Yuviwel Approved for Achondroplasia

On Feb. 27, 2026, the US Food and Drug Administration (FDA) approved Ascendis Pharma’s Yuviwel^® (navepegritide) for the treatment of children aged two years and older with achondroplasia who have open epiphyses, meaning their bone growth plates have not yet closed. Achondroplasia is a rare genetic condition caused by a variant in the fibroblast growth factor receptor 3 (FGFR3) gene that disrupts normal bone growth and alters signaling pathways throughout the body. Yuviwel, developed as TransCon CNP^®, is the first once-weekly, subcutaneous (SC) injectable prodrug of C-type natriuretic peptide (CNP) designed to provide continuous systemic exposure to CNP over a weekly dosing interval, counteracting overactive FGFR3 signaling that restricts bone growth in achondroplasia. The recommended dose is weight-based and administered once weekly by SC injection. Commercial availability is expected early in the second quarter of 2026. Pricing has not yet been disclosed. Full prescribing information can be found here.

At a Glance

• Brand Drug: Yuviwel^® (navepegritide)
• Manufacturer: Ascendis Pharma
• Date Approved: Feb. 27, 2026
• Indication: To increase linear growth in children two years of age and older with achondroplasia with open epiphyses
• Dosage Forms Available: 1.3mg, 2.8mg, and 5.5mg in a single-dose vial for reconstitution
• Launch Date: Early Q2 2026
• Estimated Annual Cost: Not yet known.
• FDA Designation: Rare Pediatric Disease Priority Review Voucher.
• Achondroplasia is a rare genetic skeletal dysplasia and the most common cause of short-limbed dwarfism. Characteristics of achondroplasia are due to malformation of bones and include overall shortness (usually under five feet adult height), short arms, short bowed legs, spinal curvature, and unusually large heads with bulging foreheads and flattened facial areas (frontal bossing). Individuals who have it also may have breathing and lung conditions, such as sleep apnea, mainly resulting from narrow upper airways and small ribcages.
• It affects an estimated 30,000 people in the US and occurs in roughly 100 to 400 babies each year. While about one‑fifth of cases are inherited, most result from spontaneous genetic mutations. Roughly one‑quarter of affected Americans are younger than 18 years, when bone growth plates remain open, and height increases are still possible.
• Accelerated approval was based on data from three randomized, double-blind, placebo-controlled clinical trials and up to three years of open-label extension data, demonstrating improvements in annualized growth velocity in children compared with placebo. Continued approval may be contingent upon verification and description of clinical benefit in confirmatory trial(s).
• The most common side effects reported with Yuviwel are injection‑site reactions, including redness, itching, skin discoloration, bleeding, swelling, bruising, pain, and blistering.
• BioMarin’s Voxzogo^® (vosoritide), a CNP analog that’s administered as a once daily SC injection, was granted accelerated approval in 2021 for achondroplasia for children who are at least five years old and whose epiphyses have not yet closed. In 2023, the label was expanded to include all children with open growth plates, regardless of age.
• BridgeBio Pharma’s infigratinib, an oral FGFR1–3 selective tyrosine kinase inhibitor, is in phase III development for the treatment of achondroplasia in pediatric patients with open epiphyses. Approval is possible in 2027.