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

Article

Somatrogon-ghla (Ngenla; Pfizer Inc, OPKO Health Inc) is a subcutaneous injection that is administered once weekly via a device allowing for titration based on patients’ needs.

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
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