On CNBC's “Halftime Report Final Trades,” on Monday, Amy Raskin, chief investment officer at Chevy Chase Trust, named Ascendis Pharma A/S (NASDAQ:ASND) as her final trade.According to recent news, the U.S. Food & Drug Administration (FDA) on Nov. 25 extended the review period for Ascendis Pharma's New Drug Application (NDA) for TransCon CNP (navepegritide) for children with achondroplasia. The FDA said the information submitted on Nov. 5, related to the post-marketing requirement constituted a major amendme ...

Core Viewpoint - The FDA has extended the review period for Ascendis Pharma's New Drug Application for TransCon CNP, a treatment for children with achondroplasia, by three months to February 28, 2026, due to a major amendment related to post-marketing requirements [1][3]. Company Overview - Ascendis Pharma is focused on developing treatments for rare diseases, with TransCon CNP being a key product aimed at addressing achondroplasia, the most common form of skeletal dysplasia caused by a mutation in the FGFR3 gene [2][4]. - The company has reported positive results from its ApproaCH Trial, demonstrating significant improvements in annualized growth velocity and health-related quality of life for children treated with TransCon CNP compared to placebo [4][5]. Regulatory Update - The FDA's extension of the PDUFA target action date is a response to the major amendment submitted by Ascendis Pharma, which included a revised protocol for the post-marketing study [3]. - The company has addressed all outstanding requests from the FDA, indicating proactive engagement in the regulatory process [3]. Clinical Data - The pivotal week 52 results from the ApproaCH Trial were published in JAMA Pediatrics, highlighting the efficacy of TransCon CNP in improving growth metrics and overall health outcomes for children with achondroplasia [4][5]. - The treatment showed a safety and tolerability profile similar to that of placebo, reinforcing its potential as a viable therapeutic option [5]. Market Performance - Ascendis Pharma's stock price increased by 1.44%, reaching $213.03 at the time of publication, reflecting positive market sentiment regarding the company's developments [7].

Core Viewpoint - Ascendis Pharma A/S announced that the FDA has extended the PDUFA target action date for TransCon CNP by three months to February 28, 2026, due to a major amendment related to post-marketing requirements [1]. Group 1: FDA Interaction - The company has responded to all outstanding FDA requests, including a revised protocol for the post-marketing study, and is committed to working diligently with the FDA to finalize these elements [2]. - The extension of the PDUFA date indicates ongoing regulatory review and the need for additional information from the company [1]. Group 2: Company Overview - Ascendis Pharma is a global biopharmaceutical company focused on innovative therapies using its TransCon technology platform to address unmet medical needs [3]. - The company is headquartered in Copenhagen, Denmark, with additional facilities in Europe and the United States [3].

Core Insights - Ascendis Pharma reported Q3 2025 revenue of €213.6 million, a significant increase from €57.8 million in Q3 2024, driven primarily by the strong performance of YORVIPATH and SKYTROFA [6][12] - The company is progressing towards its Vision 2030 goals, with ongoing global launches and positive feedback from healthcare providers and patients [2][4] - Ascendis Pharma achieved an operating profit of €11.0 million in Q3 2025, attributed to higher revenues from YORVIPATH, despite increased operating expenses [11][10] Financial Performance - YORVIPATH revenue for Q3 2025 was €143.1 million, reflecting a negative foreign currency impact of €3.6 million compared to the previous quarter [5][6] - SKYTROFA revenue for Q3 2025 totaled €50.7 million, also impacted by a negative foreign currency effect of €1.6 million [5][6] - Total revenue from commercial products reached €193.8 million in Q3 2025, compared to €55.7 million in Q3 2024 [9][10] Product Development and Regulatory Updates - TransCon CNP is currently under FDA Priority Review for treating children with achondroplasia, with a PDUFA date set for November 30, 2025 [4][8] - The company plans to initiate a Phase 3 trial for TransCon CNP in combination with TransCon hGH following a successful end-of-Phase 2 meeting with the FDA [8][4] - Ascendis Pharma presented new analyses from the ApproaCH Trial at the ASBMR annual meeting, showing improvements in physical functioning for children treated with TransCon CNP [8] Operational Highlights - As of September 30, 2025, Ascendis Pharma had cash and cash equivalents of €539 million, a slight decrease from €560 million at the end of 2024 [13][20] - The company reported a net loss of €61.0 million for Q3 2025, an improvement from a net loss of €99.2 million in Q3 2024 [12][19] - Research and development costs decreased to €66.9 million in Q3 2025 from €73.5 million in the same period in 2024, reflecting the completion of clinical trials [10][19]

