Takeda Pharmaceuticals

“In some cases, Takeda may be able to provide patients access to its investigational medicines outside of a clinical trial if certain conditions are met. The use of investigational medicines outside of a clinical trial is known under various terms such as Pre-Approval Access, Expanded Access, or Named Patient Programs (also widely referred to as compassionate use). A patient’s treating physician may submit a request to Takeda under these circumstances, and Takeda will review each such request taking into account a number of criteria that include but are not limited to: - Patient must have a serious, life-threatening or severely debilitating disease - Patient must be unable to participate in a clinical trial due to eligibility criteria or other reasons - No satisfactory or comparable alternative treatment options are available - Potential benefits of treatment with the investigational medicine outweigh the potential risks and an appropriate dose has been established, both in the determination of Takeda - Adequate supply is available to support e.g. clinical development activities and pre-approval access - Such access must be allowed under local country laws and regulations”

In addition to announcing these results, Takeda indicated that the December 22, 2022, edition of the New England Journal of Medicine (NEJM) included two case reports written and submitted by two physicians who requested compassionate use of TAK-755 to treat patients who were facing critical health complications related to cTTP. The case reports are available on NEJM’s website.