- FDA approval marks the first major innovation to the U.S. Erosive GERD market in over 30 years
- VOQUEZNA® met the primary endpoints and key secondary superiority endpoints in the pivotal Phase 3 PHALCON-EE trial evaluating VOQUEZNA in comparison to a standard-of-care proton pump inhibitor (PPI)1
- Commercial availability of VOQUEZNA expected December 2023
- Erosive GERD approval provides Phathom $175 million under its revenue interest financing agreement
- Conference call and webcast scheduled for November 2, 2023, at 11:00 a.m. ET
FLORHAM PARK, NJ, USA I November 01, 2023 I Phathom Pharmaceuticals, Inc. (Nasdaq: PHAT), a biopharmaceutical company focused on developing and commercializing novel treatments for gastrointestinal (GI) diseases, announced today the U.S. Food and Drug Administration (FDA) has approved VOQUEZNA® (vonoprazan) tablets 10 mg and 20 mg, a novel potassium-competitive acid blocker (PCAB), as a new treatment for adults for the healing of all grades of Erosive Esophagitis, also known as Erosive GERD (gastroesophageal reflux disease), maintenance of healing of all grades of Erosive GERD, and relief of heartburn associated with Erosive GERD.2
“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. “Erosive GERD can be extremely painful and often has a significant impact on patients. 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.”
Erosive GERD, also referred to as Erosive Esophagitis or Erosive Acid Reflux, is a major type of GERD that affects approximately 20 million people in the U.S.3,4 In addition to experiencing troubling heartburn symptoms, patients with inadequately treated Erosive GERD may develop more severe diseases including Barrett’s esophagus, a condition in which esophageal tissue changes can progress to cancer.3
This approval is based on positive results from the Phase 3 PHALCON-EE study (NCT04124926). The pivotal trial was a randomized, double-blind, multicenter study that enrolled 1,024 patients with Erosive GERD in the U.S. and Europe and compared VOQUEZNA to the PPI lansoprazole in the healing and maintenance of healing of Erosive GERD and associated heartburn symptom relief.1
Results showed that VOQUEZNA 20 mg met the primary endpoint of non-inferiority (p<0.0001) for complete healing by Week 8 in patients with all grades of Erosive GERD with a healing rate of 93% compared to 85% for lansoprazole 30 mg, with superior rates of healing demonstrated in a secondary endpoint in patients with moderate-to-severe disease (LA Grade C/D) at Week 2 compared to lansoprazole (70% for VOQUEZNA 20 mg and 53% for lansoprazole 30 mg) (p=0.0008). VOQUEZNA 20 mg also demonstrated non-inferiority to lansoprazole 30 mg in the mean percentage of 24-hour heartburn free days over the healing period. In the maintenance phase of the trial, VOQUEZNA 10 mg was superior to lansoprazole 15 mg in maintaining healing at six months in all randomized patients (79% for VOQUEZNA 10 mg, compared to 72% for lansoprazole 15 mg) as well as in the subset of patients with moderate-to-severe Erosive GERD (75% for VOQUEZNA 10 mg, compared to 61% for lansoprazole 15 mg) (p=0.0490). In addition, VOQUEZNA 10 mg was evaluated as a secondary endpoint for relief of heartburn in Erosive GERD patients and demonstrated non-inferiority to lansoprazole 15 mg over six months.
Adverse event (AE) rates for VOQUEZNA were comparable to lansoprazole in the trial. The most common AEs in the healing phase (≥ 2% in the VOQUEZNA treatment arm) were gastritis (3.0% for VOQUEZNA 20 mg and 2.0% for lansoprazole 30 mg), diarrhea (2.0% for VOQUEZNA 20 mg and 3.0% for lansoprazole 30 mg), abdominal distension (2.0% for VOQUEZNA 20 mg and 1.0 % for lansoprazole 30 mg), abdominal pain (2.0% for VOQUEZNA 20 mg and 1.0% for lansoprazole 30 mg) and nausea (2.0% for VOQUEZNA 20 mg and 1.0% for lansoprazole 30 mg). The most common AEs in the maintenance phase (≥ 3% in the VOQUEZNA treatment arm) for VOQUEZNA 10 mg compared to lansoprazole 15 mg were gastritis (6.0% vs. 3.0%), abdominal pain (4.0% vs. 2.0%), dyspepsia (4.0% vs. 3.0%), hypertension (3.0% vs. 2.0%), and urinary tract infection (3.0% vs. 2.0%). These are not all of the potential side effects associated with the use of VOQUEZNA. Please see Important Safety information below and the full Prescribing Information for VOQUEZNA for more information.
“For many GERD patients with Erosive Esophagitis, the response to current treatment is suboptimal, leaving them with incomplete healing and ongoing symptoms5,” said Colin W. Howden, MD, Professor Emeritus, University of Tennessee College of Medicine. “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. 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.”
