European Commission Requests Reevaluation Of Opinion On PTC Therapeutics' Duchenne Dystrophy Drug; Pauses Annual Guidance
European Commission Requests Reevaluation Of Opinion On PTC Therapeutics' Duchenne Dystrophy Drug; Pauses Annual Guidance
Monday, PTC Therapeutics Inc (NASDAQ:PTCT) announced that the European Commission (EC) has decided not to adopt the CHMP's negative opinion on the annual renewal of the conditional marketing authorization of Translarna (ataluren) and has returned the opinion to the CHMP for re-evaluation.
週一，PTC Therapeutics Inc（納斯達克股票代碼：PTCT）宣佈，歐盟委員會（EC）已決定不採納CHMP對Translarna（ataluren）有條件上市許可年度續訂的否定意見，並已將該意見退回CHMP進行重新評估。
As a result, Translarna remains on the market and available for patients in Europe, consistent with its current marketing authorization.
因此，根據其目前的上市許可，Translarna仍在市場上供歐洲患者使用。
The EC has asked the CHMP to further consider the totality of evidence, including data from patient registries and real-world evidence, in a revised opinion.
歐盟委員會已要求CHMP在修訂後的意見中進一步考慮所有證據，包括來自患者登記處的數據和現實世界的證據。
In addition, the European Medicines Agency (EMA) has informed PTC that it has decided to consider the Scientific Advisory Group (SAG) meeting for Translarna held on September 5, 2023, and all the procedural steps that followed as invalid.
此外，歐洲藥品管理局（EMA）已通知PTC，它已決定將2023年9月5日舉行的Translarna科學諮詢小組（SAG）會議以及隨後的所有程序步驟視爲無效。
Furthermore, the input from the SAG meeting held in September 2023 and the meeting held in January 2024 will not be considered by the CHMP in any future evaluation of Translarna.
此外，CHMP在未來對Translarna進行的任何評估中都不會考慮2023年9月舉行的SAG會議和2024年1月舉行的會議的意見。
In January, the Committee for Medicinal Products for Human Use (CHMP) of the EMA issued a negative opinion following the re-examination procedure for the conditional marketing authorization of Translarna.
1月，EMA人用藥品委員會（CHMP）在對Translarna的有條件上市許可進行重新審查後發表了否定意見。
In September 2023, the CHMP gave a negative opinion on converting the conditional marketing authorization to full marketing authorization of Translarna (ataluren) for nonsense mutation Duchenne muscular dystrophy.
2023年9月，CHMP對將Translarna（ataluren）針對胡說八道的突變杜興氏肌肉萎縮症的有條件上市許可轉換爲完全的上市許可給出了否定意見。
Given the inability to accurately forecast the upside impact of the continued authorization of Translarna in Europe on 2024 revenue, PTC will pause 2024 total revenue guidance of $600 million-$680 million.
鑑於無法準確預測Translarna在歐洲的持續授權對2024年收入的上行影響，PTC將暫停2024年6億至6.8億美元的總收入預期。
Translarna, the tradename of ataluren, is licensed in multiple countries for nonsense mutation Duchenne muscular dystrophy in ambulatory patients (who can walk) aged two years and older.
Translarna的商品名爲ataluren，已在多個國家獲得許可，用於治療兩歲及以上的臥牀患者（會走路）的胡說八道突變杜氏肌肉萎縮症。
PTC plans to resubmit the U.S. marketing application for Translarna for nmDMD in mid-2024.
PTC計劃在2024年中期重新提交Translarna的NMDMD在美國的上市申請。
Translarna net product revenues were $103.6 million for the first quarter of 2024.
2024年第一季度，Translarna的淨產品收入爲1.036億美元。
Price Action: PTCT shares are up 18.3% at $39.16 at last check Monday.
價格走勢：週一最後一次檢查時，PTCT股價上漲18.3％，至39.16美元。
Photo via Shutterstock
照片來自 Shutterstock