The FDA Just Approved a Second Revolutionary Gene Therapy. A Third Is Probably on the Way

October 19, 2017, 9:58 PM UTC
Gilead Takes AIDS Drug-Cocktail Approach To Fighting Cancer
Gilead Sciences Inc. signage is seen at the Gilead laboratory in Foster City, California, U.S., on Wednesday, August 10, 2011. Gilead Sciences Inc., the world's biggest maker of AIDS drugs, is re-entering the market for cancer medicines a decade after selling its oncology unit. Photographer: David Paul Morris/Bloomberg via Getty Images
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The Food and Drug Administration (FDA) has approved Gilead Science unit Kite Pharma’s groundbreaking cancer gene therapy treatment Yescarta. It’s the second approval for this pioneering approach to cancer treatment, which involves re-engineering patients’ own immune cells to become cancer killers, after the FDA’s approval of Novartis’ Kymriah in August. And a third, different kind of gene therapy from Spark Therapeutics—to treat a rare inherited form of blindness—is likely to be approved by the end of 2017, too, making this year a landmark for treatments that manipulate the body’s own biological mechanisms in novel new ways to fight deadly diseases.

Yescarta is a so-called chimeric antigen receptor T-cell (CAR-T) treatment. This new kind of approach to fighting blood cancers is truly personalized; immune T-cells are extracted from patients, genetically tinkered to home in on an destroy cancerous cells, multiplied in a lab, and then jolted back into the patient’s body within about two weeks. Gilead’s therapy (which the biotech giant acquired after snapping up developer Kite Pharma for just under $12 billion at the end of the summer) is approved for certain types of the blood cancer non-Hodgkin lymphoma. Novartis’ Kymriah was approved for a form of aggressive leukemia.

But taking out and re-configuring cells isn’t the only kind of genetic-modification approach out there. Spark, for its part, won a unanimous FDA panel backing for its own gene therapy to treat an inherited disorder that leaves young patients blind by repairing the havoc wreaked by defective genes.

“Before surgery, my vision was dark. It was like sunglasses over my eyes while looking through a little tunnel,” one 18-year-old patient told the FDA advisory committee in heartfelt testimony supporting Spark’s therapy. “I can honestly say my biggest dream came true when I got my sight. I would never give it up for anything. It was truly a miracle.” The FDA isn’t compelled to listen to its advisers’ advice, but typically does, especially when the recommendation is so lopsided.

One remarkable aspect of these treatments is they’re essentially one-and-done—patients don’t have to keep receiving infusions over time. But they come with high prices, too. Kymriah has a list price of $475,000 (although it does come with a unique pricing model where Novartis doesn’t receive payment unless the treatment has shown signs of working within the first month); Gilead’s Yescarta will reportedly carry a list price of $373,000. The amounts paid by insurers, benefit managers, and patients typically differ significantly from list prices.

It’s unclear how expensive Spark’s therapy would be if it’s approved, though there’s been speculation it could cost as much as $1 million.

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