FDA OKs 1st Gene Therapy for Use in US


Novartis (NVS) was the first drugmaker to cross the finish line in developing cancer treatments known as CAR-T drugs Wednesday after the Food and Drug Administration approved its drug, Kymriah, for children and adults with a type of bone marrow cancer.

Kymriah, a cell-based gene therapy, is approved in the United States for the treatment of patients up to 25 years of age with B-cell precursor ALL that is refractory or in second or later relapse.

The new treatment involves removing immune cells called T cells from a patient's blood and giving them a gene for a protein called a chimeric antigen receptor, or auto, that directs the T cells to target leukemia cells.

In the FDA press release, Commissioner Scott Gottlieb says, "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".

"We've never seen anything like this before and I believe this therapy may become the new standard of care for this patient population", Dr Stephan Grupp of Children's Hospital of Philadelphia said in a statement. Results showed that the treatment produced an overall remission of 83% within 3 months.

Most patients with ALL recover through other treatments such as radiation, chemotherapy and stem cells.

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Novartis' CAR-T, which will be marketed as Kymriah, leads an emerging field of cell therapies created to engineer the body's immune cells to seek out and destroy cancers.

For more on childhood acute lymphoblastic leukemia, visit the U.S. The cells are then infused back into the patient to kill cancer cells.

"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, director of the FDA's Center for Biologics Evaluation and Research. Kymriah will carry a boxed warning for cytokine release syndrome, a potentially lethal systemic response to the activation and proliferation of CAR-T cells, causing high fever and potential for neurological problems.

Novartis announced an agreement with the U.S. Centers for Medicare and Medicaid Services under which payment for the therapy will be based on clinical outcomes achieved.

Kymriah has a $475,000 price tag; however, patients who do not respond within a month of treatment will not be charged, according to Novartis.

On Wednesday, the FDA also expanded approval for another drug, tocilizumab, to treat CRS in patients 2 and older.