Novartis FDA Panel Approval: ‘Pharma Innovation At Its Best’

Novartis AG’s pioneering cancer drug won the backing of a federal advisory panel Wednesday, paving the way for the first gene therapy to be approved in the United States.

A pioneering gene therapy created to treat leukemia has moved one step closer to becoming the first of its kind marketed in the United States, thanks to a crucial recommendation. The cells would then be refrozen and sent back to the patient. Diefenbach, who was not involved in researching the drug and has no ties to its manufacturer, Novartis, described its results as “astounding”. “It represents a paradigm shift in treating cancers”, said Maloney, who is extensively involved in vehicle T-cell research but not in the Novartis product. The disease it’s meant to treat, however, is rare – affecting only about 5,000 people annually.

Makers of immunoglobulin are reluctant to talk about the issue, since their products aren’t approved for patients who develop secondary immune deficiencies that are a complication of another medical condition or treatment.

“Leukemia has very good treatments for most patients, but for the patients it doesn’t work, this is really game-changing”, said Dr. Stephian Grupp of the Children’s Hospital of Philadelphia. And the cost of treating each patient for a lifetime is expected to increase 310 percent, to $604,000 from $147,000 during that same period.

But this therapy is also linked with threatening side effects.

“This is the beginning of something big”, she added. For one thing, the manufacturing process is extremely intensive since each individual batch of the drug needs to be tailored to each individual patient.

Slightly fewer patients experienced neurological side effects, such as seizures and hallucinations, according to the committee’s briefing document.

Patients with ALL who fail chemotherapy typically have only a 16 percent to 30 percent chance of survival. That is because they will only need to be treated once and then the therapy will work on its own. The majority were also relapse-free in the same time frame. Brody has helped design trials for similar drugs but not for Novartis.

The recommendation of ODAC was based on the evaluation of the CTL019 r/r B-cell ALL development program that included the firm-lead ELIANA study, which is claimed to be the first pediatric global CAR-T cell therapy registration trial.

Immunotherapy is a growing field in cancer treatment and medications that alter the body’s immune system to fight cancer are already on the market.

“In the early 2000’s when ACGT was founded, gene therapy was an outlier”, noted ACGT co-founder Barbara Netter, who founded ACGT with her late husband Edward in 2001.

But 15% of the cases of LAL are resistant to treatment. Brody said it could take decades to conclusively say this does not happen. “It’s not an opinion”. Once infused back into the patient, the cells reproduce at a fierce pace to attack the cancer.

“You can put someone else’s red blood cells into you”, he said. The drugmaker is seeking approval to use the one-time treatment for children and young adults with advanced leukemia.

Loncar said approval of tisagenlecleucel would represent an inflection point for investors.

Commenting on the treatment, Dr. Bryan said, “Particularly, we are concerned that the same safety and benefit seen in clinical trials may not translate into routine use”. She got auto T-cell therapy when she was 6 and close to death from leukemia. There, staff use a virus to insert genes into the T cells that encode a cellular receptor that will recognize leukaemia cells.

It was the first cancer immunotherapy to receive the breakthrough designation. Food and Drug Administration. Approval in pediatric ALL now seems all but assured; the FDA rarely bucks the advice of its advisory committees. The agency is expected to rule on the drug by the end of September.