U.S. Food and Drug Administration Accepts New Drug Application for Review, Grants Priority Review for Takeda�s TAK-721 (budesonide oral suspension) for the Treatment of Eosinophilic Esophagitis

? If approved, TAK-721 will be the first FDA-approved treatment for the chronic inflammatory disease; Takeda plans to use the trade name Eohilia (budesonide oral suspension)

OSAKA, Japan–(BUSINESS WIRE)–Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) (�Takeda”) today announced that the United States (U.S.) Food and Drug Administration (FDA) has accepted for review the company’s New Drug Application (NDA) and granted Priority Review for the investigational therapy budesonide oral suspension, TAK-721, which has been designed specifically for eosinophilic esophagitis (EoE).¹ If approved, TAK-721 will be the first FDA-approved treatment for the chronic inflammatory disease, and Takeda plans to use the trade name Eohilia (budesonide oral suspension). The FDA grants Priority Review for medicines that, if approved, have the potential to offer significant advances in the safety or effectiveness of the current treatment approaches. TAK-721 previously received both Breakthrough Therapy designation and Orphan Drug designation from the FDA.

“For too long, healthcare professionals have been without approved treatments developed specifically to target the chronic, localized esophageal inflammation of EoE,” said Asit Parikh, MD, PhD, Head, Gastroenterology Therapeutic Area Unit, Takeda. “We are enthusiastic about the pivotal Phase 3 clinical data showing the ability of TAK-721 to help address esophageal inflammation. We’re committed to working closely with the FDA to further our collective understanding of EoE, and potentially bringing a new treatment option to patients and their healthcare professionals.”

EoE is a chronic inflammatory disease that can cause damage to the esophagus.² Impacting approximately one in 2,000 people in the U.S.,^2,3,4,5,6 EoE is growing in prevalence.⁷ Though the exact cause is unknown, EoE involves a complex interplay of genetics, environmental factors and immune system dysfunction.⁸ The chronic inflammation of EoE can lead to a range of symptoms, which can vary by person and age,⁹ but most often results in difficulty swallowing.¹⁰ If left untreated, EoE can worsen over time and cause injury and inflammation to the esophagus.¹¹ In severe cases, EoE can lead to narrowing of the esophagus, which may cause food to become stuck.¹⁰

“Because food is so integral to our social lives and well-being, many facets of everyday life are impacted by the symptoms of EoE,” said Karen Lasch, MD, Vice President, Gastroenterology, U.S. Medical, Takeda. “With no FDA-approved treatments, patients have limited choices and today’s news underscores the urgent needs of this patient population.”

The TAK-721 development program is the first and largest EoE Phase 3 clinical trial program in the U.S. to report results. The NDA filing is based on data from the pivotal Phase 3 trials ORBIT1 and ORBIT2, which investigated the safety and efficacy of TAK-721 in adolescent and adult patients (11 to 55 years of age) with EoE.¹²

About TAK-721 (budesonide oral suspension)

TAK-721 (budesonide oral suspension) is a novel mucoadherent topically active oral viscous formulation of budesonide, formulated specifically as an investigational treatment for eosinophilic esophagitis (EoE).¹ The suspension is intended to treat the localized esophageal inflammation that is caused by EoE.

Priority Review, Breakthrough Therapy designation and Orphan Drug designation do not guarantee approval or commercial availability.

About Eosinophilic Esophagitis (EoE)

Eosinophilic esophagitis (EoE) is a chronic, immune-mediated, inflammatory disease localized in the esophagus.² Although the exact cause of EoE is unknown, it is believed to be triggered by a variety of stimuli including certain foods and environmental allergens.⁸ The chronic inflammation of EoE can lead to a range of symptoms, which can vary by person and age, and include difficulty swallowing, vomiting and heartburn.⁹ If left untreated, inflammation of EoE can worsen and narrow the esophagus, which can lead to food impaction¹¹ that may necessitate urgent endoscopic intervention.¹³ Currently, there are no FDA-approved therapeutic options for the treatment of EoE, though current management guidelines for EoE recommend the elimination of targeted food from patient diets, the use of proton pump inhibitors and/or treatment with topical glucocorticosteroids.¹⁴

