FDA Approves Phathom's VOQUEZNA® Tablets for Heartburn Relief in Non-Erosive GERD

Phathom Pharmaceuticals, Inc., a biopharmaceutical company dedicated to novel treatments for gastrointestinal diseases, announced that the U.S. Food and Drug Administration (FDA) has granted approval for VOQUEZNA® (vonoprazan) 10 mg tablets. This medication is now available for the relief of heartburn associated with Non-Erosive Gastroesophageal Reflux Disease (Non-Erosive GERD) in adults. Non-Erosive GERD, a major segment within GERD conditions, affects millions of individuals in the U.S. who experience frequent heartburn.

This approval is a landmark moment for GERD patients, offering a new class of treatment after decades of limited options. Terrie Curran, President, and CEO of Phathom, emphasized the significance of this new therapy, highlighting its potential to transform patient care by providing complete, 24-hour heartburn relief. This approval marks the third for VOQUEZNA, which is also approved for treating all severities of Erosive Esophagitis (EE) and, in combination with antibiotics, for eradicating Helicobacter pylori (H. pylori) infection.

Non-Erosive GERD is characterized by reflux symptoms without esophageal mucosal erosions. An estimated 45 million adults in the U.S. suffer from this form of GERD, with about 15 million receiving prescription medication annually. Despite long-standing treatments, many patients remain dissatisfied and continue to experience heartburn, impacting their quality of life both day and night. Dr. Colin W. Howden, Professor Emeritus at the University of Tennessee College of Medicine, pointed out that VOQUEZNA's pivotal study demonstrated significant and rapid reduction in heartburn episodes, establishing a novel, first-in-class treatment option for physicians to offer their patients.

The FDA approval of VOQUEZNA was based on the positive results from the PHALCON-NERD-301 study, a Phase 3 randomized, placebo-controlled, double-blind trial. This study evaluated the efficacy and safety of VOQUEZNA for daily treatment in adults with Non-Erosive GERD. The trial included 772 adult patients who experienced frequent heartburn. Over four weeks, VOQUEZNA 10 mg was compared to a placebo in terms of heartburn relief. A 20-week extension allowed all participants to receive VOQUEZNA to assess long-term treatment outcomes.

Results showed that VOQUEZNA significantly reduced heartburn episodes more effectively than the placebo, achieving more 24-hour heartburn-free days throughout the four-week period. The mean percentage of heartburn-free days for VOQUEZNA was 45% compared to 28% for placebo, and the median percentage of 24-hour heartburn-free days was 48% against 17% for the placebo group. Improvements were also seen in heartburn-free nights and days without rescue antacid use. These findings were presented at Digestive Disease Week® 2024 and published in Clinical Gastroenterology and Hepatology.

The most common adverse reactions (≥2%) associated with VOQUEZNA included abdominal pain, constipation, diarrhea, nausea, and urinary tract infections. In the 20-week extension phase, upper respiratory tract infections and sinusitis were also reported. Phathom Pharmaceuticals provides savings programs for eligible patients facing coverage or affordability issues, including co-pay assistance for those with commercial insurance.

VOQUEZNA is available by prescription and marketed exclusively by Phathom Pharmaceuticals, Inc. For further information about the drug, interested parties are encouraged to visit the official VOQUEZNA website.

The PHALCON-NERD-301 study focused on U.S. patients experiencing heartburn related to Non-Erosive GERD, confirming the drug's efficacy and safety over a four-week placebo-controlled period, with an additional 20-week extension for long-term evaluation.

Non-Erosive GERD, affecting over 65 million U.S. patients, is characterized by symptoms without esophageal mucosal damage. Symptoms often include episodic heartburn, especially at night, regurgitation, swallowing difficulties, and chest pain.

VOQUEZNA (vonoprazan) tablets, a potassium-competitive acid blocker (PCAB), represent a novel class of medicines approved in the U.S. for treating various GERD forms and H. pylori infections. Phathom Pharmaceuticals in-licensed U.S. rights for vonoprazan from Takeda, which markets it in Japan and other regions.

Phathom Pharmaceuticals focuses on developing and commercializing treatments for gastrointestinal diseases, offering VOQUEZNA in various formulations for different GI conditions.