FDA Approve First Gene Therapy in the USA
Just this morning we were discussing what CAR-T therapy involved and what sort of cost patients might be facing should it become available. Now, the US Food and Drug Administration (FDA) have announced that they’ve approved Novartis’ Kymriah (tisagenlecleucel) for paediatric and young adult patients with a specific form of Acute Lymphoblastic Leukaemia (ALL). This announcement marks the first time a gene therapy has been made available to patients in the USA and looks to be the start of a new approach to treating diseases in the clinic.
“We’re entering a new frontier in medical innovation with the ability to reprogram a patient’s own cells to attack a deadly cancer,” said Scott Gottlieb, M.D., FDA Commissioner. “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. At the FDA, we’re committed to helping expedite the development and review of ground-breaking treatments that have the potential to be life-saving.”
ALL is the most common childhood cancer in the USA, with the National Cancer Institute estimating that around 3,100 people under 21 are diagnosed every year. The disease can originate in either B-cells or T-cells, with B-cells being the most common.
In their press release, the FDA state that the therapy has been approved specifically for patients under the age of 25 (inclusive) who have B-cell precursor ALL which is refractory or in second or later relapse, covering an estimated 15-20% of patients. Kymriah works by targeting a patient’s T-cells (a type of immune cell responsible for recognising non-self material) to leukaemia cells that display a CD19 antigen on their surface, triggering an immune response attack against the cancer.
“Kymriah is a first-of-its-kind treatment approach that fills an important unmet need for children and young adults with this serious disease,” said Peter Marks, M.D., Ph.D., Director of the FDA’s Center for Biologics Evaluation and Research (CBER). “Not only does Kymriah provide these patients with a new treatment option where very limited options existed, but a treatment option that has shown promising remission and survival rates in clinical trials.”
Kymriah’s clinical trials have returned promising results, prompting the FDA’s approval. In one multicentre trial involving 63 patients within the requirements, the treatment demonstrated both safety and efficacy, with an overall remission rate after three months of 83%. However, the treatment also has side effects that can be severe in some cases, and so it is being approved with a Risk Evaluation and Mitigation Strategy (REMS) to ensure safe use.
Evaluation and monitoring of the treatment will continue now that it has been approved, but this is still a significant step forwards for Novartis and for the field in general.