FDA Approves Ascendis Pharma's Yuviwel for Dwarfism Treatment
Written by Emily J. Thompson, Senior Investment Analyst
Updated: 1 hour ago
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Should l Buy ASND?
Source: seekingalpha
- Drug Approval: The US FDA has granted approval for Ascendis Pharma's Yuviwel (navepegritide) to treat children with achondroplasia, marking a significant advancement for the company in the pediatric growth disorder treatment space.
- Dosing Regimen: Yuviwel is administered once weekly for children aged two years and older, with availability expected in Q2 2025, addressing the urgent market demand for effective treatment options.
- Post-Market Trial Requirement: The FDA mandates Ascendis to conduct post-marketing trials to confirm the treatment's benefits, which will influence the company's future clinical research and market strategy.
- Improvement in Growth Velocity: The approval was based on significant improvements in annualized growth velocity, demonstrating the drug's effectiveness in enhancing children's growth potential, which could drive Ascendis's further development in the biopharmaceutical market.
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Analyst Views on ASND
Wall Street analysts forecast ASND stock price to rise
13 Analyst Rating
13 Buy
0 Hold
0 Sell
Strong Buy
Current: 228.990
Low
240.00
Averages
277.08
High
342.00
Current: 228.990
Low
240.00
Averages
277.08
High
342.00
About ASND
Ascendis Pharma A/S is a Denmark-based company that manufactures pharmaceutical products. The Company produces products for the treatments of growth hormone deficiency, endocrinology, central nervous system disorders, infectious diseases, and diabetes. The Company operates globally though its subsidiaries: Ascendis Pharma GmbH (Germany), Ascendis Pharma, Inc. (Delaware, United States), Ascendis Pharma Endocrinology, Inc. (Delaware, United States), Ascendis Pharma, Ophthalmology Division A/S (Denmark), Ascendis Pharma, Endocrinology Division A/S (Denmark), Ascendis Pharma Bone Diseases A/S (Denmark), Ascendis Pharma Growth Disorders A/S (Denmark) and Ascendis Pharma Oncology Division A/S (Denmark).
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.
FDA Approves First Treatment for Achondroplasia
- FDA Approval: The U.S. FDA has granted approval for YUVIWEL (navepegritide), the first once-weekly treatment for achondroplasia aimed at increasing linear growth in children aged 2 and older, marking a significant advancement in treatment options.
- Clinical Trial Support: The approval is based on data from three randomized, double-blind, placebo-controlled clinical trials demonstrating significant improvements in annualized growth velocity, which is expected to provide new therapeutic options for patients.
- Patient Services Program: Ascendis plans to offer a suite of patient services for YUVIWEL through its U.S. Ascendis Signature Access Program (A.S.A.P.), including support for navigating the treatment journey and financial assistance, aimed at enhancing the patient experience.
- Priority Review Voucher: The FDA also granted YUVIWEL a Rare Pediatric Disease Priority Review Voucher, designed to encourage the development of new drugs for rare pediatric diseases, further enhancing the market potential of this treatment.
See More
FDA Approves Ascendis Pharma's Yuviwel for Dwarfism Treatment
- Drug Approval: The US FDA has granted approval for Ascendis Pharma's Yuviwel (navepegritide) to treat children with achondroplasia, marking a significant advancement for the company in the pediatric growth disorder treatment space.
- Dosing Regimen: Yuviwel is administered once weekly for children aged two years and older, with availability expected in Q2 2025, addressing the urgent market demand for effective treatment options.
- Post-Market Trial Requirement: The FDA mandates Ascendis to conduct post-marketing trials to confirm the treatment's benefits, which will influence the company's future clinical research and market strategy.
- Improvement in Growth Velocity: The approval was based on significant improvements in annualized growth velocity, demonstrating the drug's effectiveness in enhancing children's growth potential, which could drive Ascendis's further development in the biopharmaceutical market.
See More
Ascendis Pharma Receives FDA Approval for YUVIWEL, a Breakthrough Treatment for Achondroplasia
- FDA Approval Milestone: Ascendis Pharma's YUVIWEL (navepegritide) has received FDA accelerated approval as the first once-weekly treatment for increasing linear growth in children aged 2 and older with achondroplasia, with commercial availability expected in early Q2 2026, marking a significant advancement in treatment options.
- Clinical Trial Validation: The approval is based on data from three randomized, double-blind, placebo-controlled trials demonstrating significant improvements in annualized growth velocity, with continued approval contingent upon verification of clinical benefits in confirmatory trials, highlighting the company's commitment to robust scientific evidence.
