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Results of Pivotal ApproaCH Trial of TransCon® CNP (Navepegritide) in Children with Achondroplasia Published in JAMA Pediatrics
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 Clinic
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[{"type":"image","alt":"Ascendis Pharma","displaySize":"","headline":null,"caption":"Ascendis Pharma","className":"","disableSlideshowImg":false,"size":{"original":{"width":300,"height":100,"url":"https://media.zenfs.com/en/globenewswire.com/7afd0e29f0a4b40bcd5ff718a81f4afe"},"resized":{"url":"https://s.yimg.com/ny/api/res/1.2/1tA.9j4XDPXN.zyLsOvv6w--/YXBwaWQ9aGlnaGxhbmRlcjt3PTQyMDtoPTE0MDtjZj13ZWJw/https://media.zenfs.com/en/globenewswire.com/7afd0e29f0a4b40bcd5ff718a81f4afe","width":300,"height":100}},"lazy":false},{"type":"text","content":"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.","length":916,"tagName":"p"},{"type":"text","content":"“Children randomized to navepegritide had significantly better growth and improvements in important health outcomes compared with placebo,” said Ravi Savarirayan, MBBS, M.D., Group Leader of Molecular Therapies at Murdoch Children’s Research Institute in Melbourne, Australia. “These findings show that navepegritide is a promising potential new treatment option to reduce the medical burden of this condition, with once-weekly dosing and a low rate of injection site reactions.”","length":479,"tagName":"p"},{"type":"text","content":"“To help guide their healthcare decision, families want information beyond changes in height to understand how an intervention may affect the potential medical challenges of achondroplasia,” said Michael Hughes, Chair of the Biotech Industry Liaison Committee at Little People of America. “Including these endpoints in blinded, controlled st...