WARREN, N.J. - PTC Therapeutics, Inc. (NASDAQ:PTCT) today faces a setback as the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency upheld its negative opinion on the renewal of the conditional marketing authorization for Translarna™ (ataluren), a treatment for nonsense mutation Duchenne Muscular Dystrophy (nmDMD). This decision follows a re-examination and will now be forwarded to the European Commission (EC) for a final verdict, expected in approximately 67 days.
Matthew B. Klein, M.D., Chief Executive Officer of PTC (NASDAQ:PTC) Therapeutics, expressed disappointment with the CHMP's decision, which he stated was based on a selective consideration of evidence from Study 041. According to Dr. Klein, a more comprehensive review of data from three placebo-controlled trials and the STRIDE registry supports Translarna's efficacy and safety. He emphasized that the drug will remain available in Europe while the EC review is pending and assured that all possible evidence will be provided to support continued authorization.
Translarna is a protein restoration therapy aimed at treating genetic disorders caused by nonsense mutations, which prematurely stop the synthesis of essential proteins. It is currently licensed in multiple countries for treating nmDMD in ambulatory patients aged two years and older but remains an investigational new drug in the United States.
Duchenne Muscular Dystrophy is a rare and fatal genetic disorder characterized by progressive muscle weakness, leading to loss of mobility and premature death due to heart and respiratory failure. PTC Therapeutics focuses on developing medicines for rare disorders and aims to provide treatments for patients with limited options.
The company's future steps regarding Translarna and its engagement with the EMA and EC remain crucial, as the EC's decision will impact the drug's availability in Europe. This news is based on a press release statement from PTC Therapeutics.
In other recent news, PTC Therapeutics has been making strides in the pharmaceutical industry. The U.S. Food and Drug Administration (FDA) accepted the company's New Drug Application for sepiapterin, a potential treatment for phenylketonuria (PKU), and set the review target date for July 29, 2025. This follows standard review procedure, extending the timeline by an additional four months. Sepiapterin's Marketing Authorization Application is currently under review in Europe, and the company plans to submit marketing applications in other regions, including Japan and Brazil, within the year.
Analyst firms have been keeping a close eye on PTC Therapeutics, with Raymond James issuing a Market Perform rating, Baird reaffirming an Outperform rating, Goldman Sachs maintaining a Sell rating, and TD Cowen holding steady with a Hold rating. These ratings come amidst PTC Therapeutics' recent earnings report, where the company announced Q2 2024 revenue of $187 million and revised its full-year revenue guidance to range from $700 million to $750 million.
In addition to these developments, PTC Therapeutics completed the sale of its gene therapy manufacturing business, receiving an upfront payment of $27.5 million. The FDA also granted Fast Track Designation to the company's drug candidate PTC518, developed for Huntington's disease treatment. These are among the recent developments surrounding PTC Therapeutics.
InvestingPro Insights
As PTC Therapeutics faces regulatory challenges with Translarna in Europe, InvestingPro data provides additional context to the company's financial situation. Despite the setback, PTC has shown a strong market performance with a 80.54% price total return over the past year and a significant 49.9% increase in the last six months. This suggests that investors have been optimistic about the company's prospects, possibly driven by its focus on rare disease treatments.
However, the company's financial health presents a mixed picture. While PTC's revenue for the last twelve months as of Q2 2023 stood at $900.45 million, with a 9.98% growth, it's important to note that the company is not currently profitable. This aligns with an InvestingPro Tip indicating that analysts do not anticipate the company will be profitable this year.
Another InvestingPro Tip highlights that PTC's liquid assets exceed short-term obligations, which could provide some financial flexibility as the company navigates the regulatory landscape for Translarna. This could be crucial as PTC awaits the European Commission's final decision and potentially explores alternative strategies for the drug's approval and commercialization.
For investors seeking a more comprehensive analysis, InvestingPro offers 8 additional tips for PTC Therapeutics, providing deeper insights into the company's financial health and market position.
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