WARREN, N.J. - PTC Therapeutics, Inc. (NASDAQ: NASDAQ:PTCT) disclosed on Monday that the European Commission (EC) has refrained from adopting the Committee for Medicinal Products for Human Use's (CHMP) negative opinion concerning the annual renewal of Translarna™ (ataluren). The EC has returned the opinion to the CHMP for further assessment, thus maintaining Translarna's market presence in Europe under its existing authorization.
The EC's decision to remand the CHMP's January 24, 2024, opinion follows a request for additional evaluation of the totality of evidence, including patient registry data and real-world evidence. This development allows Translarna to continue being available to patients in Europe as per the current marketing authorization.
Matthew B. Klein, M.D., Chief Executive Officer of PTC (NASDAQ:PTC) Therapeutics, commented on the significance of maintaining Translarna's authorization for individuals with nonsense mutation Duchenne muscular dystrophy (nmDMD) in Europe. Klein highlighted the clinical trials and STRIDE registry data, indicating Translarna's role in addressing the unmet medical needs of nmDMD patients.
The European Medicines Agency (EMA) has also informed PTC that the proceedings from the Scientific Advisory Group (SAG) meetings on Translarna held on September 5, 2023, and January 2024, will not be considered in future evaluations of the drug by the CHMP.
Due to the uncertainties surrounding the impact of Translarna's continued authorization on the company's 2024 revenues, PTC has decided to pause its revenue guidance for the year. The company will provide updated guidance at a later date.
Translarna, developed by PTC Therapeutics, is a protein restoration therapy intended for patients with genetic disorders caused by nonsense mutations, which can lead to the premature cessation of essential protein synthesis. The drug is approved in various countries for treating nmDMD in ambulatory patients aged two years and older. In the United States, ataluren remains an investigational new drug.
Duchenne muscular dystrophy is a rare, fatal genetic disorder primarily affecting males, characterized by progressive muscle weakness leading to early loss of ambulation and premature death due to cardiac and respiratory failure.
PTC Therapeutics is a global biopharmaceutical company focused on discovering, developing, and commercializing medicines for rare disorders. The company emphasizes its commitment to providing access to treatments for patients with limited or no available options.
This article is based on a press release statement from PTC Therapeutics.
InvestingPro Insights
In light of PTC Therapeutics' recent developments with Translarna™ in Europe, it's important to consider the company's financial health and market performance. According to the latest data from InvestingPro, PTC Therapeutics has a market capitalization of 2540 million USD, reflecting its current valuation in the market. Despite the challenges, the company has shown a strong return over the last three months, with a 27.39% increase. Additionally, the last month has seen an even more impressive surge, with a 30.71% return.
However, PTC Therapeutics faces financial headwinds. Analysts, as noted in InvestingPro Tips, do not expect the company to be profitable this year, and it has not been profitable over the last twelve months. This is further supported by a negative P/E ratio of -4.34, indicating that the company is currently not generating earnings to justify its share price. Furthermore, the company does not pay a dividend, which might be a consideration for income-focused investors.
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With seven additional InvestingPro Tips listed on InvestingPro, investors can gain a more comprehensive view of PTC Therapeutics' position in the biopharmaceutical industry, especially in the context of its ongoing efforts to secure and maintain market authorization for its flagship product, Translarna™.
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