FDA approves vonoprazan for heart burn relief among patients with non-erosive GERD

The US Food and Drug Administration has approved vonoprazan for heartburn relief associated with non-erosive GERD.

The U.S. Food and Drug Administration (FDA) has approved VOQUEZNA® (vonoprazan) 10 mg tablets for the relief of heartburn associated with Non-Erosive Gastroesophageal Reflux Disease (Non-Erosive GERD) in adults. Non-Erosive GERD represents a substantial segment of the U.S. GERD population, affecting millions of individuals suffering from frequent heartburn. This is the third FDA approval for VOQUEZNA, which is also approved to treat all severities of Erosive Esophagitis (EE), also referred to as Erosive GERD, and in combination with antibiotics for the eradication of Helicobacter pylori (H. pylori) infection.

“Today marks a significant milestone for millions of GERD patients as we proudly announce the approval of VOQUEZNA for the treatment of Non-Erosive GERD,” said Terrie Curran, President, and Chief Executive Officer at Phathom. “For decades GERD sufferers had no new class of treatment to turn to in the U.S. This approval provides patients and healthcare providers with immediate access to the first and only FDA-approved treatment of its kind, from a new class of acid suppression therapy, and the power to help provide complete 24-hour heartburn-free days and nights. We are very excited to introduce VOQUEZNA to the broader GERD community and look forward to its potential to help change the way this disease is treated.”

Non-Erosive GERD is the largest category of GERD and is characterized by reflux-related symptoms in the absence of esophageal mucosal erosions. An estimated 45 million U.S. adults living with Non-Erosive GERD, and approximately 15 million are treated with a prescription medicine annually. Despite longstanding treatment options, many patients remain dissatisfied with such therapies and continue to suffer from heartburn symptoms which may impact overall quality of life with episodic heartburn, occurring during the day and at night.

“Millions of patients with Non-Erosive GERD continue to suffer from heartburn despite current treatment options,” said Colin W. Howden, M.D., Professor Emeritus, University of Tennessee College of Medicine. “The pivotal study that led to this approval showed that VOQUEZNA significantly reduced heartburn episodes in patients with Non-Erosive GERD along with an established safety profile. Today’s approval of VOQUEZNA provides physicians with a novel, first-in-class treatment that can quickly and significantly reduce heartburn for many adult patients.”

This approval is supported by the positive results from the PHALCON-NERD-301 study (NCT05195528), a Phase 3 randomized, placebo-controlled, double-blind, multi-site U.S. study evaluating the efficacy and safety of VOQUEZNA for the daily treatment of adults with Non-Erosive GERD. The trial enrolled 772 adult patients with Non-Erosive GERD who experienced four or more days of heartburn per week, with the majority having six to seven days of heartburn per week, and compared patients treated with VOQUEZNA 10 mg to placebo in the relief of heartburn over four weeks. The trial also included a 20-week extension period where all patients received VOQUEZNA to evaluate long-term treatment.

In the pivotal trial, VOQUEZNA quickly and significantly reduced heartburn with daily treatment through week 4. VOQUEZNA demonstrated the power of more complete all-day and all-night heartburn-free days with significantly more 24-hour heartburn-free days through week 4 versus placebo, the primary endpoint. The mean percentage of heartburn-free days for patients taking VOQUEZNA was 45% versus 28% for placebo (p<0.001), and the median percentage of 24-hour heartburn-free days was 48% versus 17%, respectively. Improvements for those taking VOQUEZNA were also seen in the percentage of each of heartburn-free days and nights, in addition to the percentage of days without rescue antacid use. Results from the pivotal study were previously presented at Digestive Disease Week® (DDW) 2024 and also published in Clinical Gastroenterology and Hepatology.

The most common adverse reactions (≥2%) reported in patients treated with VOQUEZNA during the four-week placebo-controlled trial include abdominal pain, constipation, diarrhea, nausea, and urinary tract infection. Upper respiratory tract infection and sinusitis were also reported in patients who received VOQUEZNA in the 20-week extension phase of the trial.

Phathom offers savings programs for eligible patients who face coverage or affordability issues, including co-pay assistance for patients with commercial insurance.