Novartis AG Wednesday won highly anticipated U.S. 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,
The approval was widely expected after an FDA advisory panel last month unanimously recommended the action, and Novartis shares closed virtually unchanged in Swiss trading on Wednesday.
Novartis announced an agreement with the U.S. Centers for Medicare and Medicaid Services under which payment for the therapy will be based on clinical outcomes achieved.
The treatment, to be sold under the name Kymriah, was approved for patients up to 25 years of age who have relapsed or were not helped by initial treatment for B-cell acute lymphoblastic leukemia (ALL).
Kymriah belongs to a new class of drugs called chimeric antigen receptor T-cell, or CAR-T therapies, that involves removing disease-fighting T cells from a patient, genetically modifying them to better recognize and attack cancer and then replacing them, where they can circulate for years seeking out the disease.
U.S. biotech Gilead Sciences Inc jumped into the field this week with its announcement of an $11.9 billion deal to buy Kite Pharma. Gilead shares were up more than 5 percent on Wednesday as Kite is widely seen as likely to receive the next U.S. approval of a CAR-T therapy.
Shares of Bluebird Bio Inc, which is also developing a treatment in the emerging field, were up 11.8 percent at $114.47 on the Nasdaq.
“Two years ago many people would have told you these types of treatments were science fiction,” said Brad Loncar, Chief Executive of Loncar Investments, which runs the Loncar Cancer Immunotherapy ETF
In clinical trials, CAR-T therapies have shown remarkable efficacy against blood cancers. In the pivotal Novartis trial, 83 percent of patients achieved remission with a disease that has historically poor outcomes.
“We’ve never seen anything like this before and I believe this therapy may become the new standard of care for this patient population,” Dr Stephan Grupp of Children’s Hospital of Philadelphia said in a statement.
However, this type of therapy is not without risk of severe side effects as the immune system goes into high gear. Kymriah will carry a boxed warning for cytokine release syndrome, a potentially lethal systemic response to the activation and proliferation of CAR-T cells, causing high fever and potential for neurological problems.
Juno Therapeutics Inc last year reported a handful of patient deaths during trials of its CAR-T therapy for leukemia. Its shares were down 5.7 percent at $41.30.
“I think this is the most exciting thing I’ve seen in my lifetime,” said Dr. Tim Cripe, an oncologist who was part of the FDA advisory committee panel that voted in July to recommend the drug’s approval.
The highly personalized treatment is called CAR T-cell therapy (CAR is short for chimeric antigen receptor). It’s a type of cancer immunotherapy, which harnesses the body’s immune system to take on cancer cells. It removes a person’s cells, reengineers them, then puts them back in their body to attack cancer cells.
“We’re entering a new frontier in medical innovation with the ability to reprogram a patient’s own cells to attack a deadly cancer,” the FDA commissioner, Scott Gottlieb, said in a statement. “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.”
The one-time treatments won’t come cheap — Novartis said it would cost $475,000, though it also said in a news release Wednesday that it was working with the Centers for Medicare and Medicaid Services to come up with a payment system that reflects how well the drug works in a person. As part of the collaboration, CMS will have to pay “only when pediatric and young adult [acute lymphoblastic leukemia] patients respond to Kymriah by the end of the first month,” Novartis said.
Novartis’ therapy is one of two cutting-edge treatments for blood cancers that are poised to get approved by the end of the year.
The FDA is also expected to make a decision about another CAR-T therapy, from Kite Pharma, which just got acquired by Gilead Sciences, for aggressive B-cell non-Hodgkin lymphoma. Kite said in February that of the 101 patients in its trial, 36% had a complete response to the treatment (meaning the cancer had disappeared) after six months.
It’s a type of cancer that Novartis also wants to get approval to treat. In June, Novartis released data from its Phase 2 trial of tisagenlecleucel in adult patients with diffuse large B-cell lymphoma, an aggressive form of lymphoma. Of the 51 patients in the trial, 23 had either a complete response or a partial response (meaning their tumor displayed signs of shrinking).
New treatments like Kymriah face some other challenges:
- Manufacturing the drugs is no small feat, considering the personalized treatment requires taking out a person’s cells, reprogramming them, then reinserting them. The company that can do that quickly and safely could have the advantage in the competitive CAR-T therapy space.
- Trials of these kinds of treatments have been deadly in the past. In May, Kite disclosed that one person had died in a clinical trial for its late-stage CAR-T therapy from cerebral edema, a condition in which excessive fluid causes the brain to swell. And last year, another company, Juno Therapeutics, said five people in its clinical trials had died, all from cerebral edema.
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