FDA Approves Pfizer’s Ngenla for Pediatric Growth Hormone Deficiency

The U.S. FDA has approved Pfizer and OPKO Health Inc.’s Ngenla, a once-weekly, human growth hormone analog for the treatment of pediatric patients aged three years and older who have growth failure due to inadequate secretion of endogenous growth hormone. Ngenla is expected to become available in August 2023.

Growth hormone deficiency (GHD) is a rare disease characterized by the inadequate secretion of the growth hormone somatropin from the pituitary gland, affecting one in approximately 4,000 to 10,000 children. Without treatment, children will have persistent growth attenuation, a very short height in adulthood, and puberty may be delayed. Children living with GHD may also experience challenges in relation to their physical health and mental well-being.

The FDA approval is supported by results from a multi-center, randomized, open-label, active-controlled Phase 3 study which evaluated the safety and efficacy of Ngenla when administered once-weekly compared to once-daily somatropin. The study met its primary endpoint of Ngenla non-inferiority compared to somatropin, as measured by annual height velocity at 12 months. Ngenla was generally well tolerated in the study and had a safety profile comparable to somatropin.

Ngenla is approved for the treatment of pediatric GHD in more than 40 markets including Canada, Australia, Japan, and EU Member States.