Skip to main content Exclusive news, data and analytics for financial market professionalsLearn more aboutRefinitiv Ascendis wins U.S. approval for therapy for children with dwarfism February 27, 202611:04 PM UTCUpdated ago By Reuters Read Next Business Delta orders 34 more Airbus A321neo jets in fleet renewal push February 27, 2026 · 9:43 PM UTCAerospace & Defensecategory · · ago Companies Ascendis Pharma A/S Biomarin Pharmaceutical Inc BridgeBio Pharma Inc Feb 27 (Reuters) - The U.S. Food and Drug Adminis ...

侏儒症的治疗，正迎来被全面颠覆的时刻。 作为最常见的侏儒症类型，软骨发育不全（ACH）过去很长时间无特效疗法，直到2021年，"孤儿药之王"BioMarin研 发的Vosoritide，作为首个获批药物，才开启了药物对症治疗时代。凭借独家优势，其2025年销售额高达9.27亿美元。 但这种垄断格局，即将被打破，多款在研新药正从作用机制到给药方式，向Vosoritide发起挑战。Ascendis的TransCon- CNP通过包裹技术延长了药物作用时间，实现了周剂给药，目前正处于FDA审批阶段；而BridgeBio的口服药 Infigratinib，则从作用机制到用药体验实现全面突破。 Infigratinib靶向的FGFR3（成纤维细胞生长因子受体3）正是驱动ACH疾病发生的关键靶点，2月12日，BridgeBio公布 Infigratinib在ACH中取得的首个具有统计学显著改善意义的3期顶线结果，公司计划下半年向FDA提交新药申请。 获批后的长期扩展临床数据显示，Vosoritide的生长促进效应可持续至少7年。 尽管目前全球仅有5000名婴幼儿使用该药，但罕见病药物的高定价模式，叠加长期持续给药的治疗需 ...

Ascendis Pharma (NASDAQ:ASND) is one of the best NASDAQ growth stocks to buy for the next 2 years. On February 11, Ascendis Pharma reported that it nearly doubled its total revenue for 2025 to €720 million, fueled by the rapid global adoption of its core endocrinology products. Q4 was particularly significant, as the company achieved an operating profit of €10 million and generated €73 million in operating cash flow. This was largely driven by YORVIPATH, which brought in €477 million for the full year, and ...

Core Insights - Ascendis Pharma is experiencing strong commercial growth for its products, particularly YORVIPATH and SKYTROFA, with significant revenue increases reported for Q4 2025 and full-year 2025 [3][4][7]. Commercial Performance - YORVIPATH generated €187 million in Q4 2025, contributing to a total of €477 million for the full year [3][7]. - SKYTROFA reported Q4 revenue of €53 million and full-year revenue of €206 million, with a U.S. market share of approximately 7% [11]. - The company has prescribed YORVIPATH to over 5,300 patients in the U.S. through nearly 2,400 healthcare providers, indicating a substantial long-term opportunity as less than 5% of U.S. patients are currently on treatment [2][7]. Financial Outlook - Ascendis ended 2025 with €616 million in cash and expects to achieve around €500 million in operating cash flow in 2026, with a long-term goal of at least €5 billion in annual product revenue by 2030 [5][20]. - Total revenue for Q4 2025 was €248 million, with operating expenses of €214 million, resulting in an operating profit of €10 million [17]. Pipeline and Regulatory Updates - The U.S. NDA for TransCon CNP is under review with a PDUFA date set for February 28, 2026, and the company anticipates full enrollment in its trial for infants with achondroplasia by late 2026 [6][13]. - Management highlighted the potential of a once-weekly TransCon PTH candidate, which aims to match the efficacy of daily therapies while improving patient convenience [8][12]. Market Expansion and Strategy - Ascendis plans to launch YORVIPATH in 10 additional countries in 2026, building on its existing commercial presence in over 30 countries [1]. - The company is also working to expand the label for SKYTROFA and is conducting trials to evaluate its growth hormone in additional indications, which could represent a significant market opportunity [10][11].

Core Insights - Ascendis Pharma A/S (NASDAQ:ASND) is recognized as a high-growth European stock, with RBC Capital raising its price target to $250 from $245 while maintaining an Outperform rating [1] Group 1: Market Potential - The European market for Ascendis Pharma A/S is projected to be significantly larger, with an estimated 150,000-200,000 patients compared to 70,000-90,000 in the U.S., although the complex payer system may require patience [2] - The company anticipates a revenue split of 60% from the U.S. and 40% from non-U.S. markets [2] Group 2: Financial Projections - Ascendis Pharma A/S aims for €500 million in operating cash flow for the current year and over €5 billion in top-line revenue by 2030, exceeding the consensus estimate of approximately €3.5 billion [3] - Yorvipath is expected to generate between €5 billion and €8 billion at its peak [3] Group 3: Company Overview - Ascendis Pharma A/S is a biopharmaceutical company focused on developing and distributing innovative treatments for unmet medical needs, particularly in oncology and endocrinology [3]

