Royalty Pharma Acquires Evrysdi Royalty for $240 Million Upfront
Written by Emily J. Thompson, Senior Investment Analyst
Updated: Dec 31 2025
0mins
Should l Buy PTCT?
Source: Benzinga
- Stock Performance: Royalty Pharma's stock has surged approximately 53% year-to-date, reflecting strong market confidence in its acquisition strategy and enhancing investor sentiment.
- Acquisition Details: Royalty Pharma acquired the remaining Evrysdi royalty from PTC Therapeutics for $240 million upfront, with potential sales-based milestones of up to $60 million, further solidifying its position in the SMA treatment market.
- Sales Growth Potential: Evrysdi generated approximately 1.6 billion Swiss francs ($1.9 billion) in sales in 2024, representing an 18% year-over-year growth, and is projected to reach 2.3 billion Swiss francs ($2.9 billion) by 2030, promising substantial long-term revenue for Royalty Pharma.
- Competitive Landscape: With Biogen and Novartis also advancing their products in the SMA treatment space, Royalty Pharma's acquisition positions it favorably in the competitive landscape, enhancing its market share.
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Analyst Views on PTCT
Wall Street analysts forecast PTCT stock price to rise
14 Analyst Rating
8 Buy
5 Hold
1 Sell
Moderate Buy
Current: 67.760
Low
54.00
Averages
78.18
High
118.00
Current: 67.760
Low
54.00
Averages
78.18
High
118.00
About PTCT
PTC Therapeutics, Inc. is a global biopharmaceutical company. The Company is focused on the discovery, development, and commercialization of clinically differentiated medicines that provide benefits to children and adults living with rare disorders. Its diversified therapeutic portfolio includes several commercial products and product candidates in various stages of development, including discovery, research and clinical stages, focused on the development of new treatments for multiple therapeutic areas for rare diseases relating to neurology and metabolism. It has two products, Translarna (ataluren) and Emflaza (deflazacort), for the treatment of Duchenne muscular dystrophy (DMD) a rare, life-threatening disorder. Its Upstaza, a gene therapy for the treatment of Aromatic L-Amino Decarboxylase (AADC) deficiency, a rare central nervous system (CNS) disorder. Its Tegsedi and Waylivra are for the treatment of rare diseases. Its Evrysdi, a treatment for spinal muscular atrophy (SMA).
About the author
Emily J. Thompson
Emily J. Thompson, a Chartered Financial Analyst (CFA) with 12 years in investment research, graduated with honors from the Wharton School. Specializing in industrial and technology stocks, she provides in-depth analysis for Intellectia’s earnings and market brief reports.
PTC Therapeutics Announces Upcoming Conference Schedule
- Conference Schedule: PTC Therapeutics will participate in three significant healthcare conferences on March 2, March 10, and March 11, 2026, showcasing its latest advancements in rare disease treatments.
- Live Webcast: Presentations will be webcast live on PTC's investor section of its website and archived for 30 days post-event, ensuring that investors unable to attend can still access the information.
- Company Mission: PTC is dedicated to providing clinically differentiated medicines for patients with rare disorders, advancing a diversified pipeline to address unmet medical needs, highlighting its strategic positioning in the biopharmaceutical sector.
- Science and Business Integration: The company plans to leverage its scientific expertise and global commercial infrastructure to optimize value for patients and stakeholders, further solidifying its competitive advantage in the biopharmaceutical industry.
See More
PTC Therapeutics Q4 2025 Earnings Call Highlights
- Successful Global Launch: PTC Therapeutics' Sephience received approvals and launched in the U.S., EU, and Japan, generating $92 million in revenue in Q4 2025, establishing itself as a cornerstone for future growth, indicating strong market demand and broad patient acceptance.
- Financial Performance Exceeds Expectations: The company reported $263 million in net product and royalty revenue for Q4 2025, with total revenue for the year reaching $831 million, surpassing the guidance of $750 million to $800 million, reflecting exceptional execution in product sales and cost management.
- Strong Cash Position: PTC Therapeutics ended 2025 with $1.95 billion in cash, bolstered by the $240 million sale of Evrysdi royalties, ensuring financial flexibility for future R&D and market expansion initiatives.
- Optimistic 2026 Outlook: The company anticipates product revenue of $700 million to $800 million in 2026, representing a year-over-year growth of 19% to 36%, with potential for cash flow breakeven, demonstrating management's confidence in future growth and effective strategic planning.
See More
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- Market Size Growth: The phenylketonuria treatment market is expected to continue growing by 2034, with the U.S. holding the largest market size, as evidenced by 18,800 diagnosed PKU cases in 2024, indicating strong demand and economic opportunities in this sector.
