USA —In a significant development, the U.S. Food and Drug Administration (FDA) has granted approval to Pfizer Inc. and OPKO Health Inc for their groundbreaking treatment targeting growth hormone deficiency in children.
This decision comes as a reversal from the FDA’s initial rejection in January of the previous year, and it has resulted in a slight rise in Pfizer’s stock and a notable surge of nearly 19% in OPKO Health’s premarket trading.
The newly approved therapy, to be marketed under the brand name Ngenla, is an injectable hormone treatment designed for pediatric patients aged three years and above.
Pfizer has confirmed that the once-weekly injection will carry a monthly list price of approximately US$8,300, based on a child weighing 35 kg.
To enhance accessibility and alleviate financial burden for patients, the company has also committed to offering various benefits to reduce out-of-pocket costs and implement assistance programs.
Growth hormone deficiency is a rare condition that affects a small proportion of children, with an estimated incidence of one in every 4,000 to 10,000 individuals.
This disorder is characterized by insufficient production of the growth hormone, leading to stunted growth and delayed puberty if left untreated.
Additionally, the deficiency can negatively impact other aspects of a child’s physical health, including bone and cardiovascular health, as well as their mental well-being.
Developed by OPKO, Ngenla functions by replenishing the deficient growth hormone within the body.
Compared to Pfizer’s Genotropin, an FDA-approved medication for pediatric growth hormone deficiency, Ngenla offers the advantage of a longer duration of action, reducing the frequency of injections required.
Pfizer’s collaboration with OPKO for Ngenla began in December 2014, with Pfizer acquiring exclusive global commercialization rights for the treatment.
Under the agreement, Pfizer paid an upfront amount of US$295 million and committed to additional milestone payments of up to US$275 million.
Unfortunately, the FDA initially rejected Ngenla’s regulatory application in January 2022, and specific reasons for the denial were not disclosed.
To support their resubmission, Pfizer and OPKO presented data from a Phase III study, which was randomized, open-label, and active-controlled.
The study demonstrated that Ngenla, compared to Genotropin, achieved non-inferior improvements in the annual growth rate after 12 months of treatment.
Moreover, the safety profile of Ngenla was similar to that of Genotropin, with no significant differences in adverse events observed.
Traditionally, daily growth hormone injections have been the standard treatment approach. However, in recent years, pharmaceutical companies have been exploring the development of long-acting growth hormones to enhance patient convenience and adherence to treatment regimens.
Originally anticipated in October 2021, the FDA’s decision regarding Ngenla was delayed by three months to allow Pfizer to submit additional data on the drug.
Pfizer’s approval of Ngenla positions the company in competition with Ascendis Pharma’s Skytrofa, a once-weekly growth hormone injection that obtained FDA approval in 2021.
Notably, Ngenla has already received regulatory clearance for the treatment of pediatric growth hormone deficiency in over 40 markets, including Canada, Japan, and various European countries.
Pfizer and OPKO anticipate launching Ngenla in the United States in August 2023, marking a significant advancement in the treatment options available for children with growth hormone deficiency.
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