– Pfizer Inc. (NYSE: PFE) and OPKO Health Inc. (NASDAQ: OPK) announced today that the U.S. Food and Drug Administration (FDA) has extended the review period for the Biologics License Application (BLA) for somatrogon, a once-weekly long-acting recombinant human growth hormone, for the treatment of growth hormone deficiency (GHD) in pediatric patients. The Prescription Drug User Fee Act (PDUFA) goal date has been extended by three months to January 2022, as a result of Pfizer’s submission of additional data to the original BLA.
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 the product for GHD.
Somatrogon is an investigational 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 half-life of the molecule. Somatrogon has received Orphan Drug designation in the U.S. and the EU for the treatment of children and adults with growth hormone deficiency.
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.1,3 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 very short height in adulthood.3,4 Children may also experience other problems with physical health and mental well-being.3,4
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