A Pfizer biologic drug developed to supply extra handy dosing for kids with progress hormone deficiency has been turned down by the FDA.
Pfizer disclosed the regulatory motion late Friday. FDA full response letters should not public paperwork, and the corporate didn’t say what considerations or questions had been raised by the company in regards to the drug, somatrogon. Pfizer solely stated it’s evaluating the FDA’s feedback and can work with the company to find out the trail ahead for the drug.
Progress hormone deficiency is a uncommon illness that impacts each youngsters and adults. In youngsters, the situation results in progress and growth issues. Progress hormone deficiency that develops in adults may cause a rise in fats and a lower in muscle mass, in addition to excessive levels of cholesterol. Genetic mutations may cause progress hormone deficiency; when it’s acquired, the trigger will be linked to trauma, an infection, radiation remedy, or a tumor within the mind. In some circumstances, there isn’t any recognized trigger.
Remedy for the deficiency is injections of human progress hormone, sometimes given day by day. Pfizer already markets one such product, Genotropin, a drug that generated $427 million in income in 2020. Somatrogon is a model of human progress hormone engineered to supply a long-acting impact that allows once-weekly dosing. The drug is run by way of a pre-filled injection pen. In Section 3 testing in youngsters, somatrogon met the principle purpose of exhibiting that it was not inferior to day by day injections of Genotropin as assessed by measuring the speed of peak progress at 12 months. Sufferers within the scientific trial tolerated somatrogon effectively and antagonistic occasions reported within the scientific trial had been comparable throughout each arms.
Sufferers have already got a brand new possibility for once-weekly human progress hormone injections. Final August, the FDA accredited Skytrofa, which was developed by Copenhagen, Denmark-based Ascendis Pharma. The regulatory determination made the product the primary once-weekly product accredited for treating progress hormone deficiency in youngsters. On Jan. 13, European regulators accredited the Ascendis drug in the identical indication. Pivotal pediatric scientific trials are underway in Japan and China. Additionally, a worldwide Section 3 research is evaluating Skytrofa as a remedy for grownup human progress hormone deficiency.
Pfizer acquired international rights to somatrogon in a 2014 deal that paid Opko Well being $295 million up entrance. An extra $275 million is tied to regulatory milestones, and the deal additionally places Opko in line for royalties and a share of earnings from the product’s gross sales. Below the phrases of the settlement, Opko was chargeable for scientific growth of the drug whereas Pfizer would deal with regulatory and commercialization work, together with manufacturing.
Although the entire response letter for somatrogon is a setback, Pfizer is urgent onward with commercialization and regulatory purposes in different markets. The drug is accredited in Japan, the place it’s marketed below the title Ngenla. It’s additionally accredited to be used in Canada and Australia. A regulatory determination in Europe is anticipated early this 12 months.
Picture by Flickr person Sean MacEntee by way of a Artistic Commons license