October 30, 2024

FDA Accepts Translarna NDA Resubmission for Review

A Letter From PPMD’s Pat Furlong I am thrilled to share that the FDA has officially accepted PTC Therapeutics’ New Drug Application (NDA) resubmission for Translarna (ataluren). This acceptance is an important step, meaning the…

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August 12, 2024

PPMD Urges FDA to Conduct a Full Review of Ataluren (Translarna) Following Resubmission of NDA

PTC Therapeutics has shared that it has resubmitted its New Drug Application (NDA) to the FDA for review and potential approval of ataluren (Translarna). Ataluren is an oral small molecule treatment that allows for stop-codon…

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May 20, 2024

PTC Therapeutics Announces European Commission Returns Translarna™ Opinion to CHMP For Re-evaluation

PTC Therapeutics, Inc. announced today that the European Commission (EC) has decided not to adopt the Committee for Medicinal Products for Human Use’s (CHMP) January 2024 negative opinion on the annual renewal of the conditional…

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March 19, 2024

PTC Therapeutics Announces Plans to Resubmit Translarna™ NDA to FDA

PTC Therapeutics, Inc. has announced that based on recent feedback from the FDA, the company plans to re-submit a New Drug Application (NDA) for Translarna™ (ataluren), a dystrophin restoration therapy via stop-codon readthrough for the…

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January 25, 2024

CHMP Issues Negative Opinion for Renewal of Conditional Marketing Authorization for Translarna™ (ataluren) in Europe

PPMD is disappointed to learn that the European Medicines Agency (EMA)’s Committee for Medicinal Products for Human Use (CHMP) today issued a negative opinion following the re-examination procedure for the conditional marketing authorization of Translarna™…

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December 5, 2023

PTC Therapeutics Shares Update on Translarna™ Regulatory Activities

PTC Therapeutics, Inc. has shared an update on regulatory activities in Europe and the United States for Translarna™ (ataluren), a treatment for Duchenne patients with nonsense variants.  Europe: European Medicines Agency (EMA) Update Following the…

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September 15, 2023

PTC Therapeutics Announces Negative European Medicines Agency Opinion Regarding Full Authorization of Translarna

PTC Therapeutics today shared that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended not renewing the Marketing Authorization for Translarna™ (ataluren), a treatment for Duchenne patients…

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January 18, 2023

PPMD Urges FDA to Conduct a Full Review of Ataluren (Translarna)

Last month, PPMD held a webinar with PTC Therapeutics for a discussion regarding PTC’s history with the community and plans for the future, including activities to bring ataluren (Translarna) to US patients. Ataluren is an…

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December 19, 2022

WATCH: A Conversation with PTC Therapeutics (Webinar Recording)

WATCH: A Conversation with PTC Therapeutics (Webinar Recording) PTC Therapeutics recently joined PPMD for a community webinar on Friday, December 9, 2022 for a discussion regarding PTC’s history with the community and plans for the…

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November 21, 2022

PTC Shares Community Letter Updating on Activities To Bring Ataluren (Translarna) To US Patients

PTC Therapeutics has shared a community letter updating on the company’s activities to bring ataluren (Translarna) to US patients with nonsense mutations. PPMD will host a community webinar with PTC on Friday, December 9th at…

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