FDA Green Lights Very first CAR-T Cancer Drug – Scientific American
FDA Green Lights Very first CAR-T Cancer Drug
The leukemia treatment approval opens up a fresh front for gene therapies in the U.S.
- By Damian Garde, STAT on August 30, 2017
The Food and Drug Administration on Wednesday approved a futuristic fresh treatment to treating cancer, clearing a Novartis therapy that has produced unprecedented results in patients with a infrequent and deadly cancer.
The treatment, called a CAR-T, is made by harvesting patients` white blood cells and rewiring them to home in on tumors. Novartis`s product is the very first CAR-T therapy to come before the FDA, leading a pack of novel treatments that promise to switch the standard of care for certain aggressive blood cancers.
Novartis`s therapy is approved to treat children and youthfull adults with relapsed acute lymphoblastic leukemia. It will be marketed as Kymriah.
In a clinical trial, Kymriah left eighty three percent of participants cancer-free after three months, results oncologists have hailed as a major advance for patients with few other options. The most frequent side effect was an inflammatory storm called cytokine release syndrome, a reaction to CAR-T that can prove fatal in some patients but is commonly managed with immunosuppressant drugs.
«I think this is most arousing thing I`ve seen in my lifetime,» said Dr. Tim Cripe, an oncologist with Nationwide Children`s Hospital, at an FDA meeting on Kymriah in July.
Now the question now turns to price. Novartis did not instantly disclose how much it intends to charge for Kymriah, but analysts expect it to cost as much as $700,000 for a course of treatment, and the Swiss drug maker has already come under pressure from a patient advocacy group.
In what is perhaps an effort to assuage cost concerns, Novartis said Wednesday that it is working with Medicare on a system in which the government would only pay for CAR-T treatments if patients react within a month.
Unlike well-understood pills and commonly injected biotech drugs, CAR-T presents a radical fresh paradigm for doctors, regulators, and payers. Each dose is custom-tailored for an individual patient, requiring a elaborate process in which human cells must be securely ferried across the country, reliably re-engineered, and soundly returned.
And it remains unclear is just how lucrative a business chance Kymriah presents, whatever the price. There are about Three,100 fresh cases of ALL each year, but harshly seventy percent can be shoved into remission by standard therapy. That could leave just a few hundred patients who might be eligible for Novartis`s therapy, casting doubt on whether the company can get an outsize come back on what will be a substantial manufacturing investment.
Republished with permission from STAT. This article originally appeared on August 30, 2017