Nippon Shinyaku’s Basic Policy for R&D
Offering quality pharmaceuticals at the earliest possible time
True to Nippon Shinyaku’s business philosophy, “Helping people lead healthier, happier lives,” in the pharmaceuticals division we aspire to offer innovative quality pharmaceutical drugs to people troubled by ill health, thus alleviating their suffering and enhancing their wellbeing. We strive to do this by focusing on the areas where Nippon Shinyaku’s strengths shine in particular.
R&D in FY 2019
Two drugs launched for diseases with unmet therapeutic needs
In September 2019, we launched Defitelio in Japan, following the obtainment of governmental approval in June 2019. This drug is indicated for the treatment of sinusoidal obstruction syndrome, also known as veno-occlusive diseases (SOS/VOD), which can occur following hematopoietic stem cell transplant mainly conducted as part of blood cancer treatment. Defitelio, designated an orphan drug in May 2019, is the first drug approved for this indication in Japan. We are quite excited to be able to contribute to the treatment of this disease through this product.
In May 2020, we launched Viltepso in Japan. This drug, indicated for the treatment of Duchenne muscular dystrophy (DMD), was granted governmental approval in March 2020. Prior to this, it had been selected in October 2015 for the Ministry of Health, Labour and Welfare’s Sakigake initiative, which is designed to accelerate the review of highly promising innovative candidate drugs and medical devices. This was followed by orphan drug designation in August 2019. We expect this drug to make a significant contribution to DMD patients’ treatment. In the United States, we filed an application for new drug approval for Viltepso in February 2020, which was received in August of the same year. The new drug has since been marketed. At present, joint international Phase III clinical trials are underway for Viltepso.
Future Prospects for R&D
Broad-based R&D creating products of new value and expanding the pipeline
On the foundation of our drug discovery capabilities that have been strengthened over the years and have mainly produced small molecule and nucleic acid drugs, we intend to add AI, iPS cells, and other new technologies, as well as new modalities such as gene therapy, so as to broaden the spectrum of our drug discovery. We will also promote open innovation to fully avail ourselves of internal and external resources.
Through such R&D activities, we hope to constantly create products of new value, offering innovative new drugs that fulfill hitherto unmet therapeutic needs. Furthermore, we intend to construct a mechanism that enables us to conduct global clinical development as a step to providing our innovative products to patients the world over.
We strive to do this by concentrating our managerial resources on promoting R&D and maximizing the product value in four fields of focus: urology, hematology, intractable and rare diseases (e.g., pulmonary arterial hypertension [PAH], muscular dystrophy), and gynecology.
For the future, on our drug discovery foundation that has given birth to the small molecule drug Uptravi, we are adding new modalities and technologies, including nucleic acid molecules, gene therapy, and nucleic DDS technology, further building distinctive drug discovery capabilities not found among other pharmaceutical companies.
CMC (Chemistry, Manufacturing and Controls) activities are indispensable to develop candidate compounds identified in explorative research into drugs and bring them to patients who need them. CMC activities include process research to establish technologies to mass-produce active pharmaceutical ingredients, research into analytical methods for quality assurance, and formulation development to facilitate product use by patients.