FDA Approves First Gene Therapy in US

31 August, 2017, 01:46 | Author: Clint Gray
  • Novartis has developed the ground-breaking leukaemia therapy

Novartis AG on Wednesday won highly anticipated USA approval for the first of a new type of potent gene-modifying immunotherapy for leukemia, a $475,000 treatment that marks the start of a potential new treatment paradigm for some cancers.

In response to the possibility of CRS development, the FDA has also expanded the approval of tocilizumab (Actemra; Genentech) to treat CAR-T cell-induced severe or life-threatening CRS in patients 2 years of age and older.

Kymriah is a type of personalized immunotherapy known as CAR-T, or chimeric antigen receptor T-cell therapy.

"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 Dr. Scott Gottlieb said in an FDA news release. The company did say that it's made an agreement with the USA government to pay for the drug only when pediatric or young adult patients with the cancer respond to treatment by the end of their first month.

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Based on available data, patients on the treatment have had an 89% chance of surviving at least six months and a 79% chance of surviving at least a year, with most being relapse-free at that point.

What's perhaps most exciting about CAR-T is that it operates similarly to a vaccination, providing the patient with lifetime protection against the targeted cancer. Gilead shares jumped 5.5 percent on Wednesday as Kite is widely seen as likely to receive the next US approval of a CAR-T therapy.

ALL is an aggressive cancer that affects some 3,100 people aged 20 and younger each year in the United States, making it the nation's most common childhood cancer.

It's too soon to tell how widely applicable CAR-T will be - it's especially suited to leukemia since infusing patients with T-cells is relatively simple.

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The one-time treatment has a boxed warning for cytokine release syndrome or CRS, a life-threatening side effect that can cause blood pressure to drop dangerously low.

The company said it plans to work with the Centers for Medicare and Medicaid Services to implement the pricing strategy.

"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", said Peter Marks, MD, PhD, director of the FDA.

Novartis is required to conduct followup study to assess the safety of the treatment long-term.

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