FDA Denies Approval of Long-Acting Pediatric Growth Hormone Therapy

The Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) to Pfizer and OPKO Health regarding the Biologics License Application for somatrogon for the treatment of growth hormone deficiency (GHD) in pediatric patients.

Somatrogon is an investigational once-weekly long-acting recombinant human growth hormone. The application included data from a phase 3 trial (ClinicalTrials.gov Identifier: NCT02968004) that compared the efficacy and safety of once-weekly somatrogon to daily injections with somatropin (Genotropin) in 224 pediatric patients with growth hormone deficiency.

Findings showed that treatment with somatrogon was noninferior to somatropin, as measured by annual height velocity at 12 months (primary endpoint; treatment difference in height velocity [cm/year]: 0.33 [95% CI, -0.39, 1.05]). Somatrogon was also associated with higher height standard deviation scores at 6 and 12 months compared with somatropin. The safety profile of somatrogon was observed to be comparable to that of somatropin.

Pfizer is evaluating the FDA’s comments in the CRL and will work with the Agency to determine the appropriate steps forward. “We remain confident in the potential treatment benefits that somatrogon has to offer patients around the world,” said Brenda Cooperstone, MD, Chief Development Officer, Rare Disease, Pfizer Global Product Development.

References

1. Pfizer and OPKO provide update on the Biologics License Application for somatrogon for pediatric growth hormone deficiency. News release. Pfizer Inc. and OPKO Health, Inc. January 21, 2022. Accessed January 24, 2022. https://www.businesswire.com/news/home/20220121005474/en/Pfizer-and-OPKO-Provide-Update-on-the-Biologics-License-Application-for-Somatrogon-for-Pediatric-Growth-Hormone-Deficiency
2. US FDA accepts regulatory submission from Pfizer and OPKO for review of somatrogon to treat pediatric patients with growth hormone deficiency. News release. Pfizer Inc. and OPKO Health, Inc. January 4, 2021. Accessed January 24, 2022. https://www.opko.com/news-media/press-releases/detail/417/us-fda-accepts-regulatory-submission-from-pfizer-and-opko