Pfizer is on an FDA heater. With a blessing Wednesday for growth hormone deficiency (GHD) drug Ngenla (somatrogon-ghla), the New York pharma giant has racked up four approvals from the agency over the last five weeks.
Last week, the FDA signed off on alopecia drug Litfulo. In late May, the regulator also endorsed Pfizer’s RSV vaccine Abrysvo and gave a full approval to COVID-19 antiviral pill Paxlovid.
Overall this year, including a green light in March for migraine treatment Zavzpret, Pfizer has racked up five U.S. approvals.
Wednesday’s win was hard-earned. A decision on the next generation, once-weekly injected treatment was initially set for October 2021 but the FDA asked Pfizer and its partner on the med, OPKO Health, for longer-term data.
Then in January of last year, the regulator sent a complete response letter to the companies, who didn’t reveal the regulator’s issues with the application.
The approval for Ngenla covers those age 3 and older who have stunted growth because of inadequate secretion of endogenous growth hormone from the pituitary gland.
GHD affects approximately one in every 4,000 to 10,000 children. Without treatment, children will have very short height in adulthood and puberty can be delayed.
Ngenla’s thumbs-up was supported by a phase 3 study, which evaluated its safety and effectiveness versus Genotropin, Pfizer’s standard-of-care drug that is administered daily. Ngenla achieved non-inferiority as measured by height increases after a year and its safety profile was comparable.
“As a new, longer-acting option that has the ability to reduce treatment frequency from daily to weekly, Ngenla could become an important treatment option that can improve adherence,” Joel Steelman, M.D., a pediatric endocrinologist at Cook Children’s Health Care System, said in a release.
In 2014, Pfizer partnered with Miami-based OPKO on the therapy, paying $295 million up front. Ngenla has been approved for at least a year in Europe, Japan, Australia and Canada.
Pfizer said it expects to launch it in the U.S. in August and will be sold at a monthly list price of $8,300 for a child weighing 35 kg (75 pounds).
The companies are playing catch up against Ascendis Pharma’s Skytrofa, which became the first once-weekly treatment for GHD when it was approved in August 2021. Sales of Skytrofa reached 36 million euros ($39 million) in 2022, but are increasing rapidly.
Pfizer felt the pressure applied by Denmark-based Ascendis as its sales of Genotropin fell from $389 million in 2021 to $360 million last year.