NEW YORK & MIAMI–(BUSINESS WIRE) June 28, 2023 — Pfizer Inc. (NYSE: PFE) and OPKO Health Inc. (NASDAQ: OPK) announced today that the U.S. Food and Drug Administration (FDA) has approved Ngenla (somatrogon-ghla), a once-weekly, human growth hormone analog indicated for treatment of pediatric patients aged three years and older who have growth failure due to inadequate secretion of endogenous growth hormone. Ngenla is expected to become available for U.S. prescribing in August 2023.
Growth hormone deficiency (GHD) is a rare disease characterized by the inadequate secretion of the growth hormone somatropin from the pituitary gland, affecting one in approximately 4,000 to 10,000 children.1,2 Without treatment, children will have persistent growth attenuation, a very short height in adulthood, and puberty may be delayed.1,2,3 Children living with GHD may also experience challenges in relation to their physical health and mental well-being.1,2,3
“For more than 30 years, Pfizer has been committed to supporting children and adults living with growth hormone deficiency, beginning with the delivery of a medicine that has long been a part of the standard of care,” said Angela Hwang, Chief Commercial Officer, President, Global Biopharmaceuticals Business, Pfizer. “We are excited to bring this next-generation treatment to patients in the United States, continuing our commitment to helping children living with this rare growth disorder reach their full potential.”
The FDA approval is supported by results from a multi-center, randomized, open-label, active-controlled Phase 3 study which evaluated the safety and efficacy of Ngenla when administered once-weekly compared to once-daily somatropin. The study met its primary endpoint of Ngenla non-inferiority compared to somatropin, as measured by annual height velocity at 12 months. Ngenla was generally well tolerated in the study and had a safety profile comparable to somatropin.
“The approval of Ngenla will be significant for children with growth hormone deficiency in the U.S. It holds potential to reduce the treatment burden that can come with daily growth hormone injections,” said Joel Steelman, M.D., Pediatric Endocrinologist, Cook Children’s Health Care System. “As a new, longer-acting option that has the ability to reduce treatment frequency from daily to weekly, Ngenla could become an important treatment option that can improve adherence for children being treated for growth hormone deficiency.”
“Throughout our collaboration with Pfizer, we have worked tirelessly toward our shared goal of helping children living with growth hormone disease and their families,” said Phillip Frost, M.D., Chairman and Chief Executive Officer, OPKO Health. “We are proud of the clinical development program that supported the FDA approval of Ngenla and are excited about its potential for these patients and their families as it becomes available in the United States.”
Ngenla is approved for the treatment of pediatric GHD in more than 40 markets including Canada, Australia, Japan, and EU Member States.
About Ngenla(somatrogon-ghla) Injection
Ngenla (somatrogon-ghla) is a human growth hormone that works by replacing the lack of growth hormone in the body. Ngenla is taken by injection just below the skin, administered via a device that allows for titration based on patient need. Compared to the growth hormone GENOTROPIN® (somatropin), its action in the body lasts longer, enabling weekly injections instead of daily.
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 the Ngenla Clinical Program
The safety and efficacy of Ngenla (somatrogon-ghla) was demonstrated in a multi-center, randomized, open-label, active-controlled Phase 3 study (NCT 02968004). The Phase 3 study enrolled and treated 224 pediatric patients, treatment-naïve children with growth hormone deficiency who were randomized 1:1 into two arms: Ngenla (somatrogon-ghla) once-weekly at a dose of 0.66 mg/kg/day vs somatropin, once-daily at a dose of 0.034 mg/kg/day. The study met its primary endpoint of Ngenla non-inferiority compared to somatropin, measured by annual height velocity at 12 months.
About Growth Hormone Deficiency
Growth hormone deficiency is a rare disease characterized by the inadequate secretion of growth hormone from the pituitary gland and affects one in approximately 4,000 to 10,000 children.1,2 In children, this disease can be caused by genetic mutations or acquired after birth.1,4 Because the patient’s pituitary gland secretes inadequate levels of somatropin, the hormone that causes growth, a child’s height may be affected and puberty may be delayed.1,2,5 Without treatment, affected children will have persistent growth attenuation and a very short height in adulthood.1,2 Children may also experience challenges in relation to physical health and mental well-being.1,2
Important Ngenla (somatrogon-ghla) Safety Information
About GENOTROPIN (somatropin)
GENOTROPIN is a man-made, prescription treatment option. The indications GENOTROPIN is approved for vary by market. GENOTROPIN is approved for growth failure due to 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.
