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WARREN, N.J. - The U.S. Food and Drug Administration has approved Sephience (sepiapterin) for the treatment of hyperphenylalaninemia in patients with phenylketonuria (PKU), PTC Therapeutics, Inc. (NASDAQ:PTCT) announced Monday. The company, currently valued at $3.51 billion, has demonstrated strong financial performance with a 91% revenue growth over the last twelve months and maintains a healthy 67% gross profit margin, according to InvestingPro data.
The approval covers both adult and pediatric patients one month of age and older with sepiapterin-responsive PKU. Patients must use the medication in conjunction with a phenylalanine-restricted diet.
The FDA’s decision was based on results from the Phase 3 APHENITY clinical trial and its long-term extension study, which demonstrated the drug’s efficacy and safety profile.
"The broad labeling reflects the potential of Sephience to meet the significant unmet need of PKU patients," said Matthew B. Klein, Chief Executive Officer of PTC Therapeutics, in a press release statement.
PKU is a rare inherited metabolic disorder caused by a defect in the gene that helps create the enzyme needed to break down phenylalanine, an essential amino acid found in proteins. Without treatment, phenylalanine can build up to harmful levels in the body, potentially causing permanent intellectual disability, seizures, and other serious complications. For detailed analysis of PTC Therapeutics’ market position and growth potential, investors can access comprehensive Pro Research Reports available exclusively on InvestingPro, which covers over 1,400 US stocks with expert insights and actionable intelligence.
Catherine Warren, Executive Director of the National PKU Alliance, called the approval "an exciting milestone for the PKU community" that brings "renewed hope" for patients.
The medication works as a natural precursor of the enzymatic co-factor BH4, which is critical for phenylalanine hydroxylase function. Through this mechanism, Sephience reduces blood phenylalanine levels.
Common adverse reactions reported in clinical trials included diarrhea, headache, abdominal pain, hypophenylalaninemia, feces discoloration, and oropharyngeal pain.
Sephience recently received marketing authorization from the European Commission, with regulatory reviews ongoing in several other countries including Japan and Brazil.
In other recent news, PTC Therapeutics has received significant attention from several investment firms due to recent developments. The European Union’s approval of the company’s drug Sephience has led Evercore ISI to reiterate its Outperform rating, highlighting the potential impact on the upcoming PDUFA date for sepiapterin in the United States. UBS has also maintained a Buy rating with a $71 price target, as PTC Therapeutics awaits an FDA decision on sepiapterin for phenylketonuria (PKU). Truist Securities initiated coverage with a Buy rating and an $80 price target, citing the anticipated launches of sepiapterin and vatiquinone as key growth drivers. RBC Capital raised its price target from $58 to $60, maintaining an Outperform rating, based on expectations of favorable payer responses ahead of the sepiapterin PDUFA date. Meanwhile, Citi analysts have maintained a Neutral rating with a $40 price target, focusing on the ongoing evaluation of PTC518 for Huntington’s disease. These developments underscore the diverse perspectives of analysts regarding PTC Therapeutics’ future prospects.
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