Phathom Pharmaceuticals submits Vonoprazan NDA to FDA for treatment of erosive esophagitis
Category: Small Molecules
Posted on Wednesday, March 16, 2022 11:59 AM
FLORHAM PARK, NJ, United States I March 14, 2022 I Phathom Pharmaceuticals, Inc. (Nasdaq: PHAT), a late-stage clinical-stage biopharmaceutical company focused on developing and commercializing novel treatments for gastrointestinal diseases, today announced that it has submitted a New Drug Application (NDA) to the United States Food and Drug Administration (FDA) for the use of vonoprazan as a treatment in adults for the healing of all grades of erosive esophagitis (EE) and the relief of heartburn , and maintaining healing of all grades of EE and relief from heartburn.
Erosive esophagitis, a major type of gastroesophageal reflux disease (GERD), affects approximately 20 million people in the United States. In addition to experiencing troubling symptoms of heartburn, insufficiently treated EE patients can progress to more serious conditions, including Barrett’s esophagus, a condition in which tissue changes can progress to cancer.
“The submission of this NDA is another exciting step towards bringing the first major innovation to the US GERD market in over 30 years,” said Azmi Nabulsi, MD, Chief Operating Officer at Phathom. “Proton pump inhibitors (PPIs) are currently the standard of care for EE, yet approximately half of all US patients advance their lines of treatment each year. We believe there is great interest among patients and healthcare providers for new treatment options to fill gaps in current treatments.If approved, vonoprazan has the potential to address the high unmet needs of millions of patients and establish a new treatment paradigm in EE.
This NDA is based on previously announced positive data from Phathom’s pivotal Phase 3 PHALCON-EE trial, a randomized, double-blind, multicenter trial that enrolled 1,024 patients with EE in the United States and Europe. and compared vonoprazan to lansoprazole, a PPI standard of care, in healing and maintaining healing of EE and relieving heartburn symptoms. PHALCON-EE successfully met its primary endpoints and its primary secondary superiority endpoints.
About erosive esophagitis
Erosive esophagitis is a condition characterized by the presence of breaks or erosions in the esophageal tissue caused by constant irritation of the mucosal surface and the subsequent loss of defense mechanisms against acids and digestive enzymes. Chronic erosive esophagitis can lead to complications including peptic stricture, narrowing of the esophagus that causes difficulty swallowing, and Barrett’s esophagus, a condition in which changes in the esophageal tissue can progress to cancer. Uncontrolled reflux can also lead to extra-esophageal diseases such as breathing problems, chest pain, angina, and increased mortality.
PHALCON-EE was a randomized, double-blind, two-phase, multicenter Phase 3 trial that enrolled 1,024 patients with EE in the United States and Europe. The first phase of the trial evaluated the efficacy and safety of vonoprazan 20 mg given once daily (QD) versus lansoprazole 30 mg QD for healing EE for up to eight weeks. The second phase of the trial evaluated the efficacy and safety of vonoprazan 10 mg QD and 20 mg QD compared to lansoprazole 15 mg QD for the maintenance of EE healing for 24 weeks. Both phases also assessed heartburn symptoms.
Vonoprazan is an investigational oral small molecule potassium-competitive acid blocker (P-CAB). P-CABs are a new class of drugs that block acid secretion in the stomach. Vonoprazan has shown the potential for rapid, potent, and long-lasting antisecretory effects as a single agent in the treatment of gastroesophageal reflux disease (GERD) and in combination with antibiotics for the treatment of Helicobacter pylori (H. pylori) infection. The FDA has granted Qualified Infection Disease Product (QIDP) status and Fast Track designation to vonoprazan in combination with amoxicillin and clarithromycin and with amoxicillin alone for the treatment of H. pylori infection. Phathom has licensed the US, European and Canadian rights to vonoprazan from Takeda, which has completed 19 Phase 3 trials for vonoprazan and received marketing approval in Japan and many other Asian countries and Latin America.
Phathom Pharmaceuticals is a biopharmaceutical company focused on developing and commercializing novel treatments for gastrointestinal diseases and disorders. Phathom has licensed exclusive rights in the United States, Europe and Canada to vonoprazan, a novel competitive potassium acid (P-CAB) inhibitor in late-stage development for the treatment of acidity. For more information about Phathom, visit the company’s website at www.phathompharma.com and follow the company on LinkedIn and Twitter.
THE SOURCE: Phathom