Carlos Bacino, MD, is the medical director of the cytogenetics laboratory at Baylor Genetics.

Carlos Bacino, MD

Once-Weekly Navepegritide Safe and Efficacious for Achondroplasia, With Carlos Bacino, MD

 approved Ascendis Pharma’s navepegritide treatment for 

 met with Carlos Bacino, MD, medical director of the Cytogenetics Laboratory at Baylor Genetics and a lead author on the pivotal ApproaCH study, which drove the FDA’s approval under the Accelerated Approval Program. Bacino discussed the reassuring safety data coming from the trial program.

“This is a drug that has been shown to be very safe in that the side effects have been mild and not clinically significant,” Bacino told 

. “These drugs do have an effect on blood pressure, potentially decreasing it – in this particular situation, this is something that has been looked at very carefully to determine if it has caused any clinical hypotension and decreasing blood pressure. In most cases, these side effects are minimal.”

Navepegritide is a prodrug of C-type natriuretic peptide (CNP) utilizing the TransCon technology platform, which is designed to transiently link an inert carrier to a parent drug with known biology. The moiety of CNP in navepegritide is identical to the amino acid sequence 89-126 of endogenous CNP – after weekly subcutaneous administration, autocleavage of the linker occurs under physiologic pH and temperature. This provides predictable, sustained release and continuous systemic exposure to active CNP.

The FDA’s decision was based on the pivotal ApproaCH phase 2b randomized, double-blind, placebo-controlled, multicenter trial, conducted across 10 academic hospitals across 7 countries. Patients aged 2-11 years with a genetically confirmed achondroplasia diagnosis who were naïve to treatment with growth-promoting agents and height recorded ≥6 months prior to randomization were eligible for inclusion. Patients with radiographic evidence of closed growth plates, severe untreated sleep apnea, or conditions known to affect growth, among other criteria, were excluded.

Patients were randomly assigned in a 2:1 ratio to receive either subcutaneous navepegritide at 100 µg/kg/week or placebo; both drugs were administered once weekly for 52 weeks, followed by 52 weeks of follow-up. A total of 86 patients were screened and 84 were enrolled, with 57 randomized to navepegritide and 27 to placebo. Navepegritide demonstrated superiority to placebo at week 52, as children receiving it achieved a least-squares mean AGV of 5.89 versus 4.42 cm/year for those treated with placebo.

Bacino also discussed the promising potential of weekly injections as opposed to daily treatment. He mentions the likelihood of increased adherence and more direct drug effects via the TransCon platform.

“The weekly injection will allow a much more physiologic delivery of the drug and will definitely improve adherence, as it is certainly better for children, who are often reluctant to needles,” Bacino said. “It’s also better for parents who are not necessarily happy about the frequency of injections, and it’s better for the busy family to be able to handle the drug regimen.”

Editors’ Note: Bacino reports disclosures with Ascendis Pharma, BioMarin Pharmaceutical, IONIS Pharmaceuticals, and Teladoc Health.

Ascendis Pharma. FDA Approves Once-Weekly YUVIWEL (navepegritide) for Children with Achrondroplasia Aged 2 Years and Older. February 27, 2026. Accessed April 15, 2026. 

https://investors.ascendispharma.com/news-releases/news-release-details/fda-approves-once-weekly-yuviwelr-navepegritide-children

Savarirayan R, McDonnell C, Bacino CA, et al. Once-weekly Navepegritide in children with achondroplasia. JAMA Pediatrics. 2026;180(1):18. 

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Bacino highlights the recently approved treatment’s safety data and emphasizes the likelihood of increased adherence due to longer intervals between administration.

Carlos Bacino, MD

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