Core Viewpoint - Ascendis Pharma A/S (NASDAQ:ASND) is recognized as one of the best stocks to buy for investment, with a Buy rating reiterated by BofA and a price target set at $246 [1]. Group 1: Analyst Ratings and Price Targets - BofA has maintained a Buy rating on Ascendis Pharma, with a price target of $246 [1]. - Wells Fargo analyst Derek Archila upgraded the price target for Ascendis Pharma from €322 to €330, while keeping an Overweight rating on the stock [2]. - Archila highlighted Ascendis Pharma's operating cash flow guidance of €500 million, excluding TransCon CNP, which suggests a revenue range of €1.1-€1.2 billion for Yorvipath [2]. Group 2: Product Development and Market Potential - The TransCon CNP combined with Skytrofa is expected to potentially become the standard of care over time, indicating significant upside for Ascendis Pharma [3]. - Skytrofa is currently used for treating pediatric growth hormone deficiency, while TransCon is being developed for achondroplasia [3]. - Ascendis Pharma focuses on developing TransCon-based therapies to meet unmet medical needs globally [3].

Core Insights - Ascendis Pharma announced positive topline results from the Week 52 of the COACH trial, demonstrating that the combination therapy of once-weekly TransCon CNP and TransCon hGH showed durable growth without compromising safety or tolerability [2][3] Group 1: Trial Results - The combination therapy resulted in an annualized growth velocity (AGV) that exceeded the 97th percentile of average stature children [11] - For the treatment-naïve cohort, the mean AGV was 8.80 cm/year, with a mean ACH height Z-score improvement of +1.02, indicating a tripling of efficacy compared to TransCon CNP monotherapy [5][6] - The treated cohort showed a mean AGV of 8.42 cm/year, with an increase from baseline of 3.28 cm/year and a mean ACH height Z-score improvement of +0.86, increasing from 1.28 to 2.15 over 52 weeks [5][6] Group 2: Safety and Tolerability - The combination therapy was generally well tolerated, with mild treatment-emergent adverse events (TEAEs) consistent with those observed for the monotherapies [2][11] - Safety and tolerability profiles were consistent with TransCon CNP and TransCon hGH monotherapies, indicating a low incidence of injection site reactions [11] Group 3: Implications for Treatment - The results suggest that TransCon CNP may become a foundational therapy for achondroplasia, with TransCon hGH providing complementary benefits [2][3] - The trial highlights the potential for dual-agent regimens to set new standards of care in treating achondroplasia and other growth disorders [3][11] - Improvements in body proportionality and arm span were observed, aligning with the increase in linear growth, indicating benefits beyond just height [2][11] Group 4: Future Developments - Ascendis Pharma has submitted a protocol for a Phase 3 trial of TransCon CNP and TransCon hGH in pediatric achondroplasia and held an end of Phase 2 meeting with the FDA [7][11] - The U.S. FDA has granted Priority Review for TransCon CNP, with a PDUFA target action date of February 28, 2026 [3][11]

Summary of BridgeBio Pharma FY Conference Call Company Overview - **Company**: BridgeBio Pharma (NasdaqGS:BBIO) - **Key Product**: Attruby, a treatment for ATTR-CM (transthyretin amyloid cardiomyopathy) - **Market Context**: The company has seen a stock price increase of approximately 160% year-to-date, outperforming the XBI index which increased by 30% [1] Core Insights and Arguments Attruby Launch and Market Share - **Launch Success**: The launch of Attruby is described as going extremely well, with the company achieving a market share in the first-line treatment setting now well into the 20% range, with a long-term goal of 30-40% [2][3] - **Clinical Data**: Attruby is noted for having near-complete clinical data on its label, including a 50% reduction in hospitalization at 30 months and a 42% reduction in all-cause mortality and hospitalization [3] - **Market Access Initiatives**: The company has implemented a 28-day free trial program and offers extensive support through patient access liaisons and reimbursement managers, facilitating easier access to Attruby [4] Q4 Expectations and Market Dynamics - **Q4 Performance**: The company anticipates a strong performance in Q4, despite having three fewer selling weeks compared to other quarters. The discontinuation of Pfizer's Vyndaqel is expected to be more beneficial in 2026 rather than Q4 [6][8] - **Switching Patients**: The company aims to convert patients from Vyndaqel to Attruby, particularly in accounts where Vyndaqel was previously used [9][10] Competitive Landscape - **Competitor Actions**: Pfizer's introduction of a similar free trial program for Vyndamax is acknowledged, but the company believes that clinical differentiation will maintain its competitive edge [11][12] - **Diagnosis Rates**: The diagnosis of ATTR-CM is still considered underdiagnosed, with significant room for growth. The number of PYP scans has grown at a CAGR of about 30% since 2019 [13][14] Future Market Opportunities - **Generic Competition**: The potential entry of generics for Vyndaqel in 2028 is discussed, but the company emphasizes that Attruby's clinical differentiation will sustain its market position [16][18] - **Achondroplasia Market**: The company is preparing for the launch of infigratinib for achondroplasia, positioning it as a potential first oral treatment targeting the FGFR3 mutation [19][21] Pricing Strategy - **Pricing Considerations**: The company plans to price new products in accordance with their opportunity size, with potential pricing for rare diseases ranging from $400,000 to $900,000 [38][39] Additional Important Points - **Regulatory Filings**: The company expects to file NDAs for Encaleret and BBP-418 in the first half of next year, with launches anticipated in Q4 2026 and Q1 2027 [36] - **Safety and Efficacy**: The company aims to maintain low rates of hyperphosphatemia in patients treated with infigratinib, with a target of no more than 20% experiencing low-grade hyperphosphatemia [28] This summary encapsulates the key points discussed during the conference call, highlighting BridgeBio Pharma's strategic positioning, market dynamics, and future outlook.

