FDA Issues Complete Response Letter for Pfizer/OPKO Growth Hormone Drug
The U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter for the Biologics License Application of somatrogon, a long-acting human growth hormone treatment for pediatric growth hormone deficiency from Pfizer and OPKO Health.
Pfizer Inc. and OPKO Health, Inc. announced that the U.S. Food and Drug Administration (FDA) issued a Complete Response Letter (CRL) for their Biologics License Application (BLA) for somatrogon. Somatrogon is an investigational, once-weekly, long-acting recombinant human growth hormone intended for the treatment of growth hormone deficiency (GHD) in pediatric patients.
The companies stated that they are evaluating the FDA's comments and will work with the agency to determine an appropriate path forward. Pfizer and OPKO entered into a worldwide agreement for the development and commercialization of somatrogon in 2014, with OPKO responsible for the clinical program and Pfizer for registration and commercialization.
Growth hormone deficiency is a rare disorder that affects approximately 1 in 4,000 to 10,000 children. It is characterized by inadequate secretion of growth hormone from the pituitary gland, potentially causing growth failure and delayed puberty. Without treatment, affected children may experience significantly reduced adult height and other physical and psychosocial issues.
While the U.S. application received a CRL, somatrogon has seen recent approvals in other regions. Japan's Ministry of Health, Labour and Welfare approved NGENLA® (somatrogon) for the long-term treatment of pediatric GHD. Health Canada and Australia's Therapeutic Goods Administration (TGA) have also approved NGENLA® for the same indication. Additionally, the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) issued a positive opinion in December 2021 recommending marketing authorization in the EU, with a decision from the European Commission expected in early 2022.