On Tuesday, Citi reaffirmed its Sell rating and a $26.00 stock price target for NASDAQ:PTCT, PTC Therapeutics (NASDAQ:PTCT). The focus of this confirmation was the U.S. Food and Drug Administration's (FDA) recent acceptance of the New Drug Application (NDA) for sepiapterin, a treatment developed by PTC (NASDAQ:PTC) for phenylketonuria (PKU). The FDA has scheduled the Prescription Drug User Fee Act (PDUFA) target action date for July 29, 2025, which is based on the standard 10-month review timeline.
PTC Therapeutics previously sought a priority review for sepiapterin, which would have resulted in a shorter, 6-month review period. However, the FDA's decision indicates a standard review process, extending the timeline by an additional four months. This is in contrast to Biomarin's PKU therapies, Kuvan and Palynziq, which both received priority reviews in 2007 and 2017, respectively.
The analyst noted that sepiapterin's Marketing Authorization Application (MAA) is currently under review in Europe, having been validated and accepted in May. PTC Therapeutics is also preparing to submit marketing applications in other regions, with plans to file in countries including Japan and Brazil within the current year.
The FDA's acceptance of the NDA is a critical step for PTC in bringing sepiapterin to the market for PKU patients. The longer review process reflected in the July 2025 PDUFA date is a significant detail for investors monitoring the progress and potential market entry of the treatment.
In other recent news, PTC Therapeutics announced the FDA's target date of July 29, 2025, for the review of its New Drug Application for sepiapterin, a potential treatment for phenylketonuria (PKU). The company also reported Q2 2024 revenue of $187 million and revised its full-year revenue guidance to range from $700 million to $750 million.
Analyst firms Raymond James, Baird, Goldman Sachs, and TD Cowen have assessed the company, 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.
PTC Therapeutics also completed the sale of its gene therapy manufacturing business, receiving an upfront payment of $27.5 million. The FDA granted Fast Track Designation to the company's drug candidate PTC518, developed for Huntington's disease treatment. These are the recent developments surrounding PTC Therapeutics.
InvestingPro Insights
PTC Therapeutics' recent FDA acceptance of its NDA for sepiapterin aligns with several key financial indicators and market trends. According to InvestingPro data, the company's market capitalization stands at $3.01 billion, reflecting investor confidence in its pipeline and potential. Despite the extended FDA review timeline, PTC has shown strong market performance, with a remarkable 91.96% price total return over the past year and a 52.41% return in the last six months.
InvestingPro Tips highlight that PTC is trading near its 52-week high, with the current price at 93.61% of that peak. This suggests that investors remain optimistic about the company's prospects, including the potential of sepiapterin. However, it's important to note that analysts anticipate a sales decline in the current year, which could be influenced by the extended timeline for sepiapterin's review.
The company's financial health shows mixed signals. While liquid assets exceed short-term obligations, indicating good short-term financial stability, PTC is not currently profitable. The revenue for the last twelve months as of Q2 2024 was $900.45 million, with a 9.98% growth rate, but the company reported an adjusted operating income of -$192.45 million for the same period.
For investors seeking more comprehensive analysis, InvestingPro offers 11 additional tips for PTC Therapeutics, providing a deeper understanding of the company's financial position and market outlook.
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