2024.11.11

Breakthrough Designation for Devices and Regenerative Medicine Products in Japan

In 2015, Japan introduced a new breakthrough designation for treatments in Japan. That system is referred to as the Sakigake designation system and products that receive that designation can benefit from an expedited regulatory review process and additional support similar to orphan designation. The key criteria for Sakigake (breakthrough) designation are as follows:

(1) the new treatment is innovative with a new mechanism of action

(2) the new treatment addresses a severe condition

(3) the new treatment is considered to be highly efficacious and/or safe

(4) the new treatment is likely to be developed prior to (or within 3 months of) other similar countries

Since April 2015, 14 device products, 12 regenerative medicine products, and 2 in-vitro diagnostic (IVD) products have received Sakigake (breakthrough) designation. For devices, more recently, Sound Wave Innovation’s IPUS-Brain transcranial low-intensity pulsed ultrasound therapy device (LIPUS-Brain) received Sakigake (breakthrough) designation for the treatment of patients with early Alzheimer’s disease in September 2024 and iCorNet Laboratory’s cardiac shape correcting net product received Sakigake (breakthrough) designation in June 2020 for reduction of excessive left ventricular wall tension and prevention of the development of cardiac remodeling in patients with non-ischemic dilated cardiomyopathy with progressive cardiac enlargement.

For regenerative medicine products, more recently, JUNTEN BIO’s inducible inhibitory T-cells (JB-101) received Sakigake (breakthrough) designation in June 2020 for the reduction or interruption of immunosuppressive drugs after liver transplantation. Moreover, SanBio’s Akuugo (vandefitemcel) suspension for intracranial implantation received Sakigake (breakthrough) designation in April 2019 as a treatment for improving chronic motor paralysis from traumatic brain injury (TBI). For IVDs, Toray Industries’s DNA chip microarray pancreatic and biliary tract cancer test kit received Sakigake (breakthrough) designation in April 2019 for the diagnosis of pancreatic and biliary tract cancer and Sysmex’s OncoGuide NCC Oncopanel system received designation in February 2019 as a comprehensive genomic profiling test for tumor tissue in patients with solid tumors.

In April 2022, a new Sakigake (breakthrough) premium was also introduced for newly listed devices that allows for a reimbursement premium of +10% if Sakigake (breakthrough) designation is received prior to listing. Since then, among the devices that received Sakigake (breakthrough) designation and were approved/listed in Japan, only one device, Teijin Medical Technologies’s novel surgical patch, SYNFOLIUM, has received a Sakigake (breakthrough) premium. Among the 3 regenerative medicine products that received Sakigake (breakthrough) designation and are already approved / listed in Japan, all 3 products received a Sakigake (breakthrough) reimbursement premium of +10%. Those products include Novartis’s Zolgensma (onasemnogene abeparvovec), Daiichi-Sankyo’s Delytact (teserpaturev), and Nipro’s Stemirac autologous mesenchymal stem cells. Lastly, for the two IVDs mentioned that received Sakigake (breakthrough) designation, the OncoGuide NCC Oncopanel system was ultimately not reimbursed as a stand-alone product and therefore did not receive a premium and information on Toray’s DNA chip microarray kit was not found.

Closing thoughts:

While Sakigake (breakthrough) designation can offer a lot of advantages when it comes to regulatory approval and potentially an additional reimbursement premium, launching in Japan early (or around the same time as other markets) is a prerequisite, so early planning is needed. In fact, a quick glance at the list(s) of device and regenerative medicine products that have received Sakigake (breakthrough) designation shows that, unlike products that received orphan designation, nearly all the products that have received Sakigake (breakthrough) designation are manufactured by companies headquartered in Japan. This suggests that early planning is needed to coordinate the development schedule in Japan alongside other markets and to obtain designation early. If you want to take advantage of this designation for your product in Japan, then please contact GinePro as early as possible so that we can help accelerate your evidence development for Japan.

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