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WARREN, N.J. - PTC Therapeutics, Inc. (NASDAQ: PTCT), a biopharmaceutical company with a market capitalization of $3.91 billion and an impressive 94% stock return over the past year according to InvestingPro, has received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) for Sephience™ (sepiapterin) to treat phenylketonuria (PKU) in children and adults. The endorsement, announced today, encompasses all ages and disease severities, paving the way for its European launch.
Sephience is designed to reduce blood phenylalanine (Phe) levels in PKU patients, a rare metabolic disease that can lead to severe and irreversible disabilities if untreated. The drug’s dual mechanism of action involves serving as a precursor to tetrahydrobiopterin (BH4), a critical cofactor for the phenylalanine hydroxylase (PAH) enzyme, and as a pharmacological chaperone to correct PAH misfolding.
Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics, expressed optimism about the CHMP’s opinion, citing the significant unmet medical need for the PKU patient community in Europe. The company anticipates the European Commission to confirm the marketing authorization in about two months, with the decision applicable to all 27 European Union member states and the European Economic Area countries.
The European launch strategy focuses on Germany and other key markets, where access for named patients will be available immediately after authorization. With a healthy current ratio of 2.35, indicating strong liquidity to support the launch, and annual revenue of $806.78 million, PTC appears well-positioned for expansion. Meanwhile, the U.S. Food and Drug Administration (FDA) is reviewing the New Drug Application (NDA) for sepiapterin, with a decision expected by July 29, 2025.
Globally, an estimated 58,000 people live with PKU, a condition diagnosed typically during newborn screening. PTC Therapeutics specializes in developing and commercializing medicines for rare disorders, and Sephience represents a potential new treatment option for a broad range of PKU patients. According to InvestingPro analysis, while currently showing negative EBITDA, the company’s strong financial health score and upcoming earnings report on May 6, 2025, could provide additional insights into its growth trajectory. For deeper analysis, investors can access the comprehensive Pro Research Report, available exclusively to InvestingPro subscribers. This development is based on a press release statement from PTC Therapeutics.
In other recent news, PTC Therapeutics has faced a regulatory challenge as the European Commission chose not to renew the marketing authorization for Translarna in Europe. This decision follows a negative opinion from the European Medicines Agency’s Committee for Medicinal Products for Human Use. Despite this setback, PTC Therapeutics continues to explore options to provide access to Translarna through individual EU member states. Meanwhile, the company has reported progress in its Phase 3 APHENITY trial for treating phenylketonuria (PKU) with sepiapterin, showing promising results in dietary liberalization for patients.
In terms of analyst ratings, BofA Securities upgraded PTC Therapeutics from Underperform to Neutral, raising the price target to $55, reflecting optimism due to the FDA’s expedited review of vatiquinone for treating Friedrich’s ataxia. Scotiabank initiated coverage with a Sector Perform rating and a $55 price target, suggesting the stock is fairly valued. Cantor Fitzgerald maintained its Overweight rating with a $113 price target, adjusting its financial model to align with PTC Therapeutics’ guidance for 2025. These recent developments indicate a mixed but cautiously optimistic outlook from analysts regarding PTC Therapeutics’ future performance.
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