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Ascendis Pharma's Rare Dwarfism Candidate Shows Promise In Trial, FDA Submission Expected Next Year
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Ascendis Pharma's Rare Dwarfism Candidate Shows Promise In Trial, FDA Submission Expected Next Year
Sep 16, 2024 12:20 PM

Ascendis Pharma A/S ( ASND ) released topline data on Monday from a trial that included 84 children with achondroplasia, a genetic disorder that causes dwarfism and disproportionate short stature.

TransCon CNP (navepegritide), an investigational prodrug of C-type natriuretic peptide (CNP) administered once weekly and designed to provide sustained release and continuous exposure of active CNP.

CNP regulates blood flow, blood pressure, and the reactivity of platelets and leukocytes.

Also Read: Ascendis Pharma Faces Challenges With Skytrofa And Yorvipath Pricing: Analysts Weigh In.

In the trial, children treated with once-weekly TransCon CNP demonstrated annualized growth velocity (AGV) superior to placebo.

For the primary endpoint of AGV at Week 52, children treated with TransCon CNP (n=57) demonstrated an LS mean AGV of 5.89 cm/year compared to 4.41 cm/year in the placebo arm (n=27), an LS mean difference of 1.49 cm/year (p

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