US FDA Granted Approval to Pfizer-OPKO's Drug for Growth Hormone Deficiency in Kids

The US FDA has authorized Pfizer Inc and partner OPKO Health Inc's medication for growth hormone insufficiency in children, the firms announced this week. The drug's clearance, which comes after an initial rejection by the FDA in January last year, raised Pfizer's shares modestly and Opko Health's by more than 27% in premarket trading. The injectable hormone therapy, to be marketed as Ngenla, has been approved for the treatment of paediatric patients aged three and up. According to the firms, it will be accessible in the United States in August. The firms did not react quickly to Reuters' request for comment on the drug's pricing.

Growth hormone deficiency is a rare disease that affects one in about 4,000 to 10,000 children, and is characterised by inadequate secretion of the growth hormone, resulting in very short height in adulthood and delayed puberty without treatment. For years, daily growth hormones injections have been the standard of care. But in recent years, some drugmakers have been focusing on long-acting growth hormones to improve patient convenience. Pfizer's Ngenla in a late-stage-study showed that the once-weekly injection was non-inferior compared to the company's approved growth hormone Genotropin, which is for daily administration.

The FDA was initially expected to make a decision on Ngenla by October 2021 but had pushed it back by three months after Pfizer submitted some additional data on the drug. With the latest approval of Ngenla, Pfizer will compete with Ascendis Pharma's once-weekly growth hormone injection, Skytrofa, which was approved by the FDA in 2021. Ngenla is already approved for treatment of pediatric GHD in more than 40 markets, including Canada, Japan, and countries in Europe.