FDA Approves Pfizer, OPKO’s Once Weekly Ngenla for Human Growth Deficiency in Pediatric Patients

The FDA has approved somatrogon-ghla (Ngenla; Pfizer Inc, OPKO Health Inc) for treatment of pediatric individuals age 3 years and older who have growth failure due to inadequate secretion of endogenous growth hormone.¹

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Somatrogon-ghla is a once-weekly human growth hormone that replaces the lack of growth hormone in the body. It is a subcutaneous injection that is administered via a device that allows for titration based on the patient’s need.¹

“The approval of [somatrogon-ghla] will be significant for children with growth hormone deficiency in the [United States]. It holds potential to reduce the treatment burden that can come with daily growth hormone injections,” Joel Steelman, MD, a pediatric endocrinologist at Cook Children’s Health Care System, said in the statement. “As a new, longer-acting option that has the ability to reduce treatment frequency from daily to weekly, [somatrogon-ghla] could become an important treatment option that can improve adherence for children being treated for growth hormone deficiency.”¹

The FDA approval was supported by results from a multicenter, randomized open-label, active-controlled phase 3 study (NCT02968004), evaluating the safety and efficacy of somatrogon-ghla administered once weekly compared to once daily somatrogon (Genotropin; Pfizer, OPKO Health).¹

The study included 224 treatment-naïve pediatric individuals with growth hormone deficiency. Individuals were randomized 1:1 into 2 arms, with 1 arm receiving somatrogon-ghla once weekly at the dose strength of 0.66 mg/kg/day and the other receiving somatrogon once daily at the dose strength of 0.034 mg/kg/day.¹

Results showed that somatrogon-ghla met its primary end point of somatrogon-ghla non-inferiority compared with somatropin measured by annual height velocity at 12 months.¹

Additionally, somatrogon-ghla was generally well tolerated in the study and had a comparable safety profile to somatrogon.²

Individuals also had the opportunity to enrolled in the global, open-label, multicenter, long-term expansion study, which allowed them to either continue receiving or switch to somatrogon-ghla. Investigators reported that approximately 95% of individuals switched into the open-label extension study and received somatrogon-ghla.²

Furthermore, an additional phase 3 study (NCT03831880) assessed the perception of the treatment burden of the once weekly injection compared to the once daily injection among children aged 3 to 17 years old with growth hormone deficiency as well as their caregivers.²

The study included 87 individuals who were on stable growth hormone therapy, with 43 randomized to 12 weeks of treatment with somatrogon followed by 12 weeks of treatment with somatrogon-ghla and 44 randomized to 12 weeks of treatment with somatrogon-ghla followed by 12 weeks of treatment with somatrogon.²

Investigators found that treatment with somatrogon-ghla improved the mean overall Life Interference total score after 12 weeks of treatment when compared with somatrogon.²

Somatrogon-ghla is expected to become available for prescribing in the United States in August 2023, according to a statement by Pfizer.¹

Somatrogon-ghla is currently approved for the treatment of pediatric growth hormone deficiency in more than 40 markets, including Canada, Australia, Japan, and European Union Member States, according to the statement.¹

References

1. FDA approvals Pfizer’sNgenla, a long-acting once-weekly treatment for pediatric growth hormone deficiency. News release. Pfizer. June 28, 2023. Accessed June 29, 2023. https://www.pfizer.com/news/press-release/press-release-detail/fda-approves-pfizers-ngenlatm-long-acting-once-weekly
2. Pfizer and OPKO’s once-weekly Ngenla (somatrogon) injection receives marketing authorization in European Union for treatment of pediatric growth hormone deficiency. News release. Pfizer. February 15, 2022. Accessed June 29, 2023. https://www.pfizer.com/news/press-release/press-release-detail/pfizer-and-opkos-once-weekly-ngenlatm-somatrogon-injection