The US Food and Drug Administration (FDA) on Wednesday approved the first gene therapy, Novartis' Kymriah (tisagenlecleucel) for certain pediatric and young adult patients with a form of acute lymphoblastic leukemia (ALL).
Dubbed an "historic action" by the agency, the approval comes a little more than a month after FDA's Oncologic Drugs Advisory Committee voted unanimously, 10 to 0, in favor of the benefit-risk profile for the first of this new kind of cancer therapy, known as a Chimeric Antigen Receptor T-cell (CAR-T) therapy.
Novartis said the one-time treatment will cost $475,000, which it said was below their internal value-based assessment, though eyebrows will likely be raised over the sticker shock. FDA Commissioner Scott Gottlieb said in a press call that Novartis' application was approved in seven months.
Wednesday's approval also came with an interesting twist: Novartis announced that it will collaborate with US Centers for Medicare and Medicaid Services (CMS) "to make an outcomes-based approach available to allow for payment only when pediatric and young adult ALL patients respond to Kymriah by the end of the first month. Future potential indications would be reviewed for the most relevant outcomes-based approach."
Novartis CEO Joseph Jimenez stressed Wednesday that there will be no charge for the therapy if there is no response within the first month.
Novartis, which won a priority review voucher alongside the approval, noted Kymriah will be manufactured for each individual patient using their own cells at a facility in Morris Plains, New Jersey, where the company says it has designed a manufacturing and supply chain platform that allows for an individualized treatment approach on a global scale.
"This process includes cryopreservation of a patient's harvested (or leukapheresed) cells, giving treating physicians and centers the flexibility to initiate therapy with Kymriah based on the individual patient's condition," the company added.
In addition to Kymriah's approval, FDA also announced the expanded approval of Roche's Actemra (tocilizumab) to treat CAR T-cell-induced severe or life-threatening cytokine release syndrome in patients 2 years of age or older.
"In clinical trials in patients treated with CAR-T cells, 69 percent of patients had complete resolution of CRS within two weeks following one or two doses of Actemra," FDA said.
Gottlieb also noted that there are 76 other active INDs for CAR-T therapies (and 550 active INDs for gene therapies) while CBER Director Peter Marks said other potential areas for CAR-T treatments include hematologic malignancies, adult leukemias and certain non-Hodgkins lymphomas.
FDA Approval Letter