FDA Approves Vonoprazan for the Treatment of Erosive Esophagitis

The FDA has approved vonoprazan (Voquenza; Phathom Pharmaceuticals) to treat adults with the gastroesophageal reflux disease erosive esophagitis (erosive GERD). Phathom Pharmaceuticals announced that it anticipates the potassium-competitive acid blocker to be commercially available by December 2023.

“This approval demonstrates Phathom's commitment to changing the GI treatment landscape for patients and healthcare providers, bringing the first major innovation to the U.S. Erosive GERD market in over 30 years,” said Terrie Curran, president and chief executive officer at Phathom, in a press release. “Research has shown patients and healthcare 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.”

The FDA approved vonoprazan tablets at doses of 10 mg and 20 mg to treat all grades of erosive GERD and to relieve associated heartburn. Currently, more than 20 million patients are afflicted with erosive esophagitis.

The FDA action carries particular significance because it is the first major innovation for the condition in the past three decades, according to Phathom Pharmaceuticals. In clinical trials, vonoprazan was found to produce rapid, powerful, and strong acid suppression. Besides the pain of heartburn, patients with erosive GERD who lack adequate treatment may develop go on to Barrett’s esophagus, which can ultimately lead to cancer.

The approval was based on positive data from the randomized-double-blind, multicenter, phase 3 PHALCON-EE study, which enrolled 1024 patients with erosive GERD in the United States and Europe. The study evaluated vonoprazan to the proton pump inhibitor (PPI) lansoprazole in its ability to heal the symptoms of erosive GERD or to treat associated heartburn.

The 20 mg strength of vonoprazan achieved the primary endpoint of non-inferiority (P< .0001) for full healing by week 8 in patients with all grades of erosive GERD. Further, there was a healing rate of 93%, which was higher than the 85% for the 30 mg dose of lansoprazole.

Vonoprazan 20 mg also showed non-inferiority to lansoprazole 30 mg during 24-hour heartburn-free days, according to the study. For the maintenance phase of the trial, vonoprazan 10 mg was found to be superior to lansoprazole 15 mg in consistent healing at 6 months.

In the healing phase, the adverse events (AEs) for vonoprazan and lansoprazole were similar, with AEs that included gastritis, diarrhea, abdominal distension, abdominal pain, and nausea.

Comparable AEs for the maintenance phase of vonoprazan and lansoprazole included abdominal pain, dyspepsia, hypertension, and urinary tract infection.

"The FDA approval of [vonoprazan] provides healthcare providers with a new first-in-class therapeutic option that demonstrated faster healing in the more difficult to treat GERD patients with erosive esophagitis,” said Colin W. Howden, MD, professor emeritus of University of Tennessee College of Medicine, in the press release. “In addition, [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.”

Reference

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