Novartis Wins FDA Approval for Groundbreaking Cancer Gene Therapy

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Access is also an issue: vehicle T immunotherapies are very complex and costly to make, meaning they will be astronomically pricey.

In addition to Kymriah's approval, FDA also announced the expanded approval of Roche's Actemra (tocilizumab) to treat auto T-cell-induced severe or life-threatening cytokine release syndrome in patients 2 years of age or older.

The treatment is for people with the condition under 25 years old.

The treatment was pioneered by Carl June at the University of Pennsylvania.

Numerous pharmaceutical companies are investing in gene-therapy projects. Although the price of the therapy is high by any standard, Cooper stated that Novartis is