Ataluren is on its molecule page.

• Molecular formula: C₁₅H₉FN₂O₃
• Molecular weight: 284.25 g/mol
• CAS number: —
• InChIKey: OOUGLTULBSNHNF-UHFFFAOYSA-N
• PubChem CID: 11219835

OC(=O)C1=CC=CC(=C1)C1=NOC(=N1)C1=C(F)C=CC=C1

Frequently asked questions

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

What is Ataluren used for?

DrugCentral lists the following indications for Ataluren: Duchenne muscular dystrophy.

How does Ataluren work?

Its molecular targets in DrugCentral include BLVRB and HDAC6.

When was Ataluren first approved?

Ataluren was first approved by the EMA in 2014.

What is the chemical formula of Ataluren?

Ataluren has the molecular formula C15H9FN2O3 and a molecular weight of 284.25 g/mol.

Sources

• DrugCentral drug card 4872 — structure, indications, targets, approvals, pharmacologic classes (CC BY-SA 4.0)
• ChEMBL CHEMBL256997 — compound record and bioactivity
• PubChem CID 11219835 — chemical identifiers
• DrugBank DB05016 — drug record
• FDA UNII K16AME9I3V — substance identity
• UniProt P30043 (BLVRB) — target protein
• UniProt Q9UBN7 (HDAC6) — target protein

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