Breaking New Ground: Kymriah™ is the First Ever Gene Therapy Approved

On the 30th August 2017, KymriahTM (tisagenlecleucel, try saying that three times fast!) became the first gene therapy approved by the US Food and Drug Administration (FDA) and was granted priority review and breakthrough therapy designations. KymriahTM is a cell-based gene therapy approved for the treatment of patients up to 25 years of age with B-cell precursor acute lymphoblastic leukaemia (all) that is refractory or in second or later relapse. As a chimeric antigen receptor T-cell (CAR‑T) therapy, KymriahTM is a truly personalised medicine. A patient’s own T-cells are isolated from their blood and sent to a manufacturing center where the cells are genetically modified to include a new gene that codes for a specific protein, a chimeric antigen receptor (CAR). The modified cells are then infused back into the patient, with the modified T‑cells equipped to target and destroy leukaemia cells displaying CD19 on their surface. The cost of this personalized medicine is not cheap, it is priced at $475,000 dollars per treatment course. However, KymriahTM addresses an important unmet need. The prognosis for patients with relapsed of refractory B-cell precursor ALL is poor; less than 10% of patients survive 5 years (1).

Efficacy results highlight why KymriahTM received FDA approval. Results from the ELIANA trial reported that 83% (52/63) of paediatric patients treated with KymriahTM achieved complete remission within 3 months (2). Although the long-term efficacy results are keenly anticipated, the short-term results are promising.

KymriahTMhas been reported as a ground-breaking treatment by experts. Dr Scott Gottlieb, from the FDA, said: "We're entering a new frontier in medical innovation with the ability to reprogram a patient's own cells to attack deadly cancer. New technologies such as gene and cell therapies hold out the potential to transform medicine and create an inflection point in our ability to treat and even cure many intractable illnesses".

Other CAR-T therapies are currently in the pipeline, including UCART123 (Cellectis), NKR-2 (Celyad), and KTE-C19 (Kite Pharma) recently acquired by Gilead Sciences. In particular, Gilead Sciences are expecting a decision on the approval for KTE-C19 in October 2017.

The success of the already approved KymriahTM and other therapies which may closely follow could be the first steps toward gene therapy becoming a mainstream treatment for cancer, and potentially in other disease areas.

Preparing for FDA approval requires careful planning combined with a critical understanding of the evidence requirements for specific diseases and knowledge of the treatment options. The combination of our in-house expertise and the recent acquisition of Context Matters allows DRG to provide insight and strategic guidance to prepare for successful entry into the US market. Please talk to us at DRG if you want to learn more.


References/website links

  1. Ronson A, Tvito A, Rowe JM. Treatment of relapsed/refractory acute lymphoblastic leukemia in adults. Current oncology reports. 2016;18(6):39.
  2. Novartis. Novartis receives first ever FDA approval for a CAR-T cell therapy, Kymriah(TM) (CTL019), for children and young adults with B-cell ALL that is refractory or has relapsed at least twice. 2017. Available at:

Contributors: Eanna Connaughton, Medical Writer
Published on: 11 October, 2017