FDA Issues Complete Response Letter for Takeda’s Treatment for Eosiniphilic Esophagitis

Takeda Pharmaceutical said that it has received a Complete Response Letter from the U.S. Food and Drug Administration in response to its New Drug Application for TAK-721 (budesonide oral suspension) for the treatment of eosinophilic esophagitis, a rare chronic inflammatory disease of the esophagus.

The CRL indicates the FDA has completed its review of the TAK-721 NDA and determined that it cannot be approved in its present form. In addition, the FDA recommended an additional clinical study in order to help resolve the FDA feedback.

“We are disappointed by the outcome of the FDA’s review of TAK-721, and that EoE patients will still be without a treatment option that the FDA has approved as safe and effective,” said Ramona Sequeira, president of U.S. Business Unit and Global Portfolio Commercialization for Takeda. “Takeda is assessing the details of the CRL and evaluating a regulatory path forward.”

Eosinophilic esophagitis (EoE) is a rare disease that occurs when eosinophils (a type of white blood cells) accumulate in the esophagus, causing inflammation and can lead to difficulty swallowing or eating, discomfort, and pain. About 1 in 2,000 people suffer from the condition in the United States and there are no drugs specifically approved by the FDA to treat EoE.

TAK-721 (budesonide oral suspension) is an oral viscous formulation of budesonide, formulated as an investigational treatment for eosinophilic esophagitis. It entered the Takeda pipeline through the company’s acquisition of Shire.

The FDA had missed the drug’s original PDUFA date in April, pushing its decision by eight months. TAK-721 had met the co-primary endpoints in a phase 3 trial in 2019, reducing eosinophil counts and easing the difficulty of swallowing, and although the majority of patients reported treatment-emergent side effects, none of them were severe and no deaths were reported. Takeda also conducted a 36-week extension study that it said helped responders maintain the benefits of the treatment compared to those who switched to placebo after initial treatment. The FDA granted TAK-721 priority review at the end of 2020.

Author: Rare Daily Staff