FDA approves drug for pediatric patients with most common form of dwarfism

The U.S. Food and Drug Administration (FDA) has approved Yuviwel (navepegritide) for injection to improve growth in pediatric patients aged two years and older with achondroplasia (the most common form of dwarfism) with open epiphyses (growth plates). This indication is approved under accelerated approval based on an improvement in annualized growth velocity (AGV), which is the rate of height growth. As a condition of the accelerated approval, there is an ongoing study to confirm that Yuviwel improves final adult height.

Condition

Achondroplasia is a genetic condition that causes short stature and disproportionate growth. The average height of an adult with achondroplasia is approximately four feet. People with achondroplasia have a genetic mutation that causes a certain growth regulation gene called fibroblast growth factor receptor 3 to be overly active, which prevents normal bone growth. Achondroplasia occurs in approximately 1 in 10,000 to 1 in 30,000 live births per year.

Data Supporting Yuviwel

The effectiveness of Yuviwel was established in a clinical trial that included a randomized, placebo-controlled 52-week double-blind treatment period, followed by a single-arm 52-week open-label extension period (Trial 1; NCT05598320).

The trial enrolled 84 pediatric patients with genetically confirmed achondroplasia who had never received treatment: 57 patients received Yuviwel 0.1 mg/kg administered subcutaneously (under the skin) once weekly, and 27 received a placebo.

The primary efficacy endpoint was the rate of height growth at Week 52. Patients receiving Yuviwel grew an average of 1.5 centimeters taller over the 52 weeks compared to those who received a placebo. In the open-label extension period, patients who received two years of Yuviwel treatment maintained the rate of height growth during the second year.

Safety Information

Transient low blood pressure has been reported with this class of medications. Patients should contact their health care provider if they experience symptoms of low blood pressure (e.g., dizziness, lightheadedness, fainting, blurred vision) while being treated with Yuviwel. The most common side effects of Yuviwel are vomiting, injection-site reactions, pain in extremity (discomfort in the limbs), and nausea.

Designations

Yuviwel received priority review designation and accelerated approval for this indication.