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September 5, 2017

First gene therapy to treat cancer gets FDA OK

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FDA on Wednesday approved the first gene therapy drug for sale in the United States.

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The drug, Novartis' Kymriah, was approved to treat B-cell acute lymphoblastic leukemia in children and adults up to age 25 for whom other treatments did not work. Novartis estimates that about 600 patients each year will be eligible for the drug.

How the drug works

Scientists call Kymriah a "living drug" because it uses a patient's own cells to target the disease. The treatment, a type of CAR-T cell immunotherapy, entails extracting immune system T-cells from each patient's blood. The cells then are frozen and shipped to a Novartis facility, where they are genetically modified to attack the patient's healthy and cancerous B-cells that have the CD-19 protein. Physicians then infuse the T-cells back into the patient, at which point they begin fighting the cancer.

Novartis said it is working to train staff at 20 hospitals to provide Kymriah within one month. The company said the drug eventually will be available at a total of 32 sites.

Research has shown that 83 percent of 63 pediatric and young adult patients who took Kymriah were in remission within three months. However, it is not clear how long the drug's effects last. For instance, some patients relapsed months after entering remission from Kymriah, the Associated Press reports.

In addition, Kymriah can have serious side effects, such as cytokine-release syndrome (CRS), in which an individual's immune system overreacts to the drug. FDA said Kymriah will include a boxed warning about CRS. In addition, the agency on Wednesday expanded its approval of Actemra (tocilizumab) to treat CRS caused by CAR T-cell treatment in patients ages 2 and older.

FDA also is requiring a post-market study on the drug and mandatory training for all individuals "involved in the prescribing, dispensing, or administering" of Kymriah.

Observers applaud Kymriah's approval 

FDA Commissioner Scott Gottlieb said, "We're entering a new frontier in medical innovation with the ability to reprogram a patient's own cells to attack a deadly cancer." He added, "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."

Peter Marks, director of FDA's Center for Biologics Evaluation and Research, said, "Kymriah is a first-of-its-kind treatment approach that fills an important unmet need for children and young adults with this serious disease."

American Hematology Society President Kenneth Anderson called Kymriah's approval a first step in expanding gene therapies. "While the importance of CAR-T cannot be overstated, this approval only pertains to a small population of children," he said, adding, "More research is needed to make this therapy more effective for a broader population, to reduce the severe side effects that patients experience during treatment, and ultimately to find a broader application beyond blood cancers."

Kevin Curran, a pediatric oncologist at Memorial Sloan Kettering Cancer Center, said Kymriah's approval is "exciting for pediatric oncology" and hopes the treatment will eliminate the need for bone marrow transplants.

Price sparks debate

Novartis announced that it would price Kymriah at $475,000 for a single course of treatment. The company said it considered various factors when setting the price, such as the cost of bone-marrow transplants. According to Novartis, such transplants can cost up to $800,000 for a typical treatment for individuals with leukemia who relapsed.

Novartis said it will offer a program to help patients with out-of-pocket costs for the drug and would work with CMS on payment structures for the drug. Novartis in a statement said it will not charge patients covered by Medicaid for Kymriah if it fails to help them within their first month of treatment.

CMS Administrator Seema Verma in a statement said, "Innovations like this reinforce our belief that current health care payment systems need to be modernized in order to ensure access to new high-cost therapies, including therapies that have the potential to cure the sickest patients. Improving payment arrangements is a critical step towards fulfilling President Trump's promise to lower the cost of drugs."

However, some patient advocates spoke out against the price, Kaiser Health News reports.

David Mitchell, president of the advocacy group Patients for Affordable Drugs, in a statement said, "Novartis should not get credit for bringing a $475,000 drug to market and claiming they could have charged people a lot more." He added, "The drug pricing system in America is completely broken. Until policy in this country changes, the vicious cycle of patients struggling under high drug prices will continue" (Szabo, Kaiser Health News, 8/30; Berkrot, Reuters, 8/30; FDA release, 8/30; Stein, "Shots," NPR, 8/30; Gever, MedPage Today, 8/30; McGinley/Johnson, "To Your Health," Washington Post, 8/30; Neergaard, AP/Sacramento Bee, 8/31; Roland/Loftus, Wall Street Journal, 8/30; Manchester, The Hill, 8/30).

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