FDA Approves Vonoprazan to treat Erosive Esophagitis

The U.S. Food and Drug Administration (FDA) approved Phathom Pharmaceutical’s vonoprazan (Voquenza) on November 1, 2023 for the treatment of adults with erosive esophagitis (Erosive GERD), a gastroesophageal reflux disease. The drug is expected to be commercially available by December 2023.

The drug approval marks a critical moment for over 20 million patients who suffer from the condition since it is the first major innovation in the U.S. Erosive GERD space in the past 30 years. The drug offers quick, powerful, and strong acid suppression. Other than dealing with heartburn, patients with Erosive GERD who are inadequately treated may develop Barrett’s esophagus—a condition which can lead to cancer.

The FDA approved tablets 10 mg and 20 mg of VOQUENZ®, a potassium-competitve acid blocker, to heal all grades of EROSIVE GERD and to relieve associated heartburn.

“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 approval was based on positive results from the Phase 3 PHALCON-EE study, a randomized-double-blind, multicenter study with 1024 patients with Erosive GERD in the U.S. and Europe. The study compared VOQUENZA to the PPI lansoprazole in terms of healing symptoms of Erosive GERD or treating associated heartburn. VOQUENZA at 20 mg met the primary endpoint of non-inferiority (P < .0001) for full healing by week 8 in patients with all grades of Erosive GERD. There was a healing rate of 93%, which was higher than the 85% for the 30 mg of lansoprazole.

VOQUENZA 20 mg also displayed non-inferiority to lansoprazole 30 mg of during the 24-hour heartburn free days. As for the maintenance phase of the trial, VOQUENZA 10 mg was superior to lansoprazole 15 mg in consistent healing at 6 months.

Adverse events for VOQEUNZA and Lansoprazole were comparable in the healing phase, with some of them being gastritis, diarrhea, abdominal distension, abdominal pain, and nausea. For the maintenance phase, comparable adverse events between VOQEUNZA and Lansoprazole were abdominal pain, dyspepsia, hypertension, and urinary tract infection.

"The FDA approval of VOQUEZNA (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, 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.”