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Details:
Translarna (ataluren) is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation. It is under phase 3 clinical development for the treatment of nonsense mutation Duchenne Muscular Dystrophy.
Lead Product(s): Ataluren
Therapeutic Area: Genetic Disease Product Name: Translarna
Highest Development Status: Phase IIIProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable January 25, 2024
Details:
Translarna (ataluren) is a dystrophin expression stimulator small molecule drug candidate, which is currently being evaluated for the treatment of duchenne muscular dystrophy from a nonsense mutated dystrophin gene, in patients aged 2 or above via oral suspension.
Lead Product(s): Ataluren
Therapeutic Area: Genetic Disease Product Name: Translarna
Highest Development Status: ApprovedProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable December 05, 2023
Details:
The strategic financing will support the acceleration of PTC’s robust and diversified pipeline having lead asset Translarna (ataluren), business development opportunities and general corporate purposes.
Lead Product(s): Ataluren
Therapeutic Area: Genetic Disease Product Name: Translarna
Highest Development Status: ApprovedProduct Type: Small molecule
Partner/Sponsor/Collaborator: Blackstone Life Sciences
Deal Size: $50.0 million Upfront Cash: Undisclosed
Deal Type: Collaboration October 27, 2022
Details:
Translarna (ataluren), discovered and developed by PTC Therapeutics, is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation.
Lead Product(s): Ataluren
Therapeutic Area: Genetic Disease Product Name: Translarna
Highest Development Status: ApprovedProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable October 11, 2022
Details:
Translarna (ataluren) is the first licensed treatment for DMD that addresses the loss of dystrophin. It works by allowing the body to read over the mutation in the DNA and continue to produce dystrophin.
Lead Product(s): Ataluren
Therapeutic Area: Genetic Disease Product Name: Translarna
Highest Development Status: Phase IIIProduct Type: Small molecule
Partner/Sponsor/Collaborator: NICE
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable September 30, 2022
Details:
Translarna (ataluren), discovered and developed by PTC Therapeutics, Inc., is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation.
Lead Product(s): Ataluren
Therapeutic Area: Genetic Disease Product Name: Translarna
Highest Development Status: Phase IIIProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable June 20, 2022
Details:
Translarna is the only treatment for the underlying cause of Duchenne caused by a nonsense mutation. Clinical trials and real-world evidence have demonstrated Translarna's potential to slow disease progression and improve patient outcomes.
Lead Product(s): Ataluren
Therapeutic Area: Genetic Disease Product Name: Translarna
Highest Development Status: Phase IIProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable October 25, 2021
Details:
Real-world results from the STRIDE patient registry demonstrating that treatment with Translarna™ (ataluren) delays loss of ambulation by more than five years in boys with nonsense mutation Duchenne muscular dystrophy (nmDMD) compared to standard of care (SoC) alone.
Lead Product(s): Ataluren
Therapeutic Area: Genetic Disease Product Name: Translarna
Highest Development Status: Phase IIProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable September 20, 2021
Details:
PTC Therapeutics is planning to hold talks with the US FDA on approval after the latest Phase II Study 045 of its drug, Translarna (ataluren), failed to significantly improve dystrophin expression in patients with nonsense mutation Duchenne muscular dystrophy.
Lead Product(s): Ataluren
Therapeutic Area: Genetic Disease Product Name: Translarna
Highest Development Status: Phase IIProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable February 04, 2021
Details:
Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended by a majority of votes to remove the statement "efficacy has not been demonstrated in non-ambulatory patients" from the SmPC for Translarna™ (ataluren).
Lead Product(s): Ataluren
Therapeutic Area: Genetic Disease Product Name: Translarna
Highest Development Status: ApprovedProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable June 29, 2020
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