Kymriah, Novartis’ new CAR-T (chimeric antigen receptor T-cell) treatment, has drawn keen attention in Korea after local cancer patients demanded the government approve the drug through an online petition on Cheong Wa Dae.
Although the drug regulator designated Kymriah as an orphan drug in March, no patient has been able to use the drug to date.
The U.S. Food and Drug Administration first granted the nod for Kymriah (ingredient: tisagenlecleucel) as a treatment for acute lymphoblastic leukemia in August 2017. Afterward, Europe and Japan also authorized the use of the first CAR-T therapy.
CAR-T therapy refers to a treatment where chimeric antigen receptor (CAR), which recognizes the characteristic antigen of target cells, is inserted in the surface of T cells. It has drawn much attention with its excellent therapeutic effects on blood cancer, which has been difficult to treat with the existing chemotherapy.
Kymriah also achieved a remission rate of 83 percent in the treatment of relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL) in pediatric and adult patients up to 25 years of age.
In late July, a patient posted an online petition on the presidential office’s website to demand local approval for CAR-T therapy. The petition has attracted about 15,000 online signatures as of Wednesday, one day before the closing of the request.
As the pharmaceutical industry’s interest in the issue and patients’ demand grew, the Ministry of Food and Drug Safety released a statement, saying “The ministry has done everything it could do, such as designating Kymriah as an orphan drug in March, before the drugmaker applies for approval.”
Novartis also said, “We are closely working with the food and drug safety ministry for quick progress.”
The ministry even published “Collected Data of Overseas Permits for Kymriah” on July 31 so that it could reference it during an approval review.
Despite the ministry’s effort, patients are facing many challenges until they can use the drug in Korea.
In CAR-T therapy, physicians separate T cells from the cancer patient’s blood, insert a CAR coding gene-delivering vector into T Cells, and return the blood to the patient.
Kymriah should follow the same manufacturing and treatment process. However, government regulations exist between sending a local patient’s T cells to Novartis’ manufacturing site in the U.S. and bringing back the finished product to Korea.
Besides, the therapy needs a certification of an eligible hospital, education for medical workers, and the transportation system.
Not only will bringing out blood and cells be complicated but also pharmaceutical goods entering Korea need a local quality inspection. However, Kymriah, a finished product, enters in cryopreservation. Unfreezing the drug means an immediate start of the treatment, which makes it difficult for the authorities to check its quality.
Only Novartis-certified hospitals and trained physicians can provide CAR-T therapy. As T cells’ entering and leaving of the country requires cryopreservation through liquefied nitrogen, the treatment needs a proper transportation system, too.
These explain why local patients have no access to Kymria, despite its designation as an orphan drug in March.
Korean biotech companies also actively developing CAR-T therapies include GC Cell, AbClon, Toolgen and Eutilex.
Industry officials noted that the government should urgently set up a regulatory environment suitable for CAR-T therapies and promote their R&D.
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