Friday, July 10, 2026

Drug profile · INN

ataluren

ataluren (TRANSLARNA) is a 80s ribosome modulator.

EMA: unknown Musculo-skeletal system (M09) M09AX03
Patents linked 0
Pipeline programs 11
News articles 0
Drug details — ataluren
Class
Musculo-skeletal system (M09)
Route
Patents
0
Programs
11
Data quality
0.83

Quick answer

ataluren (TRANSLARNA) is a 80s ribosome modulator.

Key facts

INN ataluren
Brand TRANSLARNA
Mechanism 80S Ribosome modulator
Target 80S Ribosome
Therapeutic class Musculo-skeletal system (M09)
ATC code M09AX03

Mechanism of action

80S Ribosome modulator

Primary target: 80S Ribosome

Approval history

Authority Status Date
EMA unknown

Pipeline programs

  1. Ataluren Phase 3 · PTC THERAPEUTICS · Cystic Fibrosis
  2. Ataluren Phase 3 · PTC THERAPEUTICS · Duchenne Muscular Dystrophy
  3. Ataluren Phase 3 · PTC THERAPEUTICS · Muscular Dystrophy, Duchenne
  4. Ataluren (PTC124®) Phase 3 · PTC THERAPEUTICS · Cystic Fibrosis
  5. Ataluren Phase 2 · PTC THERAPEUTICS · Amino Acid Metabolism, Inborn Errors
  6. Ataluren Phase 2 · PTC THERAPEUTICS · Aniridia
  7. Ataluren Phase 2 · PTC THERAPEUTICS · Nonsene Mutation Duchenne Muscular Dystrophy
  8. Ataluren Phase 2 · PTC THERAPEUTICS · Hemophilia A

Related & competitor drugs

  1. allopurinol Musculo-skeletal system (M04)
  2. colchicine Musculo-skeletal system (M04)
  3. denosumab Musculo-skeletal system (M05)
  4. eteplirsen Musculo-skeletal system (M09)
  5. febuxostat Musculo-skeletal system (M04)
  6. ibandronic acid Musculo-skeletal system (M05)

Frequently asked questions

What is ataluren?

ataluren (TRANSLARNA) is a 80s ribosome modulator.

What is the brand name for ataluren?

TRANSLARNA is a marketed brand name for ataluren. Check FDA Drugs@FDA for the current US label holder and prescribing information.