Applying for approval of an epidermolysis bullosa treatment following the Phase II trial
First written in FY2019. 13 of 24 entries say "Advanced", first in FY2020-Q2. Latest entry: FY2026.
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- FY2026AdvancedAnnual report
「The trial achieved closure of refractory ulcers, its primary endpoint, in 3 of 4 patients. Accordingly, the Company plans to file for marketing approval based on the results of the additional Phase II clinical trial.」
The additional Phase II trial serving as the basis for the marketing application has ended and achieved its primary endpoint in 3 of 4 patients. The plan to file for marketing approval based on the trial results remains unchanged. - FY2025AdvancedAnnual report
「The additional Phase 2 clinical trial began in July 2022, dosing of the first patient began in March 2023, and dosing of the final patient was completed in July 2025.」
Dosing of the final patient has been completed in the additional Phase 2 clinical trial preceding the marketing-approval filing for epidermolysis bullosa, advancing the planned filing based on the trial results to the next evaluation stage. - FY2024RestatedAnnual report
「Accordingly, we plan to submit an application for marketing approval based on the results of the additional Phase 2 clinical trial.」
The policy of filing for marketing approval based on the results of the additional Phase 2 clinical trial has been reaffirmed. - FY2024-Q3RestatedAnnual report
「Accordingly, the company plans to file an application for marketing approval based on the results of the additional Phase 2 clinical trial. The additional Phase 2 clinical trial is intended to evaluate the efficacy of redasemtide in treating refractory ulcers in patients with dystrophic epidermolysis bullosa who have refractory ulcers, using ulcer closure as the endpoint. Enrollment is planned at 3 or more subjects.」
The policy of filing for marketing approval based on the results of the additional Phase 2 clinical trial, along with the target population, endpoints, and number of subjects, has been reaffirmed. - FY2024-Q1SoftenedAnnual report
「Accordingly, the company plans to file an application for marketing approval based on the results of the additional Phase 2 clinical trial. The additional Phase 2 clinical trial is intended to evaluate the efficacy of redasemtide in treating refractory ulcers in patients with dystrophic epidermolysis bullosa who have refractory ulcers, using ulcer closure as the endpoint. Enrollment is planned at 3 or more subjects.」
The policy of filing for marketing approval based on the results of the additional Phase 2 trial remains in place. However, the previously stated deadline of May 2024 was not included this time, and planned enrollment was changed to “3 or more subjects.” - FY2023-Q3AdvancedAnnual report
「In May 2023, redasemtide was designated by the Ministry of Health, Labour and Welfare as an orphan drug for dystrophic epidermolysis bullosa. This designation means that the Ministry of Health, Labour and Welfare has given a certain level of recognition to the possibility that redasemtide may be effective against epidermolysis bullosa and to the validity of the current development plan. Shionogi & Co., Ltd. can also receive priority review ahead of other drugs and other support measures so that redasemtide can be made available in clinical practice as soon as possible. This is expected to enable earlier approval and launch by shortening the review period.」
Redasemtide was designated an orphan drug for dystrophic epidermolysis bullosa, providing a clearer path toward early approval and launch through priority review and other measures. - FY2023-Q2AdvancedAnnual report
「The additional Phase 2 clinical trial is scheduled to evaluate the efficacy of redasemtide in treating refractory ulcers in patients with dystrophic epidermolysis bullosa who have refractory ulcers, using ulcer closure as the endpoint. The planned number of subjects is 3, and the trial is scheduled to be conducted by May 2024.」
The target population, endpoints, planned enrollment of 3 subjects, and the trial deadline of May 2024 have been specified for the additional Phase 2 trial preceding the marketing-approval filing. - FY2023-Q1RestatedAnnual report
「Accordingly, we plan to submit an application for marketing approval based on the results of the additional Phase 2 clinical trial.」
The policy of filing an application for marketing approval based on the results of the additional Phase 2 clinical trial has been reaffirmed. - FY2022AdvancedAnnual report
「Accordingly, we plan to submit an application for marketing approval based on the results of the additional Phase 2 clinical trial.」
The additional Phase 2 clinical trial preceding the marketing-approval filing began in July 2022, and plans to file based on the results of the additional trial have taken concrete shape. - FY2022-Q1RestatedAnnual report
「Accordingly, Shionogi & Co., Ltd., the licensee for this drug, had been consulting with the regulatory authorities to file an application for marketing approval based on the results of the Phase 2 clinical trial and follow-up study. However, although the trial identified cases showing marked efficacy, it concluded that additional cases demonstrating efficacy would be needed. An additional clinical trial is therefore scheduled to confirm the reproducibility of the trial results.」
The previous policy of pursuing a marketing-approval filing remains in place, while the plan to first confirm reproducibility through an additional clinical trial has been reaffirmed. - FY2021SoftenedAnnual report
「Although the trial identified cases showing marked efficacy, it concluded that additional cases demonstrating efficacy would be needed. An additional clinical trial is therefore scheduled to confirm the reproducibility of the trial results.」
The policy has shifted from expecting to file for marketing approval based on the Phase 2 trial results to first confirming reproducibility through an additional clinical trial. - FY2021-Q3RestatedAnnual report
「The number of new patients per year is expected to be approximately 15, and because it would be difficult to plan a large-scale Phase 3 trial, we expect, as originally planned, to file an application for marketing approval based on the results of the Phase 2 trial.」
The policy of filing an application for marketing approval based on the Phase 2 trial results has been reaffirmed, on the grounds that a large-scale Phase 3 trial would be difficult for a rare disease. - FY2021-Q2RestatedAnnual report
「The number of new patients per year is expected to be approximately 15, and because it would be difficult to plan a large-scale Phase 3 trial, we expect, as originally planned, to file an application for marketing approval based on the results of the Phase 2 trial.」
The policy of filing an application for marketing approval based on the Phase 2 trial results has been reaffirmed, on the grounds that a large-scale Phase 3 trial would be difficult for a rare disease. - FY2021-Q1RestatedAnnual report
「The number of new patients per year is expected to be approximately 15, and because it would be difficult to plan a large-scale Phase 3 trial, we expect, as originally planned, to file an application for marketing approval based on the results of the Phase 2 trial.」
The policy of filing an application for marketing approval based on the Phase 2 trial results has been reaffirmed, on the grounds that a large-scale Phase 3 trial would be difficult for a rare disease. - FY2020AdvancedAnnual report
「Development of a treatment for dystrophic epidermolysis bullosa is currently in preparation for the filing of an application for marketing approval as a pharmaceutical product.」
The status has advanced from an expected marketing-approval filing to preparation for the filing itself. - FY2020-Q2AdvancedAnnual report
「As it would be difficult to plan a large-scale Phase 3 trial, we expect, as originally planned, to file an application for marketing approval based on the results of the Phase 2 trial.」
Following confirmation of efficacy and safety in the preliminary Phase 2 trial results, the expectation of filing an application for marketing approval based on the trial results has been maintained. The filing itself has not yet been reported. - FY2019First statedAnnual report
「Accordingly, based on the results of the Phase 2 trial, we expect to file an application for marketing approval of the drug.」
Given that this is a rare, intractable disease, the company expects to file an application for marketing approval based on the Phase 2 trial results.