Core Insights - Ascendis Pharma announced pivotal Week 52 results from the ApproaCH Trial of TransCon® CNP (navepegritide) in children with achondroplasia, showing significant improvements in annualized growth velocity compared to placebo [1][4][5] - The trial demonstrated favorable impacts on body proportionality and health-related quality of life, with a safety profile similar to placebo [1][5][6] Group 1: Trial Results - The ApproaCH Trial was a randomized, double-blind, placebo-controlled study involving 84 children aged 2-11, with a 2:1 randomization to receive either TransCon CNP at 100 μg/kg/week or placebo over 52 weeks [3] - Treatment with TransCon CNP resulted in a significant increase in annualized growth velocity (AGV) and improvements in lower-limb alignment and body proportionality compared to placebo [1][4] - The trial also reported improvements in health-related quality of life across several domains, without accelerating bone age or negatively affecting spinal curvature [5] Group 2: Treatment Profile - TransCon CNP is an investigational prodrug designed for continuous inhibition of the overactive FGFR3 pathway in achondroplasia, administered once weekly [2][6] - The treatment showed a low rate of injection site reactions and no observed symptomatic hypotension or bone fractures during the trial [5] - The FDA has placed TransCon CNP under Priority Review, with a target date of November 30, 2025, and it is also under review by the European Medicines Agency [6] Group 3: Condition Overview - Achondroplasia is a rare genetic condition affecting over 250,000 individuals globally, leading to various medical complications beyond skeletal dysplasia [7][8] - Complications include spinal abnormalities, impaired muscle strength, and chronic pain, which can significantly impact quality of life and require multiple surgeries [8]

Core Insights - Medtronic (MDT) is scheduled to announce its second-quarter fiscal 2026 results on November 18, with expectations of strong performance based on previous earnings surprises [1][2] Group 1: Financial Performance Estimates - The Zacks Consensus Estimate for Medtronic's revenues in the fiscal second quarter is $8.86 billion, indicating a 5.4% year-over-year increase [2] - The consensus estimate for EPS is projected to rise 4% to $1.31, with earnings estimates remaining unchanged over the past 60 days [2] Group 2: Segment Performance Cardiovascular - The Cardiovascular segment is expected to show strong momentum, with revenues projected to increase by 8.4% year over year, driven by the uptake of PulseSelect and Affera Sphere-9 systems [3][5] - The Evolut FX+ TAVR device is likely to gain traction in various markets, including Japan, benefiting from a competitor's exit [4] Neuroscience - Neuroscience revenues are anticipated to grow by 1.5% year over year, supported by strong sales in Cranial & Spinal Technologies and the adoption of AiBLE spine technology [6][8] - Neuromodulation performance is expected to benefit from U.S. growth in Pain Stim and the approval of the Altaviva device for treating urge urinary incontinence [7] Medical Surgical - Medtronic's Medical Surgical segment is projected to see a 5.4% year-over-year revenue increase, driven by demand for LigaSure technology and growth in the Advanced Energy product line [10][11] Diabetes - The Diabetes unit is expected to experience a 9.2% year-over-year revenue growth, fueled by the MiniMed 780G and Simplera Sync sensors [9][14] - Key FDA approvals during the quarter, including the SmartGuard algorithm and MiniMed 780G for type 2 diabetes, are likely to contribute to revenue growth [13][14] Group 3: Earnings Expectations - Medtronic currently holds a Zacks Rank of 3 (Hold) with an Earnings ESP of 0.00%, indicating a neutral outlook for beating estimates [15]