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17.11.25 - 22:03
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Results of Pivotal ApproaCH Trial of TransCon® CNP (Navepegritide) in Children with Achondroplasia Published in JAMA Pediatrics (GlobeNewswire EN)
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COPENHAGEN, Denmark, Nov. 17, 2025 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced that pivotal Week 52 results from its randomized double-blind, placebo-controlled ApproaCH Trial of investigational once-weekly TransCon® CNP (navepegritide) in children with achondroplasia have been published in JAMA Pediatrics, a journal of the American Medical Association. In the publication, titled “Once-Weekly Navepegritide in Children with Achondroplasia: The ApproaCH Randomized Clinical Trial,” the authors report that treatment with TransCon CNP led to significantly higher annualized growth velocity (AGV) at Week 52 compared to placebo (primary endpoint), as well as improved lower-limb alignment and body proportionality and positive changes in health-related quality of life, with a safety and tolerability profile similar to placebo. The abstract can be accessed on the JAMA Pediatrics website....
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08.10.25 - 22:03
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Ascendis Submits Marketing Authorisation Application to the European Medicines Agency for TransCon® CNP for Treatment of Children with Achondroplasia (GlobeNewswire EN)
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COPENHAGEN, Denmark, Oct. 08, 2025 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced it has submitted a Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) for TransCon CNP (navepegritide) as a treatment for children with achondroplasia, a rare genetic condition that causes skeletal dysplasia and, for many affected individuals, significant health, physical functioning, and quality of life impacts. TransCon CNP is an investigational prodrug of C-type natriuretic peptide (CNP) administered once weekly, designed for continuous inhibition of the overactive FGFR3 pathway in achondroplasia by providing continuous exposure of active CNP to receptors on tissues throughout the body, including growth plates and skeletal muscle....
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02.09.25 - 22:03
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Ascendis to Share Latest Endocrinology Rare Disease Data in 3 Oral Presentations at ASBMR 2025 (GlobeNewswire EN)
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COPENHAGEN, Denmark, Sept. 02, 2025 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced three oral presentations of its latest achondroplasia and hypoparathyroidism data at ASBMR 2025, the annual meeting of the American Society for Bone & Mineral Research being held in Seattle from September 5-8, 2025. These include a new analysis of pivotal trial data correlating improvements in leg bowing with improved physical functioning in children with achondroplasia treated with TransCon® CNP (navepegritide); the first medical congress presentation of clinical trial data for children with achondroplasia treated with TransCon CNP and TransCon hGH (lonapegsomatropin) combination therapy; and clinical trial data demonstrating the sustained improvements in skeletal dynamics in adults with hypoparathyroidism treated for 4 years with TransCon PTH (palopegteriparatide)....
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