Kymriah becomes the first FDA-approved CAR-T (2017)
The FDA approved Novartis's tisagenlecleucel (Kymriah) for children and young adults with relapsed B-cell acute lymphoblastic leukemia. Pivotal trial data showed an 83% remission rate in a population with few options. The agency cleared it three months ahead of its decision date.
Kymriah launched at $475,000 per infusion and forced insurers, hospitals, and Medicare to build new payment and delivery systems for one-time cell therapies.
Eight more CAR-T therapies have since been approved, building the regulatory and manufacturing template Immix is following with NXC-201.
Kymriah set the precedent for high-priced, one-time cell therapies in blood cancers with no other good options. AL amyloidosis would be the next major category if NXC-201 clears.