VOQUEZNA is expected to be available in the U.S. in December 2023 and will be marketed exclusively by Phathom Pharmaceuticals, Inc.
Based on the terms of Phathom’s revenue interest financing agreement, the FDA approval of VOQUEZNA for Erosive GERD also entitles the company to receive a $175.0 million payment. This non-dilutive capital will help fund the commercial launch.
Conference Call and Webcast on November 2, 2023, at 11:00 a.m. ET
Phathom will host a live conference call and webcast on Thursday, November 2, 2023, at 11:00 a.m. ET to discuss the FDA approval and the Company’s U.S. commercialization plans for Erosive GERD and H. pylori infection. The conference call will be available via a listen-only webcast on the investor page of the Company’s website at https://investors.phathompharma.com/news-events/events-and-presentations.
About VOQUEZNA®
VOQUEZNA® tablets, VOQUEZNA® TRIPLE PAK® (vonoprazan, amoxicillin, clarithromycin), and VOQUEZNA® DUAL PAK® (vonoprazan, amoxicillin) 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, and VOQUEZNA is the first PCAB to be approved in the U.S. Vonoprazan has shown the potential to provide acid suppression that can achieve pH levels that are important in enhancing treatment effectiveness. Phathom in-licensed the U.S., European, and Canadian rights to vonoprazan from Takeda, which markets the product in Japan and numerous other countries in Asia and Latin America.
About Erosive GERD
Erosive GERD is a major type of gastroesophageal reflux disease (GERD) characterized by erosions in the gastric mucosa caused by acidic reflux of stomach contents into the esophagus.3 There are estimated to be over 65 million individuals with GERD in the U.S., of which approximately 30% have Erosive GERD.4,6,7 In addition to experiencing troubling heartburn symptoms, patients with inadequately treated Erosive GERD may progress to more severe diseases including Barrett’s esophagus and esophageal cancer.4
About Phathom Pharmaceuticals, Inc.
Phathom Pharmaceuticals is a biopharmaceutical company focused on the development and commercialization of novel treatments for gastrointestinal diseases. Phathom has in-licensed the exclusive rights in the United States, Europe, and Canada to vonoprazan, a first-in-class potassium-competitive acid blocker (PCAB). For more information about Phathom, visit the Company’s website at www.phathompharma.com and follow the Company on LinkedIn and Twitter.
Please see Patient Information and full Prescribing Information for VOQUEZNA.
Visit www.voquezna.com to learn more about VOQUEZNA.
INDICATION AND IMPORTANT SAFETY INFORMATION
What is VOQUEZNA?
VOQUEZNA® (vonoprazan) is a prescription medicine used in adults:
- for 8 weeks to heal acid-related damage to the lining of the esophagus (called Erosive Esophagitis or Erosive Acid Reflux) and for relief of heartburn related to Erosive Acid Reflux.
- for up to 6 months to maintain healing of Erosive Acid Reflux and for relief of heartburn related to Erosive Acid Reflux.
It is not known if VOQUEZNA is safe and effective in children.
REFERENCES
- Laine L, DeVault K, Katz P, et al. Vonoprazan Verses Lansoprazole for Healing and Maintenance of Healing of Erosive Esophagitis: A Randomized Trial. Gastroenterology. October 10, 2022. https://doi.org/10.1053/j.gastro.2022.09.041
- VOQUEZNA Prescribing Information, Phathom Pharmaceuticals, 2023.
- Dickman R, Maradey-Romero C, Gingold-Belfer R, Fass R. Unmet Needs in the Treatment of Gastroesophageal Reflux Disease. J Neurogastroenterol Motil. 2015 Jul 30;21(3):309-19. doi: 10.5056/jnm15105.
- Savarino E, de Bortoli N, De Cassan C, et al. The natural history of gastro-esophageal reflux disease: A comprehensive review. Dis Esophagus. 2017;30(2):1-9.
- Vaezi MF, Brunton S, Mark Fendrick A, et al. Patient journey in erosive oesophagitis: real-world perspectives from US physicians and patients. BMJ Open Gastroenterology. 2022;9:e000941. doi: 10.1136/bmjgast-2022-000941
- Machicado J.D., Greer J.B., Yadav D. (2020) Epidemiology of Gastrointestinal Diseases. In: Pitchumoni C., Dharmarajan T. (eds) Geriatric Gastroenterology. Springer, Cham. https://doi.org/10.1007/978-3-319-90761-1_7-1.
- U.S. Census Bureau. U.S. and World Population Clock. Accessed May 2022. https://www.census.gov/popclock.
