The NDA submission was supported by positive data from the phase 3 PHALCON-NERD-301 study and has been assigned a standard 10-month review with a PDUFA target action date of July 19, 2024.
The US Food and Drug Administration (FDA) has accepted Phathom Pharmaceuticals’ New Drug Application (NDA) for vonoprazan (Voquenza) as a daily treatment of heartburn associated with Non-Erosive gastroesophageal reflux disease (GERD) in adults.
Announced on December 6, 2023, the FDA has assigned the application a standard 10-month review with a Prescription Drug User Fee Act (PDUFA) target action date of July 19, 2024.1
"Today marks an important step forward for Phathom Pharmaceuticals as we announce the FDA's acceptance for filing of our New Drug Application for VOQUEZNA as a daily treatment of heartburn for patients with Non-Erosive GERD,” said Azmi Nabulsi, MD, chief operating officer at Phathom.1 “Non-Erosive GERD represents a substantial segment of the U.S. GERD population, affecting millions of individuals and impacting their quality of life. The supportive data in our regulatory application include the positive Phase 3 results from the investigational PHALCON-NERD-301 trial, along with a safety profile consistent with prior studies.”
An investigational potassium-competitive acid blocker, vonoprazan is indicated for the healing and maintenance of healing of all grades of Erosive GERD as well as the relief of heartburn associated with Erosive GERD in adults. Phathom announced the submission of an NDA to the FDA for vonoprazan on September 26, 2023, supported by positive data from the phase 3 PHALCON-NERD-301 study evaluating the efficacy and safety of vonoprazan for the daily treatment of adults with Non-Erosive GERD.1,2
Topline results from the 4-week, double-blind, placebo-controlled period of PHALCON-NERD-301 were announced on January 8, 2023, and showed vonoprazan 10 mg and 20 mg met the study’s primary endpoint for efficacy as a daily dosing treatment compared to placebo for the relief of heartburn in participants with symptomatic Non-Erosive GERD. The trial also included a blinded 20-week long-term extension period to further evaluate the safety and efficacy of both doses of vonoprazan after 6 months of continuous use.3
Data showed treatment with vonoprazan resulted in a significantly greater percentage of 24-hour heartburn-free days compared to placebo (mean 46.4% vonoprazan 10 mg, 46.0% vonoprazan 20 mg, compared to 27.5% for placebo; P < .0001). The median percentage of 24-hour heartburn-free days was 48.3% for vonoprazan 10 mg, 46.7% for vonoprazan 20 mg, and 17.0% for placebo.3
Vonoprazan was generally well tolerated in the initial 4-week double-blind, placebo-controlled phase of the trial. The most commonly reported adverse event was nausea (2.3% vonoprazan 10 mg, 3.1% vonoprazan 20 mg, 0.4% placebo) with no other events reported above 3.0% in either vonoprazan dose arm.3
According to the December 6 release, Phathom is also finalizing its plans to initiate an additional Phase 3 study in 2024 evaluating VOQUEZNA as an investigational As Needed treatment for episodic heartburn relief in adults with Non-Erosive GERD.1
“We look forward to working closely with the FDA throughout the review process and if approved, expect to launch VOQUEZNA for this new indication in the third quarter of 2024,” Nabulsi said.1