FDA Approves New GERD Treatment Vonoprazan for Heartburn Relief

• The FDA approved vonoprazan (Voquezna) as a daily treatment for heartburn associated with non-erosive gastroesophageal reflux disease (GERD) in adults.
• GERD is a condition in which acidic stomach contents flow backward into the esophagus. In non-erosive GERD, the esophagus is not damaged by stomach acid.
• This approval provides an alternative treatment to proton pump inhibitors such as Prevacid, Prilosec, and Nexium.

Adults with a certain type of heartburn now have another option for finding relief from their condition.

On July 18, the Food and Drug Administration approved Phathom Pharmaceuticals’ medication vonoprazan (Voquezna) as a daily treatment for heartburn associated with non-erosive gastroesophageal reflux disease (GERD) in adults.

GERD is a condition in which acidic stomach contents flow backward into the esophagus. In non-erosive GERD — also known as NERD — the esophagus is not damaged by the stomach acid. However, it can cause symptoms such as heartburn.

An estimated 45 million American adults have non-erosive GERD, according to Phathom Pharmaceuticals’ release .

The FDA’s decision is based on a phase 3 clinical trial that evaluated the efficacy and safety of Voquezna as a treatment for this condition.

The trial found that Voquezna significantly reduced heartburn episodes in adults with non-erosive GERD, with “an established safety profile,” Colin W. Howden, MD, professor emeritus at the University of Tennessee College of Medicine, said in the release.

“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,” he added.

Effectiveness and safety of Voquezna

Voquezna, which is taken by mouth, is a type of drug known as a small-molecule potassium-competitive acid blocker (PCAB). This group of medicines blocks the secretion of acid in the stomach.

The drug is also approved in the United States to treat erosive esophagitis (known as erosive GERD), in which the stomach acid damages the lining of the esophagus, and for treating Helicobacter pylori infection, in combination with antibiotics.

The four-week clinical trial for Voquezna included more than 700 adults with non-erosive GERD who experienced heartburn at least four days per week. Participants were randomly assigned to take Voquezna 10 mg, Voquezna 20 mg, or a non-acting placebo.

The company said in its release that, on average, people taking Voquezna had a greater percentage of heartburn-free days (45%) than those taking the placebo (28%).

In addition, the average percentage of 24-hour heartburn-free days was higher in the Voquezna group (48%) than in the placebo group (17%).

The most common adverse reactions reported by patients taking Voquezna included abdominal pain, constipation, diarrhea, nausea, and urinary tract infection.

Some patients who took Voquezna during a 20-week extension phase of the trial also reported upper respiratory tract infection and sinusitis.

Results from the study were published May 13 in Clinical Gastroenterology and Hepatology .

Patient information and full prescribing information for Voquezna are available.

How Voquezna compares to proton pump inhibitors

With the FDA approval of Voquezna, “we now have a new medication to treat patients with non-erosive GERD,” said Rudolph Bedford, MD , a gastroenterologist at Providence Saint John’s Health Center in Santa Monica, Calif. “This also allays many of the concerns that people have about being on long-term proton pump inhibitors.”

Proton pump inhibitors (PPIs) relieve symptoms of GERD by reducing the production of acid in the stomach. They include lansoprazole (Prevacid 24 HR), omeprazole (Prilosec) and esomeprazole (Nexium).

Long-term use of proton pump inhibitors may increase the risk of bone fracture, kidney disease , or a deficiency of certain nutrients such as magnesium and vitamin B12.

While Voquezna works differently from proton pump inhibitors, it does reduce stomach acid. Because of this, the prescribing information for Voquezna warns of the possibility of similar long-term side effects as proton pump inhibitors.

Ashkan Farhadi, MD , a gastroenterologist at MemorialCare Orange Coast Medical Center in Fountain Valley, Calif., pointed out that most of the side effects of proton pump inhibitors appear with long-term use.

“So I would not be surprised if the side effects of [Voquezna] surface many years from now,” he told Healthline. “So that is something to be concerned about.”

“While this [approval] gives me one more tool in my toolbox for managing this disease — which is good — I encourage people to be cautious about it since it is a new medication,” Farhadi said.

However, Bedford said vonoprazan “has been around for 5 to 10 years now [for other uses], so I think that’s adequate time for us to know if there would be some long-term issues.”

Overall, Bedford thinks Voquezna will improve the quality of life of people who respond well to the drug, including those who don’t benefit from proton pump inhibitors.

Farhadi said one advantage of Voquezna is that it “starts faster and lasts longer” than proton pump inhibitors. This may allow people to take the drug on an as-needed basis rather than daily.

“Let’s say you’re about to go out and have a big dinner, and you know that would bring on discomfort,” Bedford told Healthline. “You might want to take one of these pills an hour or two before to prevent that from occurring.”

“While it doesn’t provide immediate relief, typically the relief from this medication is within one day,” he said. “I’m not sure that can be said for proton pump inhibitors.”

Takeaway

The Food and Drug Administration approved Phathom Pharmaceuticals’ medication vonoprazan (Voquezna) as a daily treatment for heartburn associated with non-erosive gastroesophageal reflux disease (GERD) in adults.

GERD is a condition in which acidic stomach contents flow backward into the esophagus. In non-erosive GERD, the lining of the esophagus is not damaged by the stomach acid, but heartburn can still occur.

Voquezna increased the number of heartburn-free days in patients compared with a non-acting placebo. The main side effects of Voquezna were abdominal pain, constipation, diarrhea, nausea, and urinary tract infection.