Drug Profile
Ataluren - PTC Therapeutics
Alternative Names: PTC 124; TranslarnaLatest Information Update: 22 Mar 2024
Price :
$50
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At a glance
- Originator PTC Therapeutics
- Developer PTC Therapeutics; Sanofi Genzyme
- Class Antianaemics; Antiepileptic drugs; Antifibrotics; Antihaemorrhagics; Eye disorder therapies; Oxadiazoles; Small molecules
- Mechanism of Action Cystic fibrosis transmembrane conductance regulator stimulants; Protein synthesis stimulants
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Orphan Drug Status
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
- New Molecular Entity Yes
Highest Development Phases
- Marketed Duchenne muscular dystrophy
- Phase II Mucopolysaccharidosis I
- Preregistration Submission Withdrawal Cystic fibrosis
- Suspended Haemophilia A; Haemophilia B
- Discontinued Aniridia; Dravet syndrome; Epilepsy; Methylmalonic acidaemia
Most Recent Events
- 19 Mar 2024 PTC plans to re-submit an NDA for Ataluren for Duchenne muscular dystrophy by mid of 2024
- 19 Mar 2024 PTC Therapeutics plans to hold a follow-up meeting with the US FDA for ataluren's NDA in March 2024
- 25 Jan 2024 Committee for Medicinal Products for Human Use (CHMP) issued a negative opinion following the re-examination procedure for the conditional marketing authorization of Ataluren