FDA Approves Phathom's Voquenza for Non-Erosive GERD

Phathom Pharmaceuticals, a leading biopharmaceutical company, has announced the FDA approval of Voquenza for treating Non-Erosive Gastroesophageal Reflux Disease (GERD). This approval marks a significant development for millions of GERD patients in the United States, offering a new class of treatment that promises 24-hour relief from heartburn. Terrie Curran, the President and CEO of Phathom Pharmaceuticals, emphasized the importance of this milestone, noting that this is the first FDA-approved treatment of its kind and is expected to significantly improve the lives of GERD patients.

Understanding Non-Erosive GERD

Non-Erosive GERD is a prevalent form of GERD, characterized by reflux-related symptoms without esophageal mucosal erosions. In the U.S., approximately 45 million adults live with this condition, and around 15 million seek prescription treatments annually. Despite the availability of existing treatments, many patients remain unsatisfied and continue to experience symptoms that adversely affect their quality of life.

Dr. Colin Howden, professor emeritus at the University of Tennessee College of Medicine, highlighted the need for new treatments. He pointed out that many patients with Non-Erosive GERD continue to suffer from heartburn despite current therapies. The pivotal study that led to Voquenza's approval demonstrated its significant efficacy in reducing heartburn episodes, backed by a strong safety profile. The new approval provides physicians with a novel treatment option that can significantly alleviate heartburn for many adult patients.

Supporting Clinical Evidence

The FDA's approval of Voquenza is based on the results from the Phalcon-Nerd-301 study (NCT05195528), a Phase 3 randomized, placebo-controlled, double-blind, multi-site trial conducted in the U.S. This study evaluated Voquenza's efficacy and safety for daily treatment in adults with non-erosive GERD. The trial included 772 adult patients who experienced heartburn four or more days per week, with most participants suffering from heartburn six to seven days a week. The findings revealed that Voquenza significantly reduced heartburn episodes, providing more complete 24-hour heartburn-free days and nights compared to a placebo.

The pivotal trial showed that patients taking Voquenza experienced heartburn-free days 45% of the time, compared to 28% for those on a placebo. The median percentage of 24-hour heartburn-free days was 48% for Voquenza users versus 17% for placebo users. Moreover, the trial included a 20-week extension period where all patients received Voquenza to assess long-term treatment effects.

Composition and Adverse Reactions of Voquenza

Voquenza tablets contain vonoprazan, an oral small molecule potassium-competitive acid blocker (PCAB). PCABs represent a novel class of medications that inhibit acid secretion in the stomach. Voquenza is approved in the U.S. for treating adults with erosive esophagitis, relieving heartburn associated with erosive GERD, non-erosive GERD, and for treating Helicobacter pylori infection in combination with antibiotics. Phathom Pharmaceuticals licensed the U.S. rights to vonoprazan from Takeda, which markets the product in Japan and several other countries in Asia and Latin America.

The most common adverse reactions reported during the four-week placebo-controlled trial included abdominal pain, constipation, diarrhea, nausea, and urinary tract infection. During the 20-week extension phase, upper respiratory tract infection and sinusitis were also noted.

A Significant Advancement in GERD Treatment

The FDA approval of Voquenza for non-erosive GERD signifies a substantial advancement in managing GERD. By introducing an effective, novel therapy, Phathom Pharmaceuticals aims to address the unmet needs of millions of patients, enhancing their quality of life and setting a new standard in GERD treatment.

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