First Gene Therapy in the USA “Kymriah” now licensed by FDA
On August 30th, the FDA approved Novartis’ first cell based cancer therapy Kymriah (tisagenlecleucel).
Kymriah is now licensed to treat a rare form of acute lymphoblastic leukemia. In the so-called CAR-T method, T immune cells are extracted from patients, and a gene is introduced enabling the modified cells to recognize, attack, and kill cancer cells upon re-introduction into the body. Serious side effects are associated with this method: the highly activated immune cells cause a strong systemic immune reaction which is hard to manage. In addition, antibodies must be provided to the patient externally, since the cancerous antibody producing cells are eliminated. Plus, only half of patients receiving Kymriah in clinical studies responded to this therapy. Still, many physicians see the new therapy as an essential and potentially life-saving option for juvenile patients not responding to currently available treatment.
As the first licensed gene therapy of the USA, the CAR-T method faces many points of criticism regarding gene therapies in general: The gene therapeutic method of manipulating the body’s own cells will always harbor a residual risk of cell degeneration, potentially leading to cancerous malignancies. To date, long-term effects are extremely difficult to estimate, and effects differ strongly between patients, due to genetic diversity. Finally, gene therapy is controversially discussed from an ethical point of view, offering science fiction like opportunities for manipulating human beings already as unborn children. On the other hand, gene therapy provides unprecedented chances for permanent cure of diverse cancer types and hereditary genetic defects. Thus, licensing Kymriah sets the stage for a new generation of therapeutics.
Novartis tops the race for the first place just ahead of Kite Pharma (this week bought by Gilead Sciences), who is in a second CAR-T approval procedure for Aci-Cel, treating non-Hodgkin's lymphoma. Novartis is also setting new standards of reimbursement: patient representatives oppose Kymriah's prize of $ 475,000 per application not including hospital patient care, arguing that a large part of the basic research took place in government-sponsored institutions. Pricing and reimbursement of gene therapies will be an important topic for the future, and current Novartis’ pricing negotiations, potentially including outcome-based pricing, will be highly influential.
More about the first approved gene therapy in the USA can be found under the following links:
http://www.sueddeutsche.de/gesundheit/krebs-therapie-erste-gentherapie-gegen-krebs-steht-kurz-vor-der-zulassung-1.3629362
http://www.fiercepharma.com/pharma/novartis-crosses-fda-finish-line-monumental-car-t-approval?utm_medium=nl&utm_source=internal&mrkid=54511408&mkt_tok=eyJpIjoiTlRFeFlqWmtNRFkxTWpVNCIsInQiOiJHQ2tUTFZ4Y3BsR21Qc0NnVkhydXNyakhhZjVaamFXR0NjSU5xTnJaQkxsUTlmMHMrSVhyK3FnT0hucjY2Mmd2ZExGeTZtZXltaTU3YXB4elZUdDBleG9tUmxlc2o0MzlkOTc1SEVTSFhxdFNyYmZENXQ4WEprcGNHdlArUks0NCJ9