FDA Approves Voquezna by Phathom Pharmaceuticals for Non-Erosive GERD

Phathom Pharmaceuticals recently announced the U.S. Food and Drug Administration (FDA) approval of Voquenza for the treatment of Non-Erosive Gastroesophageal Reflux Disease (GERD). This novel approval marks the third FDA endorsement for Voquenza, which is also sanctioned for treating all severities of Erosive Esophagitis (EE), commonly referred to as Erosive GERD, and in combination with antibiotics for eradicating Helicobacter pylori (H. pylori) infection.

A landmark development for GERD sufferers, this approval opens a new avenue for millions of patients who have long awaited an innovative treatment option. Terrie Curran, President and CEO of Phathom Pharmaceuticals, emphasized the significance of this milestone: “Today's approval provides patients and healthcare providers with immediate access to the first and only FDA-approved treatment from a new class of acid suppression therapy. Voquenza promises complete 24-hour heartburn-free days and nights, offering a much-needed solution to the GERD community.”

Non-erosive GERD, characterized by reflux-related symptoms without the presence of esophageal mucosal erosions, is the largest category of GERD. In the United States alone, approximately 45 million adults are affected, with around 15 million receiving prescription medication annually. Despite available treatments, many patients remain unsatisfied and continue to experience symptoms that disrupt their quality of life.

Dr. Colin Howden, Professor Emeritus at the University of Tennessee College of Medicine, underscored the urgency for new treatment options, stating: “Millions of patients with Non-Erosive GERD continue to endure heartburn despite current therapies. Clinical studies leading to Voquenza's approval demonstrated its significant efficacy in reducing heartburn episodes, with a well-established safety profile. This novel treatment provides healthcare professionals with a new tool to effectively manage heartburn in adults.”

The FDA’s decision was based on promising results from the Phalcon-Nerd-301 study, a Phase 3 randomized, placebo-controlled, double-blind, multi-site trial conducted in the U.S. This study evaluated Voquenza's effectiveness and safety for daily use in adults suffering from non-erosive GERD. It included 772 adults who experienced heartburn four or more days per week, with most reporting symptoms six to seven days weekly. The study revealed that Voquenza significantly reduced heartburn episodes and offered more comprehensive 24-hour heartburn-free periods compared to placebo.

Key findings from the trial indicated that patients taking Voquenza experienced a mean percentage of 45% heartburn-free days versus 28% for those on placebo. Furthermore, the median percentage of 24-hour heartburn-free days was 48% for Voquenza users compared to 17% for the placebo group. A 20-week extension phase, where all participants received Voquenza, further assessed long-term treatment efficacy.

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

Voquenza tablets contain vonoprazan, an oral small molecule potassium-competitive acid blocker (PCAB). This novel class of medication blocks acid secretion in the stomach, offering a new therapeutic approach for GERD. Phathom Pharmaceuticals holds the U.S. rights to vonoprazan, in-licensed from Takeda, which markets the drug in Japan and several other countries in Asia and Latin America.

The FDA approval of Voquenza for non-erosive GERD represents a significant advancement in GERD treatment. Phathom Pharmaceuticals aims to address the unmet needs of millions of patients, enhancing their quality of life and setting a new standard in GERD management through this innovative therapy.

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