PTC Therapeutics Says European Commission Returns Translarna Opinion To CHMP For Re-evaluation

RTTNews | 358 days ago
PTC Therapeutics Says European Commission Returns Translarna Opinion To CHMP For Re-evaluation

(RTTNews) - PTC Therapeutics, Inc. (PTCT) announced Monday that the European Commission (EC) has decided not to adopt the CHMP's negative opinion of January 24, 2024 on the annual renewal of the conditional marketing authorization of Translarna (ataluren) and has returned the opinion to the CHMP for re-evaluation.

As a result, Translarna remains on the market and available for patients in Europe consistent with its current marketing authorization. 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.

In addition, the European Medicines Agency (EMA) has informed PTC that the decision has been taken to consider the Scientific Advisory Group (SAG) meeting for Translarna held on September 5, 2023, and all the procedural steps that followed, as invalid.

Furthermore, the input from the SAG meeting held in September 2023 as well as the meeting held in January 2024 will not be considered by the CHMP in any future evaluation of Translarna.

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 at this time.

read more
FDA Approves PTC's Kebilidi As First Gene Therapy To Treat AADC Deficiency

FDA Approves PTC's Kebilidi As First Gene Therapy To Treat AADC Deficiency

The U.S. Food and Drug Administration approved PTC Therapeutics, Inc.'s Kebilidi (eladocagene exuparvovec-tneq) gene therapy for the treatment of aromatic L-amino acid decarboxylase or AADC deficiency. Kebilidi is the first FDA-approved gene therapy for the deficiency. PTC shares gained around 8.5 percent in the extended trading on the Nasdaq on Thursday.
RTTNews | 179 days ago
PTC Therapeutics Granted Marketing Authorization By EU For Upstaza To Treat AADC Deficiency

PTC Therapeutics Granted Marketing Authorization By EU For Upstaza To Treat AADC Deficiency

PTC Therapeutics, Inc. (PTCT) announced Wednesday that Upstaza (eladocagene exuparvovec) was granted marketing authorization by the European Commission. Upstaza is the first approved disease-modifying treatment for aromatic L-amino acid decarboxylase (AADC) deficiency and the first marketed gene therapy directly infused into the brain. It is approved for patients 18 months and older.
RTTNews | 1028 days ago
Swiss Market Ends On Weak Note

Swiss Market Ends On Weak Note

After a flat start and a subsequent modest upmove, the Switzerland market pared gains and drifted down into negative territory about an hour past noon and finally settled on a weak note. A lack of fresh triggers, and uncertainty about U.S.-European Union trade deal rendered the mood cautious.
RTTNews | 13 minutes ago
European Stocks Close Slightly Higher After Cautious Session

European Stocks Close Slightly Higher After Cautious Session

European stocks closed higher on Tuesday, lifted by easing trade tensions, fairly buoyant German investor sentiment report and data showing a less than expected increase in U.S. consumer prices in the month of April.
RTTNews | 28 minutes ago
U.S. Consumer Prices Rise 0.2% In April, Slightly Less Than Expected

U.S. Consumer Prices Rise 0.2% In April, Slightly Less Than Expected

Consumer prices in the U.S. rose by slightly less than expected in the month of April, according to a report released by the Labor Department on Tuesday. The Labor Department said its consumer price index inched up by 0.2 percent in April after edging down by 0.1 percent in March. Economists had expected consumer prices to rise by 0.3 percent.
RTTNews | 4h 2min ago
Bayer Q1 Results Down, Sees Pharma Results At Top End Of View; Stock Gains

Bayer Q1 Results Down, Sees Pharma Results At Top End Of View; Stock Gains

Bayer AG on Tuesday confirmed its currency-adjusted 2025 targets after reporting lower earnings in its first quarter with slightly lower sales. The quarterly results reflected weak results in the Crop Science Division, despite growth in Pharmaceuticals and Consumer Health segments. Shares of the Pharmaceutical and life sciences major were gaining around 6 percent on the XETRA in Germany.
RTTNews | 4h 55min ago
Bay Street May Open On Subdued Note; U.S. Inflation Data In Focus

Bay Street May Open On Subdued Note; U.S. Inflation Data In Focus

Futures indicate a somewhat subdued start for the Canadian market on Tuesday. However, U.S. consumer price inflation data is likely to make an impact on stocks as the inflation numbers from the world's largest economy could give some clues about the Federal Reserve's policy moves.
RTTNews | 5h 16min ago