FDA Issues Historic Approval for Navepegritide in Achondroplasia

The US Food and Drug Administration (FDA) has approved navepegritide (Yuviwel), a once-weekly treatment, for increasing linear growth in children 2 years of age and older with achondroplasia with open epiphyses.

Billed by Ascendis Pharma as the first approved achondroplasia therapy to provide continuous systemic exposure to CNP over the weekly dosing interval, the agent received approval on February 27, 2026 based on a trio of randomized, double-blind, placebo-controlled clinical trials and up to 3 years of open-label extension data.¹

“The approval of once-weekly [navepegritide] is a major step forward in the treatment of children with achondroplasia, giving physicians for the first time the option of prescribing a once-weekly medicine backed by compelling efficacy and excellent tolerability data from three randomized, double-blind, placebo-controlled clinical trials,” said Carlos A. Bacino, MD, professor of Molecular and Human Genetics, Baylor College of Medicine and Texas Children’s Hospital.¹ “My goal is to help children and parents develop care plans tailored to their individual needs and objectives, and I look forward to adding [navepegritide] to my discussions with them.”

Approved under the FDA’s Accelerated Approval Program, the New Drug Application (NDA) for navepegritide was filed by Ascendis Pharma on March 31, 2025. Their original application was given an expected action date in late 2025. On November 25, 2026, Ascendis Pharma announced the FDA informed them additional information submitted to the agency related to the post-marketing requirement constituted a major amendment to the NDA and the review period was extended by 3 months to February 28, 2026.^1,2

“We are confident in [navepegritide]’s potential to transform the treatment of achondroplasia and are deeply grateful to patients, clinicians, and advocates for their many contributions to this important milestone,” said Jan Mikkelsen, President and Chief Executive Officer at Ascendis Pharma.¹ “We have listened to advocacy groups for people with dwarfism to ensure we address what the community actually cares about. This reflects our ongoing commitment to pursue outcomes that patient communities have told us are important to them, and gives the achondroplasia community a new way to look at the promise of pharmacological treatment options.”

According to Ascendis Pharma, commercial availability of navepegritide is expected during early part of Q2 2026. In their approval announcement, the company noted plans to offer a suite of patient services for navepegritide through its US Ascendis Signature Access Program, including support navigating the treatment journey and financial assistance programs for eligible patients.¹

The NDA for navepegritide is supported by 3 randomized, double-blind, placebo-controlled clinical trials and up to 3 years of open-label extension data, including the pivotal ApproaCH Trial, which was published in JAMA Pediatrics in November 2025.^1,2,3

The phase 2b APPROACH trial was a randomized, double-blind, placebo-controlled study conducted between March and August 2023 across 10 hospitals in Australia, Canada, Denmark, Ireland, New Zealand, Spain, and the United States. Eligible participants were children aged 2 to 11 years with genetically confirmed achondroplasia who were naïve to growth-promoting therapies and had documented height measurements at least 6 months before randomization. Key exclusion criteria included radiographic evidence of closed growth plates, planned bone surgery, severe untreated sleep apnea, or other conditions affecting growth.³

Of 86 screened patients, 84 were enrolled and randomized. Participants were randomized 2:1 to receive once-weekly subcutaneous navepegritide (100 μg/kg; n = 57) or placebo (n = 27) for 52 weeks, followed by an ongoing open-label extension. The primary endpoint was annualized growth velocity at week 52.³

The trial met its primary endpoint, with navepegritide demonstrating superiority over placebo. The least-squares mean difference in annualized growth velocity was 1.49 cm/year (95% CI, 1.05 to 1.93; P < .001). Treatment was also associated with improvements in skeletal parameters, including tibial-femoral angle (−1.81°), mechanical axis deviation (−2.78 mm), and fibula-to-tibia length ratio (−0.016). Improvements were observed in physical functioning among children younger than 5 years.³

“Little People of America, the largest national advocacy and support organization for people with dwarfism, is committed to ensuring that the voices of people with dwarfism remain central in conversations about research and medical options such as [navepegritide],” said the Board of Directors of Little People of America. “We champion dwarf and disability pride, advocate for inclusion and respect, and foster open dialogue across diverse perspectives.¹ Our goal is to empower individuals and families to make healthcare decisions that reflect their own values and experiences, while pushing for research efforts and new treatment options such as this that could have the potential to support outcomes that truly matter to our community.”

References:

1. Ascendis Pharma. FDA Approves Once-Weekly YUVIWEL® (navepegritide) for Children with Achondroplasia Aged 2 Years and Older | Ascendis Pharma. Ascendispharma.com. Published February 27, 2026. Accessed February 27, 2026. https://investors.ascendispharma.com/news-releases/news-release-details/fda-approves-once-weekly-yuviwelr-navepegritide-children

2. Ascendis Pharma. Ascendis Announces Extension of FDA Review Period for TransCon CNP (navepegritide) for Children with Achondroplasia | Ascendis Pharma. Ascendispharma.com. Published November 25, 2025. Accessed February 27, 2026. https://investors.ascendispharma.com/news-releases/news-release-details/ascendis-announces-extension-fda-review-period-transcon-cnp

3. Savarirayan R, McDonnell C, Bacino CA, et al. Once-Weekly Navepegritide in Children With Achondroplasia: The APPROACH Randomized Clinical Trial. JAMA Pediatr. 2026;180(1):18–25. doi:10.1001/jamapediatrics.2025.4771