Translarna (ataluren)
ConditionalAdditional monitoring
- Region
- 🇪🇺 EU
- Active substance
- ataluren
- Company
- PTC Therapeutics International Limited
- Status
- Expired
- Therapeutic area
- Muscular Dystrophy, Duchenne
- CHMP opinion
- Thu 23 Jan 2014
- EU approval
- Thu 31 Jul 2014
- Designations
- Conditional, Additional monitoring
- Application
- EMEA/H/C/002720
- ATC
- M09AX03
- Official record
- www.ema.europa.eu
Indication
Translarna is indicated for the treatment of Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene, in ambulatory patients aged 2 years and older. Efficacy has not been demonstrated in non-ambulatory patients. The presence of a nonsense mutation in the dystrophin gene should be determined by genetic testing.
Regulatory milestones
No milestones recorded in the last year.
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Information from official EMA, FDA, Federal Register and SEC records. Dates marked company-announced come from the company's own SEC filing; dates marked estimate are calculated, not official. Not investment or medical advice; always confirm on the source.