Novartis has recently drawn the attention of local industry watchers concerning whether and when its anticancer therapy Kymriah, a CAR-T (chimeric antigen receptor T-cell), will be able to make its market debut in Korea.
While the U.S., EU, and Japan have authorized the use of Kymriah, the treatment is far from use here. Despite the growing interest of the pharmaceutical industry and demand from local patients, there are still some challenges that the drug has to overcome before receiving approval in Korea.
Jennifer Brogdon, executive director for exploratory immuno-oncology at Novartis Institutes for Biomedical Research (NIBR), reaffirmed her company’s commitment to fixing its limitations as well as Kymriah's development process and product features, at the "Healthcare Innovation Forum 2019" organized by the Korea Health Industry Development Institute on Thursday.
|Jennifer Brogdon, executive director for exploratory immuno-oncology at Novartis Institutes for Biomedical Research (NIBR), explains the benefits of Kymriah, during the "Healthcare Innovation Forum 2019" at Westin Chosun Seoul on Thursday.|
"Each dose of Kymriah is developed by first drawing blood from the patient and isolating blood cells, including T cells. We then send it to a company- designated manufacturing site where T cells are genetically encoded to recognize cancer cells and other cells expressing a specific antigen using an inactive virus," Brogdon said. "The resulting CAR-T cells can now recognize a specific marker produced on the surface of certain cells, including certain cancer cells."
Afterward, the CAR-T cells are multiplied, packaged, and then infused into the patient, she said.
Brogdon explained the additional benefits of Kymriah as a CAR-T treatment.
"Our treatment utilizes cryopreservation in manufacturing, which allows scheduling flexibility for physicians and patients, durability in transit in the case of unforeseen transport delays and preserved cell quality," the Novartis executive said." The treatment is also the only one that has a 4-1BB costimulatory domain, which enhances cellular expansion and persistence. "
Besides, the company has proved the drug's safety and efficacy in global clinical trials in two distinct indications, she noted, adding that such benefits have made Kymriah a groundbreaking treatment that patients can receive just once.
Kymriah, however, is entirely different from traditional small-molecule drugs or biologics in that it is a personalized cell therapy that requires the company to develop a new product per patient. “Our company, therefore, faced some difficulties in setting up the manufacturing infrastructure.”
That is also the problem Novartis is facing in Korea where not only bringing out blood and cells are complicated but also taking pharmaceutical goods into the nation needs a local quality inspection, Brogdon said. However, Kymriah, a finished product which enters in cryopreservation, and unfreezing the drug means an immediate start of the treatment, all of which makes it difficult for the authorities to check its quality.
"Preparing a highly complex production infrastructure for manufacturing the drug was one of the challenges encountered in the development of the drug," Brogdon said. "It is not easy to combine the discipline-based production process into a robust GMP process with strict follow-up of each product."
However, Brogdon stressed that the company's ultimate goal is to manufacture Kymriah for every patient who needs it.
"To do this, we have been increasing sites that can manufacture Kymriah," Brogdon said. "Starting with our production facility in the U.S., the company has expanded to seven sites located in Europe, China, Japan, and Australia."
Novartis is also currently working to develop next-generation production technologies aimed at simplifying the production process for the treatment to make it easier and safer for patients worldwide.
"Cell and gene therapies, including Kymriah, are designed to block the cause of disease or stop disease progression beyond simply managing symptoms," she said. "These treatments usually end in a single treatment and not only improve the underlying cause of the disease, but also can be expected to cure the disease."