FDA Issued Complete Response Letter for Somatrogon for Pediatric GHD

A Complete Response Letter (CRL) was issued by the US Food and Drug Administration (FDA) for the Biologics License Application (BLA) for somatrogon (NGENLA). The news was announced by Pfizer and OPKO on Friday, January 21, 2022 but there was no explanation as to why the treatment wasn’t approved.

The investigational treatment for growth hormone deficiency (GHD) in pediatric patients is a once-weekly long-acting recombinant human growth hormone. Pfizer stated that it will evaluate the FDA’s comments in order to work with the agency on an appropriate path forward.

The late-stage study results that the application was based on, showed that somotrogan, as a once a week treatment, was non-inferior when compared with Pfizer’s approved growth hormone, Genotropin, which is administered daily.

“We remain confident in the potential treatment benefits that somatrogon has to offer patients around the world,” Brenda Cooperstone, MD, Chief Development Officer, Rare Disease, Pfizer Global Product Development said in a statement. “We will work closely with the FDA to determine the best path forward to bring this important once-weekly treatment option to pediatric growth hormone deficiency patients and their families.”

Somatrogon Around the World

Pfizer and OPKO joined a worldwide agreement in 2014 for the development and commercialization of somatrogon for treating GHD. Pfizer is responsible for registering and commercializing the product for GHD while OPKO is in charge of conducting the clinical program.

Several countries have been submitting regulatory applications for review of somatrogon around the world. Earlier this month, somatrogon injectable 24 mg Pens and 60 mg Pens were approved by Japan’s Ministry of Health, Labour and Welfare, for the long-term treatment of pediatric patients who have growth failure due to an inadequate secretion of endogenous growth hormone.

Last year, Health Canada and Australia’s Therapeutic Goods Administration (TGA) both approved somatrogon for long-term treatment of pediatric patients with GHD or growth disturbance due to insufficient secretion of growth hormone.

A decision from the European Commission (EC) is expected early this year regarding a recommendation for the treatment. In December 2021, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) issued the positive opinion recommendation for somatrogon marketing authorization in the EU to treat children and adolescents from 3 years of age who have growth disturbance due to insufficient secretion of growth hormone.

About Growth Hormone Deficiency

The rare disease presents as inadequate secretion of growth hormone from the pituitary gland and is usually caused by genetic mutations or, could be acquired after birth.

Because somatropin, the growth hormone, is secreting at inadequate levels in these patients, the height and onset of puberty can be affected by this disease.

In addition to persistent growth challenges, children can also experience other problems with physical health and mental wellbeing.