Atidarsagene Autotemcel

Summary

Atidarsagene Autotemcel (CHEMBL4802262) is an approved gene (ATC A16AB21); indicated across 1 condition including metachromatic leukodystrophy.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Gene
• ATC class: A16AB21
• Indications: 1 condition
• Clinical trials: 2

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL4802262
Name	Atidarsagene Autotemcel
Type	Gene
Max phase	4
ATC	A16AB21

Also known as: Atidarsagene autotemcel, Lenmeldy, Libmeldy, OTL-200, ATIDARSAGENE AUTOTEMCEL

Targets

Targets

No target linkage available.

Bioactivity

No ChEMBL bioactivity rows at pChembl ≥ 5 (expected for biologics / antibodies).

Target pathways

No target-pathway data for this drug (no mapped target genes).

Indications & clinical

Indications

1 approved indication. FDA phase 4, plus an anticancer drug’s labelled cancer uses (which ChEMBL often logs at phase 3).

Indication	Phase	MONDO	EFO
metachromatic leukodystrophy	4	MONDO:0018868	MONDO:0018868

Clinical trials

Total trials: 2.

Phase distribution

Phase	Trials
PHASE3	1
PHASE2	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT04283227	PHASE3	ACTIVE_NOT_RECRUITING	OTL-200 in Patients With Late Juvenile Metachromatic Leukodystrophy (MLD)
NCT03392987	PHASE2	COMPLETED	A Safety and Efficacy Study of Cryopreserved OTL-200 for Treatment of Metachromatic Leukodystrophy (MLD)

Clinical evidence (CIViC)

No CIViC predictive evidence (expected for non-precision-medicine drugs).

Pharmacology

Pharmacogenomics

No PharmGKB pharmacogenomic data curated for this drug.

Related molecules

Related molecules

No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).