- Emerging Therapies Development: New therapies such as NGGT002, JNT-517, and Pegvaliase are currently in clinical trials and are anticipated to enter the market in the coming years, significantly improving treatment options for patients and driving market transformation.
- Increased Screening Rates: The expansion of newborn screening programs globally has led to higher early diagnosis rates of PKU, resulting in more patients entering care pathways, thereby enhancing the market's growth potential and meeting rising healthcare demands.
- Regulatory Support: The approvals of PTC Therapeutics' SEPHIENCE and BioMarin's Pegvaliase by the FDA and Japan's Ministry of Health signify an improved regulatory environment, providing favorable conditions for new therapies' market entry and fostering innovation and development in the industry.
See More
PTC Therapeutics Withdraws New Drug Application for Translarna
- Withdrawal of NDA: PTC Therapeutics has withdrawn its New Drug Application for Translarna (ataluren) after receiving FDA feedback indicating that the submitted data was unlikely to meet effectiveness standards, marking a significant setback after over two decades of development for this Duchenne muscular dystrophy (DMD) therapy.
- Poor Market Performance: PTC's stock closed 5.16% lower on Thursday, currently trading 10.0% below its 20-day simple moving average, indicating a bearish short-term trend, and significantly underperforming compared to the flat performance of the healthcare sector.
- Future Earnings Outlook: PTC is set to report earnings on February 19, 2026, with an estimated loss of 34 cents per share and revenue forecasted at $290.76 million, up from $213.17 million year-over-year, suggesting potential growth opportunities despite current challenges.
- Analyst Rating Changes: While PTC's stock carries a
See More
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- Application Withdrawal: PTC Therapeutics announced the withdrawal of its NDA resubmission for Translarna (ataluren) for nonsense mutation Duchenne muscular dystrophy, resulting in a ~5% drop in after-hours trading.
- FDA Feedback Impact: The decision was based on feedback from the US FDA regarding the application review, with CEO Matthew Klein stating that the data included is “unlikely to meet the Agency's threshold of substantial evidence of effectiveness.”
- European Market Status: Despite the withdrawal in the US, Translarna is approved in the European Union, highlighting regulatory differences across markets for the same drug.
- Future Revenue Outlook: PTC Therapeutics projects FY26 product revenue between $700 million and $800 million, indicating a positive outlook for the company despite the challenges posed by the FDA.
See More
PTC Therapeutics Withdraws NDA for Translarna Due to FDA Feedback
- FDA Feedback Impact: PTC Therapeutics announced the withdrawal of its NDA for Translarna (ataluren) for nonsense mutation Duchenne muscular dystrophy due to FDA feedback indicating that the submitted data is unlikely to meet effectiveness standards, resulting in the company being unable to secure approval after two decades of effort, which negatively impacts its market outlook.
- Drug Background: Translarna is a protein restoration therapy aimed at genetic disorders caused by nonsense mutations, primarily affecting patients with Duchenne muscular dystrophy who face severe health risks due to the lack of functional dystrophin protein, and the withdrawal of the application will limit treatment options for these patients.
- Market Impact: This withdrawal may diminish PTC's competitiveness in the biopharmaceutical market, particularly in the rare disease treatment sector, potentially affecting investor confidence and exerting downward pressure on the company's stock price.
- Future Outlook: Despite the withdrawal, PTC remains committed to developing innovative therapies for rare diseases and may reassess its R&D strategy in response to FDA feedback to seek approval for alternative treatment options.
See More
PTC Therapeutics Announces Upcoming Conference Schedule
- Conference Schedule: PTC Therapeutics will participate in three significant healthcare conferences on March 2, March 10, and March 11, 2026, showcasing its latest advancements in rare disease treatments.
- Live Webcast: Presentations will be webcast live on PTC's investor section of its website and archived for 30 days post-event, ensuring that investors unable to attend can still access the information.
- Company Mission: PTC is dedicated to providing clinically differentiated medicines for patients with rare disorders, advancing a diversified pipeline to address unmet medical needs, highlighting its strategic positioning in the biopharmaceutical sector.
- Science and Business Integration: The company plans to leverage its scientific expertise and global commercial infrastructure to optimize value for patients and stakeholders, further solidifying its competitive advantage in the biopharmaceutical industry.
See More
PTC Therapeutics Q4 2025 Earnings Call Highlights
- Successful Global Launch: PTC Therapeutics' Sephience received approvals and launched in the U.S., EU, and Japan, generating $92 million in revenue in Q4 2025, establishing itself as a cornerstone for future growth, indicating strong market demand and broad patient acceptance.