- Patient Support Initiatives: Ascendis plans to offer a suite of patient services through its U.S. Ascendis Signature Access Program (A.S.A.P.), including support for navigating the treatment journey and financial assistance, aimed at enhancing patient experience and accessibility, thereby strengthening market competitiveness.
- Rare Disease Priority Review: The FDA also granted YUVIWEL a Rare Pediatric Disease Priority Review Voucher, designed to encourage the development of new drugs for rare diseases, further enhancing Ascendis's strategic position in the biopharmaceutical sector.
See More
Perceptive Advisors Acquires 431,432 Shares of Praxis Precision Medicines
- Share Acquisition Details: Perceptive Advisors increased its stake in Praxis Precision Medicines by 431,432 shares during Q4 2025, with an estimated transaction value of $80.34 million, reflecting strong confidence in the company's future prospects.
- Position Value Growth: Following this acquisition, the value of Praxis's holdings reached $588.30 million, an increase of $505.38 million from the previous quarter-end, indicating significant stock price appreciation and positive investor sentiment.
- Financial Overview: As of February 17, 2026, Praxis shares were priced at $328.04, up 320% year-over-year, despite a net loss of $273.04 million in 2025, suggesting market optimism regarding its future product pipeline.
- R&D and Market Outlook: Praxis has submitted two NDAs to the FDA and is actively preparing for commercialization, with multiple clinical trial results expected in 2026, which could further enhance its market position in the central nervous system sector.
See More
Perceptive Advisors Increases Stake in Celcuity
- Stake Increase: Perceptive Advisors purchased an additional 203,881 shares of Celcuity in Q4 2026, with an estimated transaction value of $16.76 million, indicating strong confidence in the company's future growth potential.
- Ownership Proportion Rise: Following this purchase, Perceptive Advisors' stake in Celcuity increased to 5.62%, highlighting its significance within the investment portfolio and further drawing market attention to Celcuity.
- Market Performance: As of February 17, 2026, Celcuity shares were priced at $107.32, reflecting a staggering 700% increase over the past year, underscoring strong market anticipation for its drug development.
- FDA Review Progress: Celcuity's new drug application for gedatolisib has received Priority Review from the FDA, with a decision expected by July 17, 2026, a critical timeline that could significantly impact its future market performance.
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BridgeBio Pharma Stock Soars on Positive Clinical Trial Results
- Clinical Trial Results: BridgeBio Pharma's PROPEL 3 Phase 3 trial for oral infigratinib in children demonstrated a significant annualized height velocity increase, with an LS mean treatment difference of +1.74 cm/year compared to placebo, highlighting the drug's potential in treating achondroplasia.
- Statistical Significance: In a pre-specified exploratory analysis of children under 8, infigratinib showed statistical significance against placebo with an LS mean decrease of -0.05, marking a significant breakthrough in randomized trials for achondroplasia treatment.
- Regulatory Plans: BridgeBio intends to meet with regulatory authorities in the second half of 2026 to discuss plans for submitting a New Drug Application (NDA) and Marketing Authorization Application (MAA) for infigratinib, reflecting the company's confidence in future market opportunities.
- Competitive Landscape: While BioMarin's Voxzogo is the first FDA-approved drug for achondroplasia, the positive results from BridgeBio's infigratinib clinical trials may shift the competitive dynamics and enhance the company's position in the rare disease market.
See More
FDA Approves First Treatment for Achondroplasia
- FDA Approval: The U.S. FDA has granted approval for YUVIWEL (navepegritide), the first once-weekly treatment for achondroplasia aimed at increasing linear growth in children aged 2 and older, marking a significant advancement in treatment options.
- Clinical Trial Support: The approval is based on data from three randomized, double-blind, placebo-controlled clinical trials demonstrating significant improvements in annualized growth velocity, which is expected to provide new therapeutic options for patients.
- Patient Services Program: Ascendis plans to offer a suite of patient services for YUVIWEL through its U.S. Ascendis Signature Access Program (A.S.A.P.), including support for navigating the treatment journey and financial assistance, aimed at enhancing the patient experience.
- Priority Review Voucher: The FDA also granted YUVIWEL a Rare Pediatric Disease Priority Review Voucher, designed to encourage the development of new drugs for rare pediatric diseases, further enhancing the market potential of this treatment.
See More
FDA Approves Ascendis Pharma's Yuviwel for Dwarfism Treatment
- Drug Approval: The US FDA has granted approval for Ascendis Pharma's Yuviwel (navepegritide) to treat children with achondroplasia, marking a significant advancement for the company in the pediatric growth disorder treatment space.
