FDA Approves First Gene Therapy in U.S. -- 2nd Update

The Food and Drug Administration approved a first-of-its kind cancer therapy aimed at bolstering a patient's own immune cells, while the drugmaker behind the treatment attempted to allay worry over the high cost of the procedure.

Swiss pharmaceuticals giant Novartis AG said it would charge $475,000 for the treatment, which involves extracting a patients' disease-fighting blood cells, modifying them to attack cancer cells more vigorously and then reinjecting them in the patient. The $475,000 price tag was significantly cheaper than many analysts had expected.

The procedure can only be undertaken at a limited number of facilities in the U.S. It is highly tailored to individual patients and can take the better part of a month to complete. Those logistical hurdles and its expected high price had cast a shadow over what has otherwise been seen as a groundbreaking treatment.

The treatment, called CLT019 but re-christened by Novartis after the FDA approval as Kymriah, has been shown to dramatically raise the chances of survival for children and young people with an aggressive form of leukemia, who don't respond to standard treatment.

In a bid to blunt criticism over the price, Novartis said it would only take payment for patients covered by Medicaid if they respond to Kymriah within a month of treatment. The company also said it would offer financial assistance -- such as help with copay, travel and accommodation costs -- to privately insured patients.

"We have taken a very responsible approach" to pricing, said Bill Hinshaw, Novartis's U.S. oncology head. He cited independent cost-effectiveness estimates that showed Kymriah could command a price of $600,000 to $750,000.

He said that because patients will be children or young adults -- who typically fall under their parents' or caregivers' private insurance plans, Medicaid or other federal plans aimed specifically at children -- few patients would likely end up paying near the full price out of pocket. That cost will fall to the employers, insurers and taxpayer-funded government programs that fund health care costs.

Novartis Chief Executive Joe Jimenez said the company may charge a lower price in cancer types where the benefit is less dramatic. Novartis is testing Kymriah in adults with diffuse large B cell lymphoma, another form of blood cancer, but the remission rates are lower than in childhood leukemia.

Still, the company has already faced backlash over the price. "While Novartis's decision to set a price at $475,000 per treatment may be seen by some as restraint, we believe it is excessive," said David Mitchell, president of Patients for Affordable Drugs, a campaign group backed by the Laura and John Arnold Foundation which supports efforts to make prescription drugs more affordable in the U.S. "Novartis should not get credit for bringing a $475,000 drug to market and claiming they could have charged people a lot more."

Steve Pearson, head of the Institute for Clinical and Economic Review, which studies the cost-benefit of drugs, said that while the price is lower than analysts expected, it should be judged on whether it reflects the benefit to patients.

"It will leave patients and others wondering" why $475,000 is the right price "instead of 175 [thousand], 600, 750 or any other number," Dr. Pearson said. "And questions will remain about how this price will affect patients' ability to access the drug, and how it will be used to set a benchmark for other uses of this drug and perhaps other cancer drugs in the future."

ICER is currently conducting a cost-benefit analysis of Kymriah that it plans to report early next year.

On Wednesday, the FDA approved Kymriah for children and young adults up to 25 years old, who suffer from acute lymphoblastic leukemia and who have not responded to standard therapy. The decision was expected, following the backing of an FDA advisory committee of experts, who voted unanimously in favor of approval in July.

Kymriah is a form of personalized immunotherapy known as CAR-T, or chimeric antigen receptor T-cell therapy. A patient's T-cells, which are a type of white blood cells, are removed and then sent to a manufacturing center where they are genetically programmed to target leukemia cells. The cells are then infused back into the patient to kill cancer cells.

In the early days of CAR-T research in the late 1990s, "no one ever thought it would be possible to commercialize a therapy like this," said Carl June, a professor of immunotherapy at the University of Pennsylvania, whose research led to the development of Kymriah. "I think the whole cancer world's going to be changed forever," referring to the potential of CAR-T to treat more types of cancer.

So far, it has only shown promise in a few blood cancers, but Novartis is in the early stages of testing CAR-T in some forms of lung and brain cancer. Dr. June said that some of the first leukemia patients to receive the CAR-T treatments about seven years ago remain cancer-free, but it will take longer-term follow-up to see if the therapy completely eradicates cancer in these patients. Dr. June will receive royalties from the product's sales; he said he couldn't disclose specifics.

Acute lymphoblastic leukemia affects bone marrow and blood and is the most common childhood cancer in the U.S. There are about 3,100 patients ages 20 and younger diagnosed with the disease each year, according to the National Cancer Institute. The roughly 600 of these who don't respond to standard treatment would be eligible for Kymriah.

The new treatment field is growing quickly, with the FDA saying it has approved 76 applications by companies and researchers to test CAR-T treatments. "This may turn out to be more broadly applicable," especially in treating other types of blood cancer, said Peter Marks, director of the FDA's Center for Biologics Evaluation and Research. This week, Gilead Sciences Inc. agreed to pay about $11 billion for Kite Pharma Inc., in an ambitious bet on the field.

Write to Denise Roland at Denise.Roland@wsj.com and Peter Loftus at peter.loftus@wsj.com

(END) Dow Jones Newswires

August 30, 2017 16:03 ET (20:03 GMT)