- Financial Performance Exceeds Expectations: The company reported $263 million in net product and royalty revenue for Q4 2025, with total revenue for the year reaching $831 million, surpassing the guidance of $750 million to $800 million, reflecting exceptional execution in product sales and cost management.
- Strong Cash Position: PTC Therapeutics ended 2025 with $1.95 billion in cash, bolstered by the $240 million sale of Evrysdi royalties, ensuring financial flexibility for future R&D and market expansion initiatives.
- Optimistic 2026 Outlook: The company anticipates product revenue of $700 million to $800 million in 2026, representing a year-over-year growth of 19% to 36%, with potential for cash flow breakeven, demonstrating management's confidence in future growth and effective strategic planning.
See More
Phenylketonuria Treatment Market Outlook Analysis
- Market Size Growth: The phenylketonuria treatment market is expected to continue growing by 2034, with the U.S. holding the largest market size, as evidenced by 18,800 diagnosed PKU cases in 2024, indicating strong demand and economic opportunities in this sector.
- Emerging Therapies Development: New therapies such as NGGT002, JNT-517, and Pegvaliase are currently in clinical trials and are anticipated to enter the market in the coming years, significantly improving treatment options for patients and driving market transformation.
- Increased Screening Rates: The expansion of newborn screening programs globally has led to higher early diagnosis rates of PKU, resulting in more patients entering care pathways, thereby enhancing the market's growth potential and meeting rising healthcare demands.
- Regulatory Support: The approvals of PTC Therapeutics' SEPHIENCE and BioMarin's Pegvaliase by the FDA and Japan's Ministry of Health signify an improved regulatory environment, providing favorable conditions for new therapies' market entry and fostering innovation and development in the industry.
See More
PTC Therapeutics Withdraws New Drug Application for Translarna
- Withdrawal of NDA: PTC Therapeutics has withdrawn its New Drug Application for Translarna (ataluren) after receiving FDA feedback indicating that the submitted data was unlikely to meet effectiveness standards, marking a significant setback after over two decades of development for this Duchenne muscular dystrophy (DMD) therapy.
- Poor Market Performance: PTC's stock closed 5.16% lower on Thursday, currently trading 10.0% below its 20-day simple moving average, indicating a bearish short-term trend, and significantly underperforming compared to the flat performance of the healthcare sector.
- Future Earnings Outlook: PTC is set to report earnings on February 19, 2026, with an estimated loss of 34 cents per share and revenue forecasted at $290.76 million, up from $213.17 million year-over-year, suggesting potential growth opportunities despite current challenges.
- Analyst Rating Changes: While PTC's stock carries a
See More
PTC Therapeutics Withdraws NDA Resubmission for Translarna
- Application Withdrawal: PTC Therapeutics announced the withdrawal of its NDA resubmission for Translarna (ataluren) for nonsense mutation Duchenne muscular dystrophy, resulting in a ~5% drop in after-hours trading.
- FDA Feedback Impact: The decision was based on feedback from the US FDA regarding the application review, with CEO Matthew Klein stating that the data included is “unlikely to meet the Agency's threshold of substantial evidence of effectiveness.”
- European Market Status: Despite the withdrawal in the US, Translarna is approved in the European Union, highlighting regulatory differences across markets for the same drug.
- Future Revenue Outlook: PTC Therapeutics projects FY26 product revenue between $700 million and $800 million, indicating a positive outlook for the company despite the challenges posed by the FDA.
See More
PTC Therapeutics Withdraws NDA for Translarna Due to FDA Feedback
- FDA Feedback Impact: PTC Therapeutics announced the withdrawal of its NDA for Translarna (ataluren) for nonsense mutation Duchenne muscular dystrophy due to FDA feedback indicating that the submitted data is unlikely to meet effectiveness standards, resulting in the company being unable to secure approval after two decades of effort, which negatively impacts its market outlook.
- Drug Background: Translarna is a protein restoration therapy aimed at genetic disorders caused by nonsense mutations, primarily affecting patients with Duchenne muscular dystrophy who face severe health risks due to the lack of functional dystrophin protein, and the withdrawal of the application will limit treatment options for these patients.
- Market Impact: This withdrawal may diminish PTC's competitiveness in the biopharmaceutical market, particularly in the rare disease treatment sector, potentially affecting investor confidence and exerting downward pressure on the company's stock price.
- Future Outlook: Despite the withdrawal, PTC remains committed to developing innovative therapies for rare diseases and may reassess its R&D strategy in response to FDA feedback to seek approval for alternative treatment options.
See More
