Pfizer and OPKO Release Biologics Submission Update for Somatrogon in Pediatric Growth Hormone Deficiency


Pfizer Inc. (NYSE: PFE) and OPKO Health, Inc. (NASDAQ: OPK) announced today that the U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) for the Biologics License Application (BLA) issued for Somatrogon. Somatrogon is a once-weekly, long-acting recombinant human growth hormone for the treatment of growth hormone deficiency (GHD) in pediatric patients. Pfizer is evaluating the FDA’s comments and will work with the agency to determine an appropriate path forward.

“We remain committed to the potential treatment benefits that somatrogon has to offer patients around the world,” said Brenda Cooperstone, MD, Chief Development Officer, Rare Disease, Pfizer Global Product Development. “We will be working closely with the FDA to determine the best way to make this important once-weekly treatment option available to pediatric growth hormone-deficient patients and their families.”

Marketing Authorization Applications for Somatrogon have been submitted for review in several countries around the world. Earlier this week, the Japanese Ministry of Health, Labor and Welfare approved NGENLA® (Somatrogon) Inj. 24 mg pens and 60 mg pens for the long-term treatment of pediatric patients with growth failure due to inadequate endogenous growth hormone secretion. In 2021, Health Canada approved NGENLA® for the long-term treatment of pediatric patients with GHD, and the Australian Therapeutic Goods Administration (TGA) has approved NGENLA® for the long-term treatment of pediatric patients with growth failure due to insufficient growth hormone secretion. In addition, in December 2021, EMA’s Committee for Medicinal Products for Human Use (CHMP) issued a positive opinion recommending Somatrogon for marketing authorization in the EU for the treatment of children and adolescents aged 3 years and older with growth failure due to insufficient growth hormone secretion. A decision by the European Commission (EC) is expected in early 2022.

In 2014, Pfizer and OPKO entered into a global agreement to develop and commercialize Somatrogon for the treatment of GHD. Under the terms of the agreement, OPKO is responsible for conducting the clinical program and Pfizer is responsible for registering and commercializing the product for GHD.

About growth hormone deficiency

Growth hormone deficiency is a rare condition characterized by insufficient secretion of growth hormone from the pituitary gland, affecting one in about 4,000 to 10,000 children.1.2 In children, this disease can be caused by genetic mutations or acquired after birth.1.3 Because the patient’s pituitary gland secretes insufficient amounts of somatropin, the growth hormone, the child’s height may be affected and puberty may be delayed.1,3,4 Without treatment, affected children will have persistent growth retardation and very short stature in adulthood.3.4 Children may also have other physical health and mental well-being issues.3.4

Pfizer Rare Disease

Rare diseases comprise some of the most devastating of all diseases and affect millions of patients worldwide. This represents an opportunity to use our knowledge and experience to make a significant contribution to addressing unmet medical needs. Pfizer’s focus on rare diseases is based on more than two decades of experience, a dedicated research unit focused on rare diseases, and a global portfolio of multiple medicines across a range of disease priorities, including rare hematological, neurological, cardiac and inherited metabolic disorders.

Pfizer Rare Disease combines breakthrough science and a 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 breakthrough medicines and the hope of a cure.

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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 healthcare products, including innovative medicines and vaccines. Every day, Pfizer colleagues in developed and emerging markets work to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time. In line with our responsibility as one of the world’s leading innovative biopharmaceutical companies, we work with healthcare providers, governments and local communities to support and expand access to reliable and affordable healthcare around the world. For more than 150 years, we’ve worked to make a difference for everyone who counts on us. We routinely post information that may be important to investors on our website at www.Pfizer.com. Also, to learn more, visit us at www.Pfizer.com and follow us on Twitter at @Pfizer and @Pfizer News, LinkedIn, youtube and like us on Facebook at Facebook.com/Pfizer.

Disclosure Notice

The information contained in this press release is current as of January 21, 2022. Pfizer and OPKO undertake no obligation to update any forward-looking statements contained in this press release as a result of new information or future events or developments.

This press release contains forward-looking information about an investigational growth hormone deficiency therapy, Somatrogon, including a potential indication in the U.S. for the once-weekly treatment of pediatric growth hormone-deficient patients, including its potential benefits, which may involve significant risks and uncertainties that could result in that actual results may differ materially from those expressed or implied by such statements. Risks and uncertainties include, among other things, the uncertainties inherent in research and development, including the ability to achieve anticipated clinical endpoints, initiation and/or completion dates for our clinical trials, regulatory submission dates, regulatory approval dates and/or launch dates, as well as the possibility of unfavorable new clinical data and further analysis of existing clinical data; the risk that clinical trial data may be subject to differing interpretations and assessments by regulatory authorities; whether regulatory authorities will be satisfied with the design and results of our clinical trials; uncertainties regarding the Company’s ability to address the comments in the full response letter to FDA’s satisfaction;whether and when drug applications may be filed in other jurisdictions for a potential indication for somatrogon; whether and when the FDA-pending BLA for somatrogon for the treatment of pediatric patients with growth hormone deficiency may be approved and whether and when regulatory authorities in any jurisdiction may approve such other pending or filed applications (including the application filed in the EU) that are being processed by myriad factors, including determining whether the benefits of the product outweigh its known risks, determining the product’s efficacy and, if approved, whether Somatrogon will be commercially successful; decisions by regulatory authorities affecting labeling, manufacturing processes, safety and/or other matters that could affect the availability or commercial potential of somatrogon; uncertainties regarding the impact of COVID-19 on Pfizer’s and OPKO’s 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 reports on Form 10-K for the year ended December 31, 2020 and their respective subsequent reports on Form 10-Q, including in the sections headed “Risk Factors ‘ and ‘Forward-Looking Information and Factors That May Affect Future Results’ and their respective subsequent reports on Form 8-K, all of which will be filed with the US Securities and Exchange Commission and are available at www.sec.gov, www.pfizer.com and www.opko.com.

About OPKO Health, Inc.

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


1 National Organization for Rare Diseases. growth hormone deficiency. https://rarediseases.org/rare-diseases/growth-hormone-deficiency/. Retrieved August 24, 2021.

2 Stanley T. Diagnosis of childhood growth hormone deficiency. Curr Opin Endocrinol Diabetes Obes. 2012;19(1):47-52. doi:10.1097/MED.0b13e32834ec952.

3 Cerbone M, Dattani MT. Progression from isolated growth hormone deficiency to combined pituitary hormone deficiency. Growth Hormone IGF Res. 2017;37:19-25. doi:10.1016/j.ghir.2017.10.005.

4 Ergun-Longmire B, Wajnrajch M. Growth and growth failure. 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.

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