FDA Approves First Gene Therapy in the United States

Novartis has developed the ground-breaking leukaemia therapy

The FDA decision was expected after an agency advisory panel met in July to discuss the risks and benefits of the Novartis treatment.

"At Novartis, we have a long history of being at the forefront of transformative cancer treatment", said Joseph Jimenez, CEO of Novartis. "I believe this therapy may become the new standard of care for this patient population".

With the treatments available now, fewer than 10 percent of patients with the relapsed, hard-to-treat leukemia are alive five years after diagnosis.

The immunotherapy now can be used in children and young adults with B-cell ALL that will not respond to other therapies, the FDA announced.

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"We're entering a new frontier in medical innovation with the ability to reprogram a patient's own cells to attack a deadly cancer", FDA Commissioner Scott Gottlieb said. "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 groundbreaking treatments that have the potential to be life-saving".

"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), in the FDA's post. Kymriah is approved for use in the most common form of the disease and is only for patients whose cancer hasn't responded to initial treatments.

Kymriah, a cell-based gene therapy known as a CAR-T drug, was approved to treat patients up to age 25 with what's called B-cell acute lymphoblastic leukemia. The modified cells are then transfused back.

In this type of therapy, patients' own immune cells are modified ex vivo to include a gene for the auto protein, which directs them to attack leukemia cells carrying the CD19 antigen. Its approval is likely to set the stage for the FDA to approve a slew of CAR-T drugs over the next several years. The agency said that 69% of patients developing the condition during auto T-cell trials "had complete resolution of CRS within 2 weeks following one or two doses" of tocilizumab, which inhibits interleukin-6. Gilead shares were up more than 5 percent on Wednesday as Kite is widely seen as likely to receive the next USA approval of a CAR-T therapy. The company also said it anticipates making additional filings beyond the USA and European Union next year.

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