Health Canada Approves NGENLA (somatrogon) Injection for Pediatric Growth Hormone Deficiency

New offering expands Pfizer's growth hormone portfolio of medicines and treatment options for rare diseases

KIRKLAND, QC, Oct. 29, 2021 /CNW Telbec/ - Pfizer Canada ULC announced that its next generation long-acting growth hormone injection, ^PrNGENLA^® (somatrogon), has been approved by Health Canada. NGENLA is a once-weekly long-acting recombinant human growth hormone, for the long-term treatment of pediatric patients who have growth failure due to an inadequate secretion of endogenous growth hormone (growth hormone deficiency or GHD).

NGENLA provides pediatric patients, their loved ones, and care providers with a treatment option that reduces the treatment frequency for children from daily injections to once-weekly injections.

"We are pleased that Canada is the first country to approve NGENLA," said Brenda Cooperstone, MD, Chief Development Officer, Rare Disease, Pfizer Global Product Development. "Today's announcement is an important milestone in our efforts to deliver therapeutic options that can help children reach their full potential."

GHD 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 worldwide.^1,2 In Canada, this represents approximately 1,600 children.⁶ Without treatment, affected children will have persistent growth attenuation and a short height in adulthood.^3,4 "This is wonderful news for growth hormone deficient patients across Canada, who now have another treatment option," says Dr. Cheri Deal, previous Chief of Pediatric Endocrinology at the CHU Sainte-Justine in Montreal. "The Endocrine Community and the patients and families affected with GHD have waited a long time for a means of reducing the frequency of injections."

In terms of clinical data, NGENLA injection has been granted a notice of compliance (NOC) based on safety and efficacy data from a global Phase 3 trial. The somatrogon Phase 3 trial is a randomized, open-label, active-controlled study conducted in over 20 countries, evaluating the safety and efficacy of NGENLA (somatrogon) injection. This study enrolled and treated 224 treatment-naïve children with growth hormone deficiency.⁷

"Pfizer Canada is proud to bring this new therapy to Canadians, demonstrating our long-standing commitment to advancing the care of those affected by GH disorders," says Frederick Little, Canada Lead ? Rare Disease, Pfizer Canada ULC. "Pfizer's 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."

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 somatrogon.

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 approximately 1,600 Canadian children.^1,2,6 In children, this disease can be caused by genetic mutations or acquired after birth.^1,5 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.^1,3,4  Without treatment, affected children will have persistent growth attenuation and a short height in adulthood.^3,4

About NGENLA^TM (somatrogon) injection
NGENLA^TM is a biologic product that is glycosylated and comprises the amino acid sequence of human growth hormone and one copy of the C-terminal peptide (CTP) from the beta chain of human chorionic gonadotropin (hCG) at the N-terminus and two copies of CTP (in tandem) at the C-terminus. The glycosylation and CTP domains account for the extended half-life of the molecule. In Canada, NGENLA^TM (somatrogon) injection is indicated for the long-term treatment of pediatric patients who have growth failure due to an inadequate secretion of endogenous growth hormone (growth hormone deficiency).

About Pfizer Canada
Pfizer Canada ULC is the Canadian operation of Pfizer Inc., one of the world's leading biopharmaceutical companies. Our diversified healthcare portfolio includes some of the world's best known and most prescribed medicines and vaccines. We apply science and our global resources to improve the health and well-being of Canadians at every stage of life. Our commitment is reflected in everything we do, from our disease awareness initiatives to our community partnerships. To learn more about Pfizer Canada, visit pfizer.ca or you can follow us on LinkedIn, Facebook, Twitter or YouTube.

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 Canada ULC