Ascendis’ Yuviwel (Navepegritide) Receives the US FDA Accelerated Approval for Achondroplasia

Shots: FDA granted accelerated approval to Yuviwel (navepegritide, QW) to increase linear growth in children (≥2yrs.) with achondroplasia & open epiphyses; commercially available by Q2’26 Approval was based on extensive clinical data, incl. 3 PBO-controlled trials plus up to 3yrs. of OLE data, with the pivotal ApproaCH results published in JAMA Pediatrics Continued approval may…

ByRidhi Rastogi4 hours ago

Ascendis Pharma Reports the EMA’s MAA Submission for TransCon CNP to Treat Achondroplasia in Children

Shots: The EMA has received MAA for TransCon CNP (navepegritide) for the treatment of children with achondroplasia; NDA is under the US FDA’s priority review (PDUFA: Nov 30, 2025) MAA is supported by 3 trials assessing TransCon CNP vs PBO & up to 3yrs. of OLE data, incl. ApproaCH trial results in children with achondroplasia,…

ByRidhi RastogiOctober 9, 2025

Tyra Biosciences Doses First Patient in P-II (BEACH301) Trial of Dabogratinib to Treat Achondroplasia

Shots: Tyra Biosciences has dosed its first patient with dabogratinib (FGFR3 inhibitor) in P-II (BEACH301) trial for the treatment achondroplasia Trial will evaluate dabogratinib in children (3–10 yrs) with achondroplasia & open growth plates across 2 cohorts: treatment-naïve & previously treated with growth-accelerating therapy, enrolling up to 10 pts per dose level (0.125, 0.25, 0.375…

ByRidhi RastogiAugust 22, 2025

Ascendis’ Yuviwel (Navepegritide) Receives the US FDA Accelerated Approval for Achondroplasia

Ascendis Pharma Reports the EMA’s MAA Submission for TransCon CNP to Treat Achondroplasia in Children

Tyra Biosciences Doses First Patient in P-II (BEACH301) Trial of Dabogratinib to Treat Achondroplasia

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