Pfizer: Pfizer and OPKO’s Once-Weekly NGENLA™ (somatrogon) Injection Receives Marketing Authorization in European Union for Treatment of Pediatric Growth Hormone Deficiency

Pfizer Inc. (NYSE: PFE) and OPKO Health, Inc. (NASDAQ: OPK) announced today that the European Commission has granted marketing authorization for the next-generation long-acting recombinant human growth hormone NGENLA™ (somatrogon), a once-weekly injection to treat children and adolescents from 3 years of age with growth disturbance due to insufficient secretion of growth hormone. NGENLA provides pediatric patients, their caregivers and healthcare providers with a new treatment option for growth hormone deficiency (GHD) that reduces the frequency of required injections from once daily to once weekly.

“Growth hormone deficiency takes a substantial toll on children living with this rare disease and their families, and for many years, daily injections have been the standard of care, adding to the challenges they face,” said Reda Guiha, Regional President for International Developed Markets, Pfizer Rare Disease. “With NGENLA, we are proud to continue to advance the care of children in Europe who are impacted by growth hormone deficiency with a new, longer-acting option that significantly reduces treatment burden from once-daily to once-weekly injections.”

GHD is a rare disease characterized by the inadequate secretion of the growth hormone, somatropin, from the pituitary gland and affects one in approximately 4,000 to 10,000 children worldwide. The disease can be caused by genetic mutations or acquired after birth. Without treatment, affected children will have persistent growth attenuation and a very short height in adulthood, and puberty may be delayed.Children may also experience other problems with physical health and mental well-being.

“By helping to minimize disease management burden, we believe NGENLA has the potential to improve quality of life for children impacted by growth hormone deficiency and their families, as well as increase treatment adherence, which can improve outcomes,” said Phillip Frost, M.D., Chairman and Chief Executive Officer, OPKO Health. “We are pleased that the marketing authorization in the European Union will enable more children with growth hormone deficiency to benefit from once-weekly treatment.”

The European Union (EU) marketing authorization of NGENLA was supported by results from a global, randomized, open-label, active-controlled Phase 3 study which evaluated the safety and efficacy of once-weekly NGENLA compared to once-daily GENOTROPIN® (somatropin). The study met its primary endpoint of NGENLA non-inferiority compared to GENOTROPIN, as measured by annual height velocity at 12 months. NGENLA was generally well tolerated in the study and had a safety profile comparable to GENOTROPIN.

The marketing authorization of NGENLA is valid in all EU Member States as well as Iceland, Norway and Liechtenstein.

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