The approval marks the first major innovation in the United States for the treatment of erosive GERD in over 30 years.
The FDA has approved vonoprazan (Voquezna, Phatom Pharmaceuticals, Inc.) to treat adults with all grades of erosive esophagitis, or erosive gastroesophageal reflux disease (GERD)—marking the first major innovation in the United States in over 30 years to treat erosive GERD.
“Erosive GERD can be extremely painful and often has a significant impact on patients," said Terrie Curran, President and Chief Executive Officer at Phathom, in a press release. "Research has shown patients and health care providers are largely unsatisfied with current treatments and we are excited about the approval of a first-in-class treatment option that has the potential to meet a large unmet medical need.”
Erosive GERD, also known as erosive esophagitis or erosive acid reflux, is defined as an extensive type of GERD that could lead to the development of other conditions if not properly treated. One of the conditions include Barrett’s esophagus, in which esophageal tissue changes and could lead to cancer. Researchers reported that over 65 million individuals in the United States are living with GERD and 30% are diagnosed with erosive GERD. Other symptoms that individuals could experience include troubling heartburn.
Vonoprazan is an oral tablet that is a molecule potassium-competitive acid blocker that can block acid secretion in the stomach. This tablet targets GERD with its ability to “provide acid suppression that can achieve pH levels that are important in enhancing treatment effectiveness.”
The approval of vonoprazan 10 mg and 20 mg was based on the phase 3 PHALCON-EE study (NCT04124926). The study was a randomized, double-blind, multicenter trial that assessed the use of vonoprazan to heal erosive GERD and heartburn relief, compared to the proton pump inhibitor (PPI) lansoprazole.
The researchers included 1024 individuals in the study who had erosive GERD from the United States and Europe.
The primary endpoint for vonoprazan 20 mg was to display non-inferiority for complete healing by week 8 of treatment for individuals with erosive GERD. The researchers noted that the primary endpoint was met and individuals achieved a 93% healing rate on vonoprazan, compared to a 85% healing rate for lansoprazole 30 mg. Vonoprazan also met its secondary endpoint of demonstrating greater rates of healing in individuals with moderate-to-severe disease at week 2, compared to lansoprazole.
Vonoprazan 10 mg had similar results and provided heartburn relief and non-inferiority compared to individuals treated with lansoprazole 15 mg. The 10 mg dosage also achieved 79% of maintaining healing at 6 months in randomly assigned patients, compared to 72% of healing in individuals taking lansoprazole 15 mg.
Adverse events (AEs) with vonoprazan and lansoprazole were compared throughout the study within the healing and maintenance phase. AEs throughout the healing phase included gastritis, diarrhea, abdominal distension, abdominal pain, and nausea. AEs throughout the maintenance phase included gastritis, abdominal pain, dyspepsia, hypertension, and urinary tract infections, However, the researchers noted that not all of the adverse effects are connected to treatment with vonoprazan.
Vonoprazan is expected to be available in December of 2023.
“For many GERD patients with erosive esophagitis, the response to current treatment is suboptimal, leaving them with incomplete healing and ongoing symptoms,” said Colin W. Howden, MD, Professor Emeritus, University of Tennessee College of Medicine, in a press release. “The FDA approval of Voquezna (vonoprazan) provides health care providers with a new first-in-class therapeutic option that demonstrated faster healing in the more difficult to treat GERD patients with erosive esophagitis. In addition, Voquezna (vonoprazan) provided superior maintenance of healing in all grades of erosive esophagitis, compared to lansoprazole, a commonly prescribed PPI, and provided 24-hour heartburn relief on most days in the trial.”
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. Phathom Pharmaceuticals. News release. November 1, 2023. Accessed November 2, 2023. https://investors.phathompharma.com/news-releases/news-release-details/phathom-pharmaceuticals-announces-fda-approval-voqueznar.