Important GENOTROPIN (somatropin) Safety Information
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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 170 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
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 www.opko.com.
DISCLOSURE NOTICE: The information contained in this release is as of June 28, 2023. Pfizer and OPKO assume no obligation to update forward-looking statements contained in this release as the result of new information or future events or developments.
This release contains forward-looking information about Ngenla (somatrogon-ghla) injection and the U.S. FDA approval to treat pediatric patients aged three years and older with growth failure due to inadequate secretion of endogenous growth hormone, including its potential benefits, that involves substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. Risks and uncertainties include, among other things, uncertainties regarding the commercial success of Ngenla; the uncertainties inherent in research and development, including the ability to meet anticipated regulatory submission dates, regulatory approval dates and/or launch dates, as well as the possibility of unfavorable new clinical data and further analyses of existing clinical data; the risk that clinical trial data are subject to differing interpretations and assessments by regulatory authorities; whether regulatory authorities will be satisfied with the design of and results from our clinical studies; whether and when drug applications may be filed in any additional jurisdictions for Ngenla injection for the treatment of pediatric patients with growth hormone deficiency or in any jurisdictions for any other potential indications for Ngenla injection; whether and when regulatory authorities in any jurisdictions may approve any applications that may be pending or filed for Ngenla, which will depend on myriad factors, including making a determination as to whether the product’s benefits outweigh its known risks and determination of the product’s efficacy and, if approved, whether Ngenla injection will be commercially successful; decisions by regulatory authorities impacting labeling, manufacturing processes, safety and/or other matters that could affect the availability or commercial potential of Ngenla injection; uncertainties regarding the impact of COVID-19 on Pfizer’s and OPKO’s respective business, operations and financial results; and competitive developments.
A further description of risks and uncertainties can be found in Pfizer’s and OPKO’s respective Annual Report on Form 10-K for the fiscal year ended December 31, 2022 and in their respective subsequent reports on Form 10-Q, including in the sections thereof captioned “Risk Factors” and “Forward-Looking Information and Factors That May Affect Future Results”, as well as in their subsequent reports on Form 8-K, all of which are filed with the U.S. Securities and Exchange Commission and available at www.sec.gov, and www.pfizer.com in the case of Pfizer, and www.opko.com in the case of OPKO.
1 National Organization for Rare Disorders. Growth Hormone Deficiency. https://rarediseases.org/rare-diseases/growth-hormone-deficiency/. Accessed February 22, 2023.
2 Stanley T. Diagnosis of growth hormone deficiency in childhood. Curr Opin Endocrinol Diabetes Obes. 2012;19(1):47-52. doi:10.1097/MED.0b13e32834ec952.
3 Brod, M, Højbjerre, L, Alolga, SL, Beck, JF, Wilkinson, L, Rasmussen, MH. Understanding treatment burden for children treated for growth hormone deficiency. The Patient-Patient-Centered Outcomes Research. 2017;10(5):653-666.
4 Cerbone M, Dattani MT. Progression from isolated growth hormone deficiency to combined pituitary hormone deficiency. Growth Horm IGF Res. 2017;37:19-25. doi:10.1016/j.ghir.2017.10.005.
5 Ergun-Longmire B, Wajnrajch M. Growth and growth disorders. Feingold KR, Anawalt B, Boyce A, et al., editors. Endotext [Internet]. South Dartmouth (MA): MDText.com, Inc.; 2000. Available from: https://www.ncbi.nlm.nih.gov/books/NBK279142/
Source: Pfizer Inc.
Posted: June 2023
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- US FDA Accepts Regulatory Submission from Pfizer and OPKO for Review of Somatrogon to Treat Pediatric Patients with Growth Hormone Deficiency – January 4, 2021
Ngenla (somatrogon-ghla) FDA Approval History
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