Pfizer: EMA Adopts a Positive CHMP Opinion for Pfizer’s and OPKO’s Somatrogon, a Long-Acting Treatment for Pediatric Growth Hormone Deficiency

Pfizer Inc. (NYSE: PFE) announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending somatrogon, a once-weekly long-acting recombinant human growth hormone, for marketing authorization to treat children and adolescents from 3 years of age with growth disturbance due to insufficient secretion of growth hormone. A decision from the European Commission (EC) is expected in early 2022.

“The CHMP’s positive opinion of somatrogon is an important milestone in our efforts to deliver therapeutic options that can help children reach their full potential,” said Brenda Cooperstone, M.D., Chief Development Officer, Rare Disease, Pfizer Global Product Development. “For decades, Pfizer has been committed to improving the lives of those impacted by growth hormone deficiency and we look forward to working closely with the European Commission to hopefully soon provide a once-weekly treatment option for the pediatric growth hormone deficiency (GHD) community in the European Union.”

GHD is a rare disease characterized by the inadequate secretion of growth hormone, and affects one in approximately 4,000 to 10,000 children worldwide.1,2 Without treatment, affected children will have persistent growth attenuation and a very short height in adulthood.3,4 Children may also experience other problems with physical health and mental well-being.3,4

The recommendation for somatrogon is based on the results from a global, Phase 3 randomized, open-label, active controlled study which evaluated the safety and efficacy of once-weekly somatrogon compared to GENOTROPIN® (somatropin) for injection administered once-daily.5 This study met its primary endpoint of somatrogon non-inferiority compared to GENOTROPIN® (somatropin) for injection administered once daily, as measured by annual height velocity at 12 months.5

“Daily injections are often challenging for those impacted by pediatric growth hormone deficiency. Children may object to receiving a shot every day and caregivers may feel strains on their relationships,” said Jamie Harvey, CEO of the International Coalition of Organizations Supporting Endocrine Patients (ICOSEP). “At ICOSEP, we understand these challenges and are hopeful that if somatrogon receives marketing authorization in the European Union, this once-weekly treatment option may help children living with growth hormone deficiency reach their full potential.”

Separately, a Phase 3, randomized, multicenter, open-label, crossover study assessed the perception of treatment burden of once-weekly somatrogon compared to GENOTROPIN® (somatropin) for injection administered once-daily.6 Top-line results from the study demonstrated that treatment with somatrogon once-weekly improved the mean overall Life Interference total score after 12 weeks of treatment compared to treatment with somatropin administered once-daily.6

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