Core Insights - A significant rally occurred in the biotech and healthcare sectors on October 9, with multiple stocks reaching 52-week highs, driven by regulatory milestones, strategic announcements, and investor enthusiasm around clinical progress [1] Company Highlights - Aquestive Therapeutics Inc. (AQST) achieved a 52-week high of $6.86, closing at $6.69, following the announcement of a new U.S. patent for its PharmFilm, enhancing its position in oral drug delivery platforms [2] - Ascendis Pharma A/S (ASND) reached a 52-week high of $216.45, closing at $214.77, coinciding with the submission of a Marketing Authorisation Application to the European Medicines Agency for its investigational therapy targeting achondroplasia in children [3] - Cidara Therapeutics Inc. (CDTX) advanced 12.44% to close at $110.76, hitting a 52-week high of $111.35 after receiving Breakthrough Therapy designation from the FDA for its antiviral candidate CD388 [4] - Intellia Therapeutics Inc. (NTLA) surged 4.05% to end at $25.46, with a new 52-week high of $26.99, driven by optimism surrounding its in vivo CRISPR programs and recent trial updates [5] - Organigram Global Inc. (OGI) gained 4.12% to close at $2.02, reaching a 52-week high of $2.08, following the launch of its new hemp-derived delta-9 brand "happly" targeting mindful recreation consumers [6] - uniQure N.V. (QURE) rose 6.14% to finish at $63.65, with a 52-week high of $65.12, supported by analyst upgrades and renewed interest in its gene therapy candidate for Huntington's disease [7] - REGENXBIO Inc. (RGNX) closed slightly lower at $12.17 but reached a 52-week high of $12.84, presenting interim Phase II data for ABBV-RGX-314 at a major ophthalmology meeting [8] - Tempus AI Inc. (TEM) saw a decline of 3.85% to $99.28 after hitting a 52-week high of $104.32, despite being selected by ARPA-H for a federal precision cancer therapy initiative [9] - Tilray Brands, Inc. (TLRY) jumped 22.09% to close at $2.10, reaching a 52-week high of $2.32, reporting a return to profitability with record quarterly revenue of $210 million [9] - Zenas BioPharma (ZBIO) gained 7.18% to end at $27.76, hitting a 52-week high of $28.72 after announcing a licensing agreement and a $120 million private placement to support its autoimmune pipeline [10] - Brookdale Senior Living Inc. (BKD) rose 9.36% to close at $8.88, reaching a 52-week high of $9.09, reporting its highest occupancy rate in over a year at 82.5% [11]

Core Insights - Ascendis Pharma has submitted a Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) for TransCon CNP as a treatment for children with achondroplasia, a rare genetic condition impacting skeletal development and quality of life [1][2] - TransCon CNP is designed to provide continuous inhibition of the overactive FGFR3 pathway, administered once weekly to enhance the treatment's effectiveness [1][2] - The MAA is supported by data from three randomized clinical trials, including the pivotal ApproaCH Trial, demonstrating that TransCon CNP has been generally well tolerated [2] Company Overview - Ascendis Pharma is a global biopharmaceutical company focused on innovative therapies using its TransCon technology platform, aiming to address unmet medical needs [5] - The company is headquartered in Copenhagen, Denmark, with additional facilities in Europe and the United States [5] Industry Context - Achondroplasia affects over 250,000 individuals worldwide and is associated with various medical complications beyond skeletal dysplasia, including muscular, neurological, and cardiorespiratory issues [4] - The need for early pharmacological interventions in the achondroplasia community has been emphasized, highlighting the importance of treatments like TransCon CNP [3]

