First SAKIGAKE designated drug created from collaboration with academia Interviewee 
Vice President & COO, Head of Research & Development
Shigeki Shimasaki

Making external seeds into drugs that meet patient needs

RAPALIMUS® is an orphan drug that was developed from an oral medication sold as an organ transplant immunosuppressant by Pfizer Inc. in the US. Due to it having mTOR inhibitor properties, physicians in academia and researchers were actively studying and gathering data on this medication in terms of its use for other patients. Nobelpharma used their research outcomes as a base and gained marketing approval for RAPALIMUS® tablets, Japan’s first curative drug for lymphangioleiomyomatosis.
Moreover, a research group at Osaka University who were investigating expanding the indication of RAPALIMUS® cooperated with American researchers, and made it clear in physician-led clinical trials that RAPALIMUS® was extremely effective as an external gel on skin lesions accompanying tuberous sclerosis complex (TSC). These academic outcomes were passed on to Nobelpharma, and after verifying these outcomes and the pharmaceutical’s safety through phase III clinical trials and a long-term administration trial, completed it as an external medicine: RAPALIMUS® Gel.

Running business to deliver achievements of academia to patients

Academic outcomes (physician-led clinical trials) were passed on to Nobelpharma, and the first item that we developed (corporate clinical trials) and commercialized was RAPALIMUS® Gel. There was patient need due to rare diseases and ailments, and we were able to introduce this to the market as a new pharmaceutical, the results of which could be clearly felt by patients themselves and physicians.
In addition, RAPALIMUS® Gel was the first pharmaceutical to gain a SAKIGAKE designation. It was the first curative drug for skin lesions accompanying tuberous sclerosis complex (TSC) in both Japan and the world. There are patients with this rare disease all over the world, and so we are aiming to release this pharmaceutical in the US, China, and Europe as the first phase of our overseas expansion of pharmaceuticals.

Strengthening our cooperation with academia and increasing development speed

Although RAPALIMUS® Gel’s effectiveness was made clear in academia, skin lesions accompanying tuberous sclerosis complex (TSC) is a rare ailment with few patients, and other pharmaceutical companies did not attempt to work on it. On top of this, generally National Health Insurance pharmaceutical prices tend to be set low for external medications, and it was assumed that companies could not expect a desirable financial return.
Additionally, academic outcomes cannot necessarily be made into a product that can be sold on the market without changes. When it came to this medication, the research team at Osaka University had carried out non-clinical trials so that they could begin clinical trials, but marketing approval required many more non-clinical trials. There are many different restrictions on new pharmaceuticals—in the case of RAPALIMUS® Gel in particular, it would be used over a long period as an external medication, so additional non-clinical trials that administered this external medicine to animals were needed to confirm its safety. Thus, development was handed over to Nobelpharma.
The wish to help patients who are suffering with rare diseases and ailments and to deliver curative drugs to patients in need as soon as possible is shared by physicians in academia and Nobelpharma. To increase development speed, we will further strengthen our cooperation with academia, and take on the challenge of developing necessary but neglected pharmaceuticals and medical devices.

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