Vonoprazan (Voquezna) Receives FDA Approval for Heartburn Associated with Non-Erosive GERD

Credit: US Food and Drug Administration

The US Food and Drug Administration has approved Phathom Pharmaceuticals’ vonoprazan (Voquezna) as a daily treatment for heartburn associated with non-erosive gastroesophageal reflux disease (GERD) in adult patients.¹

Announced on July 18, 2024, the decision is based on data from the phase 3 PHALCON-NERD-301 study evaluating the efficacy and safety of vonoprazan as a daily treatment in adult patients with non-erosive GERD, also known as NERD.¹

“Millions of patients with Non-Erosive GERD continue to suffer from heartburn despite current treatment options,” said Colin W. Howden, MD, professor emeritus at the University of Tennessee College of Medicine.¹ “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.”

An oral small molecule potassium-competitive acid blocker (PCAB), vonoprazan blocks acid secretion in the stomach and is the first PCAB to earn approval in the US. Following the receipt of a Complete Response Letter relating to specifications and controls for a nitrosamine drug substance-related impurity, N-nitroso-vonoprazan (NVP), vonoprazan was later approved for the healing of and the maintenance of healing of all grades of erosive GERD as well as the relief of heartburn associated with erosive GERD on November 1, 2023.^2,3 Now, less than a year later, it also boasts approval for heartburn associated with non-erosive GERD.¹

The decision is supported by positive phase 3 data from the PHALCON-NERD-301 study demonstrating control of heartburn symptoms with vonoprazan 10 mg and 20 mg through the entire 6 months of the study and with a safety profile consistent with prior vonoprazan studies. A total of 776 patients with symptomatic NERD were enrolled and randomized in the multisite US trial. Positive topline results showed both doses met the primary endpoint, demonstrating a significantly greater percentage of 24-hour heartburn-free days versus placebo (46.4% with vonoprazan 10 mg and 46.0% with vonoprazan 20 mg vs 27.5% with placebo; p<0.0001). Additionally, the median percentage of 24-hour heartburn-free days was greater with vonoprazan 10 mg (48.3%) and 20 mg (46.7%) compared with placebo (17.0%).¹

Phathom is also exploring a novel dosing regimen for vonoprazan as an on-demand or “as needed” treatment for episodic heartburn relief in patients with symptomatic NERD, a dosing treatment regimen not approved in the US for proton pump inhibitors.¹

References

1. ^{Phathom Pharmaceuticals, Inc. Phathom Pharmaceuticals announces FDA approval of VOQUEZNA® (vonoprazan) tablets for the relief of heartburn associated with non-erosive GERD in adults. Phathom Pharmaceuticals, Inc. Accessed July 18, 2024. https://investors.phathompharma.com/news-releases/news-release-details/phathom-pharmaceuticals-announces-fda-approval-voqueznar-0.}
2. ^{Phathom Pharmaceuticals. Phathom Pharmaceuticals Provides Update on New Drug Application Review of Vonoprazan for Erosive Esophagitis. January 3, 2023. Accessed July 18, 2024. https://investors.phathompharma.com/news-releases/news-release-details/phathom-pharmaceuticals-provides-update-new-drug-application}
3. ^{Phathom Pharmaceuticals. Phathom Pharmaceuticals Announces FDA Approval of VOQUEZNA® (vonoprazan) Tablets for the Treatment of Erosive GERD and Relief of Heartburn Associated with Erosive GERD in Adults. November 1, 2023. Accessed July 18 , 2024. https://investors.phathompharma.com/news-releases/news-release-details/phathom-pharmaceuticals-announces-fda-approval-voqueznar}