RARE Daily

FDA Approves Pfizer’s and Opko’s Ngenla Once-Weekly Treatment for Pediatric Growth Hormone Deficiency

June 28, 2023

Rare Daily Staff

The U.S. Food and Drug Administration has approved Pfizer’s and Opko Health’s Ngenla, a once-weekly, human growth hormone analog indicated for 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 for U.S. prescribing in August 2023. Ngenla is approved for the treatment of pediatric GHD in more than 40 markets including Canada, Australia, Japan, and EU Member States.

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.

Ngenla is a human growth hormone that works by replacing the lack of growth hormone in the body. Ngenla is taken by injection just below the skin, administered via a device that allows for titration based on patient need. Compared to the growth hormone somatropin, its action in the body lasts longer, enabling weekly injections instead of daily.

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.

“The approval of Ngenla will be significant for children with growth hormone deficiency in the U.S. It holds potential to reduce the treatment burden that can come with daily growth hormone injections,” said Joel Steelman, pediatric endocrinologist, Cook Children’s Health Care System. “As a new, longer-acting option that has the ability to reduce treatment frequency from daily to weekly, Ngenla could become an important treatment option that can improve adherence for children being treated for growth hormone deficiency.”

In 2014, Pfizer and Opko entered into a worldwide agreement for the development and commercialization of Ngenla for the treatment of GHD. Under the agreement, OPKO is responsible for conducting the clinical program and Pfizer is responsible for registering and commercializing Ngenla for GHD.

Photo: Joel Steelman, pediatric endocrinologist, Cook Children’s Health Care System

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