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 Insights - Ascendis Pharma announced a pooled analysis demonstrating sustained improvements in renal function in adults with hypoparathyroidism treated with TransCon PTH through Year 3 of the Phase 2 PaTH Forward and Phase 3 PaTHway trials [1][2] Group 1: Clinical Trial Results - The pooled analysis included 141 adults with hypoparathyroidism, with 139 patients (mean age 49 years) represented in the analysis [2] - At Year 3, 91% of patients receiving TransCon PTH were independent from conventional therapy, and 84% had normocalcemia [3] - Sustained improvements in eGFR (≥ 5 mL/min/1.73 m²) were observed in 70.3% of patients, with the most significant increases occurring in the first 6 months of treatment [3] Group 2: Safety and Tolerability - TransCon PTH was generally well tolerated, with no new safety signals identified; most treatment-emergent adverse events were mild to moderate [4] - Reductions in 24-hour urinary calcium excretion were maintained within the normal range through Year 3 [4] Group 3: Company Overview and Future Directions - Ascendis Pharma focuses on applying its TransCon technology platform to develop therapies that address unmet medical needs, with TransCon PTH approved as YORVIPATH® for adults with hypoparathyroidism [5][7] - The company aims to continue discussions on the significance of PTH replacement for overall kidney health in patients with hypoparathyroidism [5]

Core Insights - Ascendis Pharma announced positive data on TransCon PTH, showing sustained improvements in renal function and safety in adults with hypoparathyroidism through Year 3 of the Phase 2 PaTH Forward and Phase 3 PaTHway trials [1][2] - The data will be presented at Kidney Week 2025, aiming to enhance awareness of TransCon PTH and its role in kidney health [2] Company Overview - Ascendis Pharma is a global biopharmaceutical company focused on innovative therapies using its TransCon technology platform, headquartered in Copenhagen, Denmark [4] - The company aims to address unmet medical needs and improve patient outcomes through its product innovations [4] Product Information - TransCon PTH is a prodrug of parathyroid hormone (PTH), designed for once-daily administration to maintain physiological PTH levels for 24 hours [2] - It is approved as YORVIPATH® in the U.S., EU, and EEA for treating adults with hypoparathyroidism [2] Disease Context - Hypoparathyroidism is characterized by insufficient PTH levels, leading to severe complications such as renal issues and neuromuscular irritability [3] - The majority of cases (70-80%) are post-surgical, with other causes being autoimmune and idiopathic [3]

Core Insights - Ascendis Pharma A/S announced three oral presentations of data related to achondroplasia and hypoparathyroidism at ASBMR 2025, highlighting the effectiveness of their therapies [1][2] Group 1: Achondroplasia - Achondroplasia is a rare genetic condition affecting over 250,000 people globally, leading to various complications beyond skeletal dysplasia, including muscular and neurological issues [3] - The company presented new analyses correlating improvements in leg bowing with enhanced physical functioning in children treated with TransCon® CNP [1] - The first medical congress presentation of clinical trial data for children treated with TransCon CNP and TransCon hGH combination therapy was also shared [1] Group 2: Hypoparathyroidism - Hypoparathyroidism is characterized by insufficient parathyroid hormone levels, leading to severe complications, with post-surgical cases accounting for 70-80% of instances [4] - Clinical trial data demonstrated sustained improvements in skeletal dynamics in adults treated for four years with TransCon PTH [1][2] Group 3: Company Overview - Ascendis Pharma is a global biopharmaceutical company focused on innovative therapies using its TransCon technology platform to address unmet medical needs [5] - The company emphasizes its commitment to patient care, scientific innovation, and passion in its operations [5]

Core Insights - Ascendis Pharma announced positive long-term data from the Phase 3 PaTHway Trial for TransCon PTH, showing sustained efficacy in treating adults with hypoparathyroidism [1][4] - The trial demonstrated significant improvements in biochemical markers, kidney function, and quality of life for patients [1][3] Group 1: Trial Details - The PaTHway Trial was a Phase 3, randomized, double-blind, placebo-controlled study involving 82 adults with chronic hypoparathyroidism, followed by an open-label extension [2] - At Week 156, 88% of patients had normal serum calcium levels, and 96% were independent from conventional therapy [3] - The trial maintained high retention rates, with 89% of participants completing the 3.5-year study [2] Group 2: Efficacy and Safety - Mean estimated glomerular filtration rate (eGFR) increased by 8.76 mL/min/1.73 m² across all participants, with a notable increase of 13.98 mL/min/1.73 m² in those with baseline eGFR < 60 [3] - Patients reported continued improvements in hypoparathyroidism-related symptoms and health-related quality of life, with normalization of 24-hour urine calcium excretion [3] - TransCon PTH treatment was well-tolerated, with no new safety signals identified and most treatment-emergent adverse events being mild or moderate [3] Group 3: Company Overview - Ascendis Pharma is a global biopharmaceutical company focused on innovative therapies using its TransCon technology platform [6] - The company aims to address unmet medical needs and improve patient outcomes through its product innovations [6]

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]