SOURCE: Phathom Pharmaceuticals
Post Views: 153
- FDA approval marks the first major innovation to the U.S. Erosive GERD market in over 30 years
- VOQUEZNA® met the primary endpoints and key secondary superiority endpoints in the pivotal Phase 3 PHALCON-EE trial evaluating VOQUEZNA in comparison to a standard-of-care proton pump inhibitor (PPI)1
- Commercial availability of VOQUEZNA expected December 2023
- Erosive GERD approval provides Phathom $175 million under its revenue interest financing agreement
- Conference call and webcast scheduled for November 2, 2023, at 11:00 a.m. ET
FLORHAM PARK, NJ, USA I November 01, 2023 I Phathom Pharmaceuticals, Inc. (Nasdaq: PHAT), a biopharmaceutical company focused on developing and commercializing novel treatments for gastrointestinal (GI) diseases, announced today the U.S. Food and Drug Administration (FDA) has approved VOQUEZNA® (vonoprazan) tablets 10 mg and 20 mg, a novel potassium-competitive acid blocker (PCAB), as a new treatment for adults for the healing of all grades of Erosive Esophagitis, also known as Erosive GERD (gastroesophageal reflux disease), maintenance of healing of all grades of Erosive GERD, and relief of heartburn associated with Erosive GERD.2
“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. “Erosive GERD can be extremely painful and often has a significant impact on patients. 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.”
Erosive GERD, also referred to as Erosive Esophagitis or Erosive Acid Reflux, is a major type of GERD that affects approximately 20 million people in the U.S.3,4 In addition to experiencing troubling heartburn symptoms, patients with inadequately treated Erosive GERD may develop more severe diseases including Barrett’s esophagus, a condition in which esophageal tissue changes can progress to cancer.3
This approval is based on positive results from the Phase 3 PHALCON-EE study (NCT04124926). The pivotal trial was a randomized, double-blind, multicenter study that enrolled 1,024 patients with Erosive GERD in the U.S. and Europe and compared VOQUEZNA to the PPI lansoprazole in the healing and maintenance of healing of Erosive GERD and associated heartburn symptom relief.1
Results showed that VOQUEZNA 20 mg met the primary endpoint of non-inferiority (p<0.0001) for complete healing by Week 8 in patients with all grades of Erosive GERD with a healing rate of 93% compared to 85% for lansoprazole 30 mg, with superior rates of healing demonstrated in a secondary endpoint in patients with moderate-to-severe disease (LA Grade C/D) at Week 2 compared to lansoprazole (70% for VOQUEZNA 20 mg and 53% for lansoprazole 30 mg) (p=0.0008). VOQUEZNA 20 mg also demonstrated non-inferiority to lansoprazole 30 mg in the mean percentage of 24-hour heartburn free days over the healing period. In the maintenance phase of the trial, VOQUEZNA 10 mg was superior to lansoprazole 15 mg in maintaining healing at six months in all randomized patients (79% for VOQUEZNA 10 mg, compared to 72% for lansoprazole 15 mg) as well as in the subset of patients with moderate-to-severe Erosive GERD (75% for VOQUEZNA 10 mg, compared to 61% for lansoprazole 15 mg) (p=0.0490). In addition, VOQUEZNA 10 mg was evaluated as a secondary endpoint for relief of heartburn in Erosive GERD patients and demonstrated non-inferiority to lansoprazole 15 mg over six months.
Adverse event (AE) rates for VOQUEZNA were comparable to lansoprazole in the trial. The most common AEs in the healing phase (≥ 2% in the VOQUEZNA treatment arm) were gastritis (3.0% for VOQUEZNA 20 mg and 2.0% for lansoprazole 30 mg), diarrhea (2.0% for VOQUEZNA 20 mg and 3.0% for lansoprazole 30 mg), abdominal distension (2.0% for VOQUEZNA 20 mg and 1.0 % for lansoprazole 30 mg), abdominal pain (2.0% for VOQUEZNA 20 mg and 1.0% for lansoprazole 30 mg) and nausea (2.0% for VOQUEZNA 20 mg and 1.0% for lansoprazole 30 mg). The most common AEs in the maintenance phase (≥ 3% in the VOQUEZNA treatment arm) for VOQUEZNA 10 mg compared to lansoprazole 15 mg were gastritis (6.0% vs. 3.0%), abdominal pain (4.0% vs. 2.0%), dyspepsia (4.0% vs. 3.0%), hypertension (3.0% vs. 2.0%), and urinary tract infection (3.0% vs. 2.0%). These are not all of the potential side effects associated with the use of VOQUEZNA. Please see Important Safety information below and the full Prescribing Information for VOQUEZNA for more information.
“For many GERD patients with Erosive Esophagitis, the response to current treatment is suboptimal, leaving them with incomplete healing and ongoing symptoms5,” said Colin W. Howden, MD, Professor Emeritus, University of Tennessee College of Medicine. “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. 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.”
