The company’s application for the investigational, oral small molecule potassium-competitive acid blocker vonoprazan is based on the positive results announced earlier from its pivotal Phase III PHALCON-EE trial.
The double-blind, multicentre, randomised, two-phase trial enrolled 1,024 patients with EE in the US and Europe and evaluated the safety and efficacy of vonoprazan as against lansoprazole.
PHALCON-EE met its primary as well as key secondary superiority endpoints successfully.
Phathom Pharmaceuticals chief operating officer Azmi Nabulsi said: “The submission of this NDA is another exciting step towards bringing the first major innovation to the US GERD market in over 30 years.
“Proton pump inhibitors (PPIs) are currently the standard of care for EE yet approximately half of all US patients progress their lines of therapy annually.
“We believe there is great interest among patients and healthcare providers for new treatment options to address the shortcomings of current treatment. If approved, vonoprazan has the potential to satisfy the large unmet needs of millions of patients and set a new treatment paradigm in EE.”
The company in-licensed the US, European, and Canadian rights to the oral therapy from Takeda.
EE affects nearly 20 million people in the US. Patients with inadequately treated EE will experience troubling heartburn symptoms and may also progress to more severe diseases including Barrett’s esophagus.