- Dosing Regimen: Yuviwel is administered once weekly for children aged two years and older, with availability expected in Q2 2025, addressing the urgent market demand for effective treatment options.
- Post-Market Trial Requirement: The FDA mandates Ascendis to conduct post-marketing trials to confirm the treatment's benefits, which will influence the company's future clinical research and market strategy.
- Improvement in Growth Velocity: The approval was based on significant improvements in annualized growth velocity, demonstrating the drug's effectiveness in enhancing children's growth potential, which could drive Ascendis's further development in the biopharmaceutical market.
See More
Ascendis Pharma Receives FDA Approval for YUVIWEL, a Breakthrough Treatment for Achondroplasia
- FDA Approval Milestone: Ascendis Pharma's YUVIWEL (navepegritide) has received FDA accelerated approval as the first once-weekly treatment for increasing linear growth in children aged 2 and older with achondroplasia, with commercial availability expected in early Q2 2026, marking a significant advancement in treatment options.
- Clinical Trial Validation: The approval is based on data from three randomized, double-blind, placebo-controlled trials demonstrating significant improvements in annualized growth velocity, with continued approval contingent upon verification of clinical benefits in confirmatory trials, highlighting the company's commitment to robust scientific evidence.
- Patient Support Initiatives: Ascendis plans to offer a suite of patient services through its U.S. Ascendis Signature Access Program (A.S.A.P.), including support for navigating the treatment journey and financial assistance, aimed at enhancing patient experience and accessibility, thereby strengthening market competitiveness.
- Rare Disease Priority Review: The FDA also granted YUVIWEL a Rare Pediatric Disease Priority Review Voucher, designed to encourage the development of new drugs for rare diseases, further enhancing Ascendis's strategic position in the biopharmaceutical sector.
See More
Perceptive Advisors Acquires 431,432 Shares of Praxis Precision Medicines
- Share Acquisition Details: Perceptive Advisors increased its stake in Praxis Precision Medicines by 431,432 shares during Q4 2025, with an estimated transaction value of $80.34 million, reflecting strong confidence in the company's future prospects.
- Position Value Growth: Following this acquisition, the value of Praxis's holdings reached $588.30 million, an increase of $505.38 million from the previous quarter-end, indicating significant stock price appreciation and positive investor sentiment.
- Financial Overview: As of February 17, 2026, Praxis shares were priced at $328.04, up 320% year-over-year, despite a net loss of $273.04 million in 2025, suggesting market optimism regarding its future product pipeline.
- R&D and Market Outlook: Praxis has submitted two NDAs to the FDA and is actively preparing for commercialization, with multiple clinical trial results expected in 2026, which could further enhance its market position in the central nervous system sector.
See More
Perceptive Advisors Increases Stake in Celcuity
- Stake Increase: Perceptive Advisors purchased an additional 203,881 shares of Celcuity in Q4 2026, with an estimated transaction value of $16.76 million, indicating strong confidence in the company's future growth potential.
- Ownership Proportion Rise: Following this purchase, Perceptive Advisors' stake in Celcuity increased to 5.62%, highlighting its significance within the investment portfolio and further drawing market attention to Celcuity.
- Market Performance: As of February 17, 2026, Celcuity shares were priced at $107.32, reflecting a staggering 700% increase over the past year, underscoring strong market anticipation for its drug development.
- FDA Review Progress: Celcuity's new drug application for gedatolisib has received Priority Review from the FDA, with a decision expected by July 17, 2026, a critical timeline that could significantly impact its future market performance.
See More
BridgeBio Pharma Stock Soars on Positive Clinical Trial Results
- Clinical Trial Results: BridgeBio Pharma's PROPEL 3 Phase 3 trial for oral infigratinib in children demonstrated a significant annualized height velocity increase, with an LS mean treatment difference of +1.74 cm/year compared to placebo, highlighting the drug's potential in treating achondroplasia.
- Statistical Significance: In a pre-specified exploratory analysis of children under 8, infigratinib showed statistical significance against placebo with an LS mean decrease of -0.05, marking a significant breakthrough in randomized trials for achondroplasia treatment.
- Regulatory Plans: BridgeBio intends to meet with regulatory authorities in the second half of 2026 to discuss plans for submitting a New Drug Application (NDA) and Marketing Authorization Application (MAA) for infigratinib, reflecting the company's confidence in future market opportunities.
- Competitive Landscape: While BioMarin's Voxzogo is the first FDA-approved drug for achondroplasia, the positive results from BridgeBio's infigratinib clinical trials may shift the competitive dynamics and enhance the company's position in the rare disease market.
See More
