Helicobacter pylori illustration.
Last month, the Food and Drug Administration (FDA) approved Voquenza Dual (vonoprazan fumarate + amoxicillin) and Triple Paks (vonoprazan fumarate + amoxicillin + clarithromycin) for the treatment of Helicobacter pylori infection in adults. Vonoprazan fumarate is the first in its class potassium competitive acid blocker. Voquenza will launch later this year.
In March, the company launching Voquenza, Phathom Pharmaceuticals, also submitted a new drug application to the FDA for the use of vonoprazan as a treatment for the healing of all grades of erosive reflux esophagitis and relief of heartburn.
Helicobacter pylori bacteria reside in the stomach and small intestines of at least 30% of the population. In most people, the bacteria do not cause symptoms. However, some experience gastrointestinal symptoms caused by Helicobacter pylori, which run the gamut from frequent stomach upset to ulcers to cancer in rare cases. For patients experiencing distress, eradication of Helicobacter pylori can be an effective remedy.
Regarding the first FDA-approved indication a vonoprazan-based regimen has been found to be superior to existing standard of care, which is a proton pump inhibitor (PPI)–based triple therapy; usually, a PPI together with two antibiotics and sometimes bismuth subsalicylate. In the treatment of Helicobacter pylori infection, combination therapies using the oral acid blocker vonoprazan produced higher eradication rates, according to combined data from a U.S. and a European phase 3 randomized, controlled trial. Also, treatment with vonoprazan is considered less complex compared to the current PPI-based triple therapy, and as such may improve patient adherence rates.
Moving forward, the next indication(s), (non)erosive reflux esophagitis present in some patients suffering from gastroesophageal reflux disease (GERD), may prove to be an even bigger prize for Phathom Pharmaceuticals.
GERD is a highly prevalent digestive disorder that negatively affects patients' quality of life. Given the large numbers of GERD sufferers, the healthcare expenses associated with the treatment of GERD are the highest among all digestive diseases. One of many complications associated with GERD is (non)erosive reflux esophagitis.
Current clinical practice guidelines recommend the administration of a standard dose of proton pump inhibitor (PPI) for eight weeks as an initial GERD treatment. However, there is some concern regarding the safety of PPI treatment, as well as the fact that a subset of patients are refractory.
Vonoprazan was approved for GERD in Japan in 2014, and has been used extensively in a large number of GERD patients. In Japan, researchers have carried out multiple comparative cost-effectiveness analyses that showed vonoprazan to be superior to the standard PPI strategy. Moreover, fewer days of medication were required.
Specifically, Japanese cost-effectiveness studies examined vonoprazan in a head-to-head analysis against lansoprazole. Here, the cost figures were predicated on vonoprazan being priced at ~$50 for 20 mg/day x 4 weeks versus lansoprazole 30 mg/day x 4 weeks* at ~$31.
This seems like a relatively small price premium for a new branded product in the U.S. Accordingly, questions remain with respect to pricing in the U.S. and how this could impact cost-effectiveness numbers.
But, in a therapeutic area in which there is at least some unmet need and there hasn’t been a new treatment in more than two decades, vonoprazan may be an important additional treatment in the gastroenterologist’s toolkit.