Takeda’s Commitment to Gastroenterology in the United States

Takeda sees an urgent need for improving patient care in gastroenterology (GI) and has focused on improving the lives of patients through the delivery of innovative medicines and dedicated patient disease support programs for more than 25 years. We push boundaries and work across modalities, taking on the most complex GI conditions and the most neglected patient needs, boldly advancing original thinking and creatively tackling barriers to make a meaningful difference for patients. Challenging expectations and enabling innovative thinking, Takeda is part of more than 200 collaborations connecting people with a mutual commitment to action. Takeda is leading in areas of gastroenterology associated with high unmet need, such as inflammatory bowel disease, short bowel syndrome, and motility and esophageal disorders. Our GI Research & Development team is also exploring solutions in immune-related diseases, motility and liver diseases.

About Takeda Pharmaceutical Company Limited

Takeda Pharmaceutical Company Limited (TSE: 4502/NYSE: TAK) is a global, values-based, R&D-driven biopharmaceutical leader headquartered in Japan, committed to discover and deliver life-transforming treatments, guided by our commitment to patients, our people and the planet. Takeda focuses its R&D efforts on four therapeutic areas: Oncology, Rare Genetic and Hematology, Neuroscience, and Gastroenterology (GI). We also make targeted R&D investments in Plasma-Derived Therapies and Vaccines. We are focusing on developing highly innovative medicines that contribute to making a difference in people’s lives by advancing the frontier of new treatment options and leveraging our enhanced collaborative R&D engine and capabilities to create a robust, modality-diverse pipeline. Our employees are committed to improving quality of life for patients and to working with our partners in health care in approximately 80 countries. For more information, visit https://www.takeda.com.

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References

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¹ Dellon ES, et al. Clin Gastroenterol Hepatol. 2019;17:666–673.

² Furuta GT, Katzka DA. N Engl J Med. 2015;373(17):1640-1648.

³ O’Shea KM, Aceves SS, Dellon ES, et al. Gastroenterology. 2018;154(2):333-345.

⁴ Dellon ES. Gastroenterol Clin North Am. 2013;42(1):133-153.

⁵ Dellon ES, Jensen ET, Martin CF, et al. Clin Gastroenterol Hepatol. 2014;12(4):589-596.

⁶ Dellon ES. Gastroenterol Clin North Am. 2014;43(2):201-218.

⁷ Dellon ES, Hirano I. Gastroenterology. 2018;154(2):319-332.e3.

⁸ Clayton F, Peterson K. Gastrointest Endosc Clin N Am. 2018;28(1):1-14.

⁹ Muir AB, Brown-Whitehorn T, Gowin B, et al. Clin Exp Gastroenterol. 2019;12:391-399.

¹⁰ Carr S, Chan ES, Watson W. Allergy Asthma Clin Immunol. 2018;14(Suppl 2):58.

¹¹ Hirano I, Futura GT. Gastroenterology. 2020;158(4):840-851.

¹² U.S. National Library of Medicine. Eosinophilic Esophagitis Clinical Studies. Available at:

https://clinicaltrials.gov/ct2/results?recrs=e&type=Intr&cond=Eosinophilic+Esophagitis&phase=2&fund=2.

Accessed November 17, 2020.

¹³ Schupack D. et al. United European Gastroenterology Journal. 2019;7(4):548–556.

¹⁴ Hirano I, Bernstein KA, Jonathan A. et al. Gastroenterology. 2020;158(6):1776-1786.

Contacts

For U.S. Media

Amy McCarthy

amy.mccarthy@takeda.com
+1 781-496-7761

For Japanese Media

Kazumi Kobayashi

kazumi.kobayashi@takeda.com
+81 (0) 3-3278-2095