https://bio.nikkeibp.co.jp/atcl/news/p0/26/02/19/00093/
Qualips’ ReHeart is a product made by inducing the differentiation of allogeneic iPS cell lines provided by the Kyoto University iPS Cell Research Foundation (CiRA_F) into cardiomyocytes, removing undifferentiated cells, and forming them into sheets. The efficacy, effectiveness, or performance of this device is to treat severe heart failure due to ischemic cardiomyopathy when standard treatments, including drug therapy and invasive therapy, are ineffective. Three sheets are implanted onto the surface of the patient’s heart via left-sided open-heart surgery. Prednisolone, tacrolimus, and mycophenolate mofetil are also administered orally for 90 days.
Sumitomo Pharma’s dopamine neural progenitor cells are transplanted into both putamen nuclei via stereotactic brain surgery, with a target of 5.4 x 10⁶ cells per side. Approximately 1.8 x 10⁶ cells per route are injected into 6-9 locations, spaced 1-2 mm apart, via three administration routes through a small hole in the skull, at a rate of approximately 0.1 μL/second. Tacrolimus is also administered orally. It is expected that the cells will take root in the patient’s brain, produce dopamine, and demonstrate therapeutic efficacy.
CiRA issued a press release following the approval. Director Takahashi commented, “We are deeply grateful to the many people who supported our research. However, even if approval (with conditions and a time limit) is obtained, that is not the end goal. We will now move on to the stage of rigorously verifying efficacy and safety by administering the cells in actual clinical settings.” Emeritus Director Shinya Yamanaka commented, “20 years after the announcement of mouse iPS cells, I am extremely pleased to have taken this major step toward social implementation. It is important to proceed steadily, one step at a time, with scientific caution, without becoming complacent.”
The conditional and time-limited approval system for regenerative medicine products is primarily utilized when a pipeline lacks a large number of target patients or the establishment of a control group makes it difficult to conduct a standard-scale Phase 3 clinical trial. If efficacy is estimated from early clinical trial data and safety is confirmed, approval is granted under this system, subject to conditions and time limits. Post-marketing surveillance involves post-marketing clinical trials and drug use results surveys, and efficacy and safety must be verified within seven years before formal approval can be obtained by resubmitting an approval application.
This system was introduced under the Pharmaceuticals and Medical Devices Act (PMD Act) that came into effect in November 2014. Developers of regenerative medicine products hoped it would allow them to market their products without the cost and effort of conducting Phase 3 clinical trials. However, in recent years, it has become clear that even with conditional and time-limited approval, developers are unable to rest easy. This is because Anges’s Collategene (beperminogen perplasmid) and Terumo’s HeartSheet (human [autologous] skeletal muscle-derived cell sheets), which had received conditional and time-limited approval and were aiming for full approval, were withdrawn from the market one after another in June and July 2024, nearing the deadlines of their respective approvals.