Core Insights - Ascendis Pharma A/S announced two oral presentations at ENDO 2025, showcasing data on the safety and efficacy of its treatments for hypoparathyroidism and achondroplasia [1][2] Group 1: Hypoparathyroidism - Dr. Aliya Khan will present data from Week 156 of the Phase 3 PaTHway Trial, highlighting the long-term safety and efficacy of TransCon PTH (palopegteriparatide) in adults with hypoparathyroidism [1][2] - The presentation will cover maintenance of serum and urine biochemistries within normal levels and sustained improvement in renal function [1] Group 2: Achondroplasia - Dr. Carlos Bacino will present safety and tolerability data from Week 52 of the pivotal ApproaCH Trial of once-weekly TransCon CNP (navepegritide) in children with achondroplasia [1][2] - The data is expected to support the transformative potential of the investigational therapy for this rare genetic condition [2] Group 3: Company Overview - Ascendis Pharma is focused on applying its innovative TransCon technology platform to develop therapies that address unmet medical needs [5] - The company is headquartered in Copenhagen, Denmark, with additional facilities in Europe and the United States [5]

Core Insights - Ascendis Pharma A/S will present new data on its hypoparathyroidism, achondroplasia, and growth hormone deficiency programs at the ESPE & ESE 2025 congress in Copenhagen from May 10-13, 2025 [1][2] Hypoparathyroidism - The company will showcase 4-year efficacy and safety data from the Phase 2 PaTH Forward Trial of TransCon PTH (palopegteriparatide) in adults with chronic hypoparathyroidism [2][4] - Presentations will include a retrospective matched cohort study using real-world data from England to estimate the risk of chronic kidney disease progression in chronic hypoparathyroidism [4][6] Achondroplasia - Ascendis will present Week 52 growth and bone morphometry data from the pivotal ApproaCH Trial of TransCon CNP (navepegritide) in children with achondroplasia [2][4] - The congress will feature discussions on assessing health-related quality of life (HRQoL) in achondroplasia across the life course [4][6] Growth Hormone Deficiency - The company will present results from the Phase 3 foresiGHt Trial supporting the efficacy and safety of once-weekly lonapegsomatropin in adults with growth hormone deficiency [2][6] - The presentations will highlight the importance of growth hormone in maintaining normal body composition and cardiometabolic health throughout adulthood [9] Company Overview - Ascendis Pharma is a global biopharmaceutical company focused on innovative therapies using its TransCon technology platform to address unmet medical needs [10] - The company is headquartered in Copenhagen, Denmark, with additional facilities in Europe and the United States [10]

Core Insights - Ascendis Pharma has submitted a New Drug Application (NDA) to the FDA for TransCon CNP, aimed at treating children with achondroplasia, supported by data from three clinical trials and up to three years of open-label extension data [1][2] - The company plans to submit a Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) in Q3 2025 [3] Group 1: Clinical Data and Benefits - Clinical data indicates that TransCon CNP not only increases growth velocity but also reduces health-related burdens, enhances muscle function, and corrects leg bowing in most treated children [3] - The treatment is administered once weekly and has a safety and tolerability profile comparable to placebo, positioning it as a potential best-in-class treatment for achondroplasia [3] Group 2: Achondroplasia Overview - Achondroplasia is a rare genetic condition affecting over 250,000 people globally, leading to various complications beyond skeletal dysplasia, including muscular, neurological, and cardiorespiratory issues [4] - Complications can include spinal deformities, impaired muscle strength, and chronic pain, significantly impacting quality of life and requiring multiple surgeries [4] Group 3: Company Background - Ascendis Pharma is a global biopharmaceutical company focused on innovative therapies using its TransCon technology platform to address unmet medical needs [5] - The company is headquartered in Copenhagen, Denmark, with additional facilities in Europe and the United States [5]