Phathom Pharmaceuticals receives FDA approval for Voquezna to treat non-erosive GERD

19-Jul-2024 - Last updated on 21-Jul-2024 at 11:35 GMT

Phathom Pharmaceuticals, Inc.dedicated to developing and commercializing treatments for gastrointestinal (GI) diseases, announced today that the US Food and Drug Administration (FDA) has approved Voquezna (vonoprazan) 10mg tablets for the relief of heartburn associated with non-erosive gastroesophageal reflux disease (non-erosive GERD) in adults.

This marks the third FDA approval for Voquenza, which is also approved for treating all severities of Erosive Esophagitis (EE), also referred to as Erosive GERD, and in combination with antibiotics for eradicating Helicobacter pylori (H. pylori) infection.

A significant milestone for GERD patients

Terrie Curran, president and CEO of Phathom Pharmaceuticals, a biopharmaceutical company, highlighted the importance of this approval: “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. 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: A major health concern

Non-erosive GERD is the largest category of GERD, characterized by reflux-related symptoms in the absence of esophageal mucosal erosions. An estimated 45 million US adults live with non-erosive GERD, with approximately 15 million treated with prescription medicine annually. Despite existing treatment options, many patients remain dissatisfied and continue to experience symptoms impacting their quality of life.

Dr Colin Howden, professor emeritus at the University of Tennessee College of Medicine, emphasized the need for new treatments, he said: “Millions of patients with Non-Erosive GERD continue to suffer from heartburn despite current treatment options. 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."

Clinical evidence supporting Voquenza

The FDA approval is based on positive results from the Phalcon-Nerd-301 study (NCT05195528), a Phase 3 randomized, placebo-controlled, double-blind, multi-site US trial. The study evaluated the efficacy and safety of Voquezna for daily treatment in adults with non-erosive GERD. The trial enrolled 772 adult patients who experienced heartburn four or more days per week, with the majority suffering six to seven days per week. Results showed that Voquezna significantly reduced heartburn episodes and provided more complete 24-hour heartburn-free days and nights compared to placebo.

In the pivotal trial, the mean percentage of heartburn-free days for patients taking Voquezna was 45% versus 28% for placebo, and the median percentage of 24-hour heartburn-free days was 48% versus 17%, respectively. The trial also included a 20-week extension period where all patients received Voquezna to evaluate long-term treatment.

Tablets contain small molecule PCAB

The most common adverse reactions reported during the four-week placebo-controlled trial included abdominal pain, constipation, diarrhea, nausea, and urinary tract infection. During the 20-week extension phase, upper respiratory tract infection and sinusitis were also noted.

Voquezna tablets contain vonoprazan, an oral small molecule potassium-competitive acid blocker (PCAB). PCABs are a novel class of medicines that block acid secretion in the stomach. Voquezna is approved in the US for treating adults with erosive esophagitis, the relief of heartburn associated with erosive GERD, non-erosive GERD, and for treating H. pylori infection in combination with antibiotics. Phathom in-licensed the U.S. rights to vonoprazan from Takeda, which markets the product in Japan and several other countries in Asia and Latin America.

The approval of Voquezna for non-erosive GERD represents a significant advancement in the treatment of GERD. By providing a novel, effective therapy, Phathom Pharmaceuticals aims to address the unmet needs of millions of patients, enhancing their quality of life and setting a new standard in GERD management.