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

NEW YORK, NY & MIAMI, FL, USA I February 15, 2022 I 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.

About the NGENLATM (somatrogon) Injection Studies

The safety and efficacy of NGENLA™ (somatrogon) was demonstrated in a randomized, open-label, active-controlled Phase 3 study conducted in over 20 countries. In this study, 224 prepubertal, treatment-naïve children with growth hormone deficiency (GHD) were randomized 1:1 to receive NGENLA once-weekly or GENOTROPIN® (somatropin) once-daily. 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. Participants had the opportunity to enroll in a global, open-label, multicenter, long-term extension study in which they were able to either continue receiving or switch to NGENLA. Approximately 95% of the patients switched into the open-label extension study and received NGENLA treatment.

An additional Phase 3 study (C0311002) assessed the perception of the treatment burden of once-weekly NGENLA versus once-daily GENOTROPIN among children three to <18 years of age with GHD and their caregivers. This randomized, multicenter, open-label, crossover study included 87 participants who were on stable growth hormone therapy: 43 were randomized to Sequence 1 (12 weeks of treatment with GENOTROPIN followed by 12 weeks of treatment with NGENLA) and 44 were randomized to Sequence 2 (12 weeks of treatment with NGENLA followed by 12 weeks of treatment with GENOTROPIN). The study demonstrated that treatment with NGENLA improved the mean overall Life Interference total score after 12 weeks of treatment compared to GENOTROPIN.

About NGENLATM (somatrogon) Injection

NGENLA™ (somatrogon) is a synthetic growth hormone that works by replacing the lack of growth hormone in the body. NGENLA is taken by injection just below the skin. Compared to the growth hormone GENOTROPIN® (somatropin), its action in the body lasts longer, enabling weekly injections instead of daily. NGENLA is approved for the treatment of pediatric growth hormone deficiency (GHD) in Canada, Australia and Japan, and the EU marketing authorization is valid in all EU Member States as well as Iceland, Norway and Liechtenstein.

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

About GENOTROPIN® (somatropin)

GENOTROPIN® (somatropin) is a man-made, prescription treatment option. The indications GENOTROPIN is approved for vary by market. GENOTROPIN is approved for growth failure due to growth hormone deficiency (GHD) and adult GHD, Prader-Willi Syndrome, Idiopathic Short Stature, Turner Syndrome, Small for Gestational Age (with no catch-up growth) and Chronic Renal Insufficiency. GENOTROPIN is taken by injection just below the skin and is available in a wide range of devices to fit a range of individual dosing needs. GENOTROPIN is just like the natural growth hormone that our bodies make and has an established safety profile.

GENOTROPIN Indications and Usage in the U.S.

GENOTROPIN is a prescription product for the treatment of growth failure in children:

  • Who do not make enough growth hormone on their own. This condition is called growth hormone deficiency (GHD).
  • With a genetic condition called Prader-Willi syndrome (PWS). Growth hormone is not right for all children with PWS. Check with your doctor.
  • Who were born smaller than most other babies born after the same number of weeks of pregnancy. Some of these babies may not show catch-up growth by age 2 years. This condition is called small for gestational age (SGA).
  • With a genetic condition called Turner syndrome (TS).
  • With idiopathic short stature (ISS), which means that they are shorter than 98.8% of other children of the same age and sex; they are growing at a rate that is not likely to allow them to reach normal adult height, and their growth plates have not closed. Other causes of short height should be ruled out. ISS has no known cause.

GENOTROPIN is a prescription product for the replacement of growth hormone in adults with GHD that started either in childhood or as an adult. Your doctor should do tests to be sure you have GHD, as appropriate.

Pfizer Rare Disease

Rare disease includes some of the most serious of all illnesses and impacts millions of patients worldwide, representing an opportunity to apply our knowledge and expertise to help make a significant impact on addressing unmet medical needs. The Pfizer focus on rare disease builds on more than two decades of experience, a dedicated research unit focusing on rare disease, and a global portfolio of multiple medicines within a number of disease areas of focus, including rare hematologic, neurologic, cardiac and inherited metabolic disorders.

Pfizer Rare Disease combines pioneering science and deep understanding of how diseases work with insights from innovative strategic collaborations with academic researchers, patients, and other companies to deliver transformative treatments and solutions. We innovate every day leveraging our global footprint to accelerate the development and delivery of groundbreaking medicines and the hope of cures.

Click here to learn more about our Rare Disease portfolio and how we empower patients, engage communities in our clinical development programs, and support programs that heighten disease awareness.

About Pfizer: Breakthroughs That Change Patients’ Lives

At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products, including innovative medicines and vaccines. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time. Consistent with our responsibility as one of the world's premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world. For more than 150 years, we have worked to make a difference for all who rely on us. We routinely post information that may be important to investors on our website at www.Pfizer.com. In addition, to learn more, please visit us on www.Pfizer.com and follow us on Twitter at @Pfizer and @Pfizer News, LinkedIn, YouTube and like us on Facebook at Facebook.com/Pfizer.

About OPKO Health, Inc.

OPKO is a multinational biopharmaceutical and diagnostics company that seeks to establish industry-leading positions in large, rapidly growing markets by leveraging its discovery, development, and commercialization expertise and novel and proprietary technologies. For more information, visit http://www.OPKO.com.

SOURCE: Pfizer

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