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Imetelstat

Oligonucleotide Telomerase Inhibitor — ANTINEOPLASTIC AND IMMUNOMODULATING AGENTS

Also known as: GRN163L, imetelstat sodium, JNJ-63935937, rytelo

EMA approvedFDA approvedOrphan designationATC L01XX80

Data last refreshed: (DrugCentral release 2024-11-01)

Key facts

Imetelstat is a drug in the Oligonucleotide Telomerase Inhibitor class catalogued in DrugCentral. It was first approved by the FDA in 2024. Its mechanism of action is classified as Oligonucleotide Telomerase Inhibitors. DrugCentral lists 1 molecular target, including TERT. Recorded indications include Low- to intermediate-1 risk myelodysplastic syndrome.

  • Drug class: Oligonucleotide Telomerase Inhibitor.
  • Mechanism of action: Oligonucleotide Telomerase Inhibitors.
  • Molecular targets: TERT (1 listed in DrugCentral).
  • Indications: Low- to intermediate-1 risk myelodysplastic syndrome.
  • ATC code: L01XX80 (Other antineoplastic agents).

Indications & contraindications

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

Indications (1)

  • Low- to intermediate-1 risk myelodysplastic syndrome

Mechanism & targets

Mechanism of action (MoA)Oligonucleotide Telomerase Inhibitors
Established pharmacologic classOligonucleotide Telomerase Inhibitor

Molecular targets (1)

TargetGeneActionActivity
Telomerase reverse transcriptaseTERTinhibitorIC50 7.25

Regulatory approvals (2)

DateAgencyMarketed asApplicantOrphan
2025-03-07EMA—Geron Netherlands B.V.Yes
2024-06-06FDA—GeronYes

ATC classification

ATC codeTherapeutic hierarchy
L01XX80ANTINEOPLASTIC AND IMMUNOMODULATING AGENTS › ANTINEOPLASTIC AGENTS › OTHER ANTINEOPLASTIC AGENTS › Other antineoplastic agents

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 termLLR
Cytopenia86.2
Thrombocytopenia75.3

Chemical structure

Molecular formula—
Molecular weight—
CAS number—
InChIKey—
PubChem CID72941969

Frequently asked questions

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

What is Imetelstat used for?

DrugCentral lists the following indications for Imetelstat: Low- to intermediate-1 risk myelodysplastic syndrome.

How does Imetelstat work?

Imetelstat is classified as Oligonucleotide Telomerase Inhibitors; its molecular targets in DrugCentral include TERT (inhibitor).

When was Imetelstat first approved?

Imetelstat was first approved by the FDA in 2024.

What drug class does Imetelstat belong to?

Imetelstat is in the Oligonucleotide Telomerase Inhibitor class.

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Sources

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

For research and educational use only — not medical advice. This page is compiled automatically from public databases. It is not written or reviewed by a clinician. Do not start, stop or change any treatment based on it; consult a qualified healthcare professional. Spotted an error? See our corrections policy or email hello@molforge.ai.

Quick facts

Status—
First approval2024-06-06
AgencyFDA
Targets1
Indications1
Formula—
DrugCentral ID5823
Interactive profile