VOQUEZNA is expected to be available in the U.S. in December 2023 and will be marketed exclusively by Phathom Pharmaceuticals, Inc.
Based on the terms of Phathom’s revenue interest financing agreement, the FDA approval of VOQUEZNA for Erosive GERD also entitles the company to receive a $175.0 million payment. This non-dilutive capital will help fund the commercial launch.
Conference Call and Webcast on November 2, 2023, at 11:00 a.m. ET
Phathom will host a live conference call and webcast on Thursday, November 2, 2023, at 11:00 a.m. ET to discuss the FDA approval and the Company’s U.S. commercialization plans for Erosive GERD and H. pylori infection. The conference call will be available via a listen-only webcast on the investor page of the Company’s website at https://investors.phathompharma.com/news-events/events-and-presentations.
About VOQUEZNA®
VOQUEZNA® tablets, VOQUEZNA® TRIPLE PAK® (vonoprazan, amoxicillin, clarithromycin), and VOQUEZNA® DUAL PAK® (vonoprazan, amoxicillin) 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, and VOQUEZNA is the first PCAB to be approved in the U.S. Vonoprazan has shown the potential to provide acid suppression that can achieve pH levels that are important in enhancing treatment effectiveness. Phathom in-licensed the U.S., European, and Canadian rights to vonoprazan from Takeda, which markets the product in Japan and numerous other countries in Asia and Latin America.
About Erosive GERD
Erosive GERD is a major type of gastroesophageal reflux disease (GERD) characterized by erosions in the gastric mucosa caused by acidic reflux of stomach contents into the esophagus.3 There are estimated to be over 65 million individuals with GERD in the U.S., of which approximately 30% have Erosive GERD.4,6,7 In addition to experiencing troubling heartburn symptoms, patients with inadequately treated Erosive GERD may progress to more severe diseases including Barrett’s esophagus and esophageal cancer.4
About Phathom Pharmaceuticals, Inc.
Phathom Pharmaceuticals is a biopharmaceutical company focused on the development and commercialization of novel treatments for gastrointestinal diseases. Phathom has in-licensed the exclusive rights in the United States, Europe, and Canada to vonoprazan, a first-in-class potassium-competitive acid blocker (PCAB). For more information about Phathom, visit the Company’s website at www.phathompharma.com and follow the Company on LinkedIn and Twitter.
Please see Patient Information and full Prescribing Information for VOQUEZNA.
Visit www.voquezna.com to learn more about VOQUEZNA.
INDICATION AND IMPORTANT SAFETY INFORMATION
What is VOQUEZNA?
VOQUEZNA® (vonoprazan) is a prescription medicine used in adults:
- for 8 weeks to heal acid-related damage to the lining of the esophagus (called Erosive Esophagitis or Erosive Acid Reflux) and for relief of heartburn related to Erosive Acid Reflux.
- for up to 6 months to maintain healing of Erosive Acid Reflux and for relief of heartburn related to Erosive Acid Reflux.
It is not known if VOQUEZNA is safe and effective in children.
REFERENCES
- Laine L, DeVault K, Katz P, et al. Vonoprazan Verses Lansoprazole for Healing and Maintenance of Healing of Erosive Esophagitis: A Randomized Trial. Gastroenterology. October 10, 2022. https://doi.org/10.1053/j.gastro.2022.09.041
- VOQUEZNA Prescribing Information, Phathom Pharmaceuticals, 2023.
- Dickman R, Maradey-Romero C, Gingold-Belfer R, Fass R. Unmet Needs in the Treatment of Gastroesophageal Reflux Disease. J Neurogastroenterol Motil. 2015 Jul 30;21(3):309-19. doi: 10.5056/jnm15105.
- Savarino E, de Bortoli N, De Cassan C, et al. The natural history of gastro-esophageal reflux disease: A comprehensive review. Dis Esophagus. 2017;30(2):1-9.
- Vaezi MF, Brunton S, Mark Fendrick A, et al. Patient journey in erosive oesophagitis: real-world perspectives from US physicians and patients. BMJ Open Gastroenterology. 2022;9:e000941. doi: 10.1136/bmjgast-2022-000941
- Machicado J.D., Greer J.B., Yadav D. (2020) Epidemiology of Gastrointestinal Diseases. In: Pitchumoni C., Dharmarajan T. (eds) Geriatric Gastroenterology. Springer, Cham. https://doi.org/10.1007/978-3-319-90761-1_7-1.
- U.S. Census Bureau. U.S. and World Population Clock. Accessed May 2022. https://www.census.gov/popclock.
SOURCE: Phathom Pharmaceuticals
Post Views: 153
