Pfizer (PFE) and OPKO (OPK) Announce EMA Accepts Marketing Application for Somatrogon to Treat Pediatric Patients with Growth Hormone Deficiency

Pfizer Inc. (NYSE: PFE) and OPKO Health Inc. (NASDAQ: OPK) announced today that the European Medicines Agency (EMA) has validated for review the Marketing Authorization Application (MAA) for somatrogon, a long-acting recombinant human growth hormone that is intended to be administered once-weekly for the treatment of pediatric patients with growth hormone deficiency (GHD). Pfizer expects a decision from the European Commission in 2022.

“Today’s announcement is an example of our decades-long commitment to actively support the pediatric growth hormone deficiency community through therapeutic options that help children reach their full potential,” said Brenda Cooperstone, MD, Chief Development Officer, Rare Disease, Pfizer Global Product Development. “If approved in the EU, somatrogon will represent an important advancement, as this long-acting, weekly treatment may reduce the burden of daily injections on children, their loved ones, and caregivers. We look forward to continuing to work with the EMA to evolve the treatment paradigm for pediatric patients with growth hormone deficiency.”

“We are very appreciative of the children and their families that participated in the clinical trials, bringing us one step closer to providing a long-acting treatment option for pediatric growth hormone deficiency. If somatrogon is approved, we look forward to positively impacting the quality of life for children living with GHD,” said Phillip Frost, Chairman and CEO of OPKO.

The submission is supported by the results of a global, Phase 3 trial evaluating the safety and efficacy of somatrogon administered once-weekly to pediatric patients with GHD. This study met its primary endpoint of non-inferiority compared to GENOTROPIN® (somatropin) for injection administered once daily, as measured by annual height velocity at 12 months. In addition, change in height standard deviation scores at 6 and 12 months, key secondary endpoints, were higher in the somatrogon dosed once-weekly cohort in comparison to the somatropin dosed once-daily cohort. Moreover, at 6 months, change in height velocity, another key secondary endpoint, was higher in the somatrogon dosed once-weekly cohort in comparison to the somatropin dosed once-daily cohort. These common measures of growth are employed in the clinical setting to measure the potential level of catch-up growth that subjects may experience relative to the heights of their age and gender matched peers.

Somatrogon was generally well tolerated in the study and comparable to that of somatropin administered once-daily with respect to the types, numbers and severity of the adverse events observed between the treatment arms.

Separately, in October 2020, Pfizer and OPKO announced findings from the C0311002 trial, a Phase 3, randomized, multicenter, open-label, crossover study evaluating somatrogon dosed once-weekly in children 3 to <18 years of age with GHD, which met its primary endpoint of improved treatment burden compared to GENOTROPIN® (somatropin) for injection administered once-daily. 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. In addition, key secondary endpoints showed an overall benefit in treatment experience with the somatrogon once-weekly dosing regimen compared to the somatropin once-daily dosing regimen. These data have also been submitted to the EMA.

In January 2021, Pfizer and OPKO announced that the US Food and Drug Administration (FDA) accepted for filing the initial Biologics License Application (BLA) for somatrogon with a target PDUFA action date of October 2021. In January 2021, a New Drug Application (NDA) was submitted to PMDA in Japan for somatrogon.

In 2014, Pfizer and OPKO 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 the product.

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