FDA Tells Pfizer and OPKO It Won’t Approve Growth Hormone Therapy
January 24, 2022
The U.S. Food and Drug Administration issued a complete response letter to Pfizer and OPKO Health notifying the companies it would not approve their Biologics License Application for their experimental growth hormone treatment somatrogon.
The companies did not disclose the reason for the agency’s decision. In a press release Pfizer said it was reviewing the FDA’s comments and would work with the agency to determine the appropriate path forward.
“We remain confident in the potential treatment benefits that somatrogon has to offer patients around the world,” said Brenda Cooperstone, chief development officer of Rare Disease at Pfizer Global Product Development. “We will work closely with the FDA to determine the best path forward to bring this important once-weekly treatment option to pediatric growth hormone deficiency patients and their families.”
Somatrogon is an experimental once-weekly long-acting recombinant human growth hormone for the treatment of growth hormone deficiency in pediatric patients.
Growth hormone deficiency is a rare disease characterized by the inadequate secretion of growth hormone from the pituitary gland. In children, this disease can be caused by genetic mutations or acquired after birth. 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. Without treatment, affected children will have persistent growth attenuation and a very short height in adulthood. Children may also experience other problems with physical health and mental well-being.
Regulatory applications for somatrogon have been submitted to several countries around the world for review. Japan’s Ministry of Health, Labour and Welfare approved Ngenla (somatrogon) for the long-term treatment of pediatric patients who have growth failure due to an inadequate secretion of endogenous growth hormone. In 2021, Health Canada approved Ngenla for the long-term treatment of pediatric patients who have growth hormone deficiency, and Australia’s Therapeutic Goods Administration approved Ngenla for the long-term treatment of pediatric patients with growth disturbance due to insufficient secretion of growth hormone.
The Committee for Medicinal Products for Human Use of EMA issued a positive opinion in December recommending somatrogon for marketing authorization in the European Union to treat children and adolescents from 3 years of age with growth disturbance due to insufficient secretion of growth hormone. A decision from the European Commission is expected in early 2022.
In 2014, Pfizer and OPKO entered into a worldwide agreement for the development and commercialization of somatrogon for the treatment of growth hormone deficiency. Under the agreement, OPKO is responsible for conducting the clinical program and Pfizer is responsible for registering and commercializing the product for growth hormone deficiency.
Author: Rare Daily Staff
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