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[{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"PTC Therapeutics Announces CHMP Recommendation of Translarna Label Update for Non-Ambulatory Patients with Duchenne Muscular Dystrophy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"June 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"PTC Therapeutics to Host Call to Review Results from its Study of Translarna in Nonsense Mutation Duchenne Muscular Dystrophy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"STRIDE Data Show Translarna\u2122 Delays Loss of Ambulation by More Than Five Years in Boys with Nonsense Mutation Duchenne Muscular Dystrophy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Blackstone Life Sciences","pharmaFlowCategory":"D","amount":"$50.0 million","upfrontCash":"Undisclosed","newsHeadline":"PTC Therapeutics Announces Strategic Financing Collaboration with Blackstone with up to $1 Billion in Funding","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"October 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"PTC Announces Expansion of Translarna\u2122 Label to Include Ambulatory Patients as Young as 2 Years Old in Brazil","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"October 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"PTC Therapeutics to Host Call to Review Topline Results from Study 041 for Translarna\u2122 (ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"NICE","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"NICE Review Does Not Recommend Routine Funding For Duchenne Muscular Dystrophy Gene Therapy Ataluren In Draft Guidance","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"PTC Therapeutics to Present Clinical and Real-World Evidence on Translarna\u2122 at the Annual Congress of the World Muscle Society","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"October 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"PTC Therapeutics Provides Updates on Translarna Regulatory Activities","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"December 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"CHMP Issues Negative Opinion for Renewal of Conditional Marketing Authorization for Translarna\u2122 (ataluren) Following Re-examination Procedure","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2024","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"}]

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            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 III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 25, 2024

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            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: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable December 05, 2023

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            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: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: Blackstone Life Sciences

            Deal Size: $50.0 million Upfront Cash: Undisclosed

            Deal Type: Collaboration October 27, 2022

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            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: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable October 11, 2022

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            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 III Product Type: Small molecule

            Partner/Sponsor/Collaborator: NICE

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable September 30, 2022

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            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 III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 20, 2022

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            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 II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable October 25, 2021

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            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 II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable September 20, 2021

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            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 II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 04, 2021

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            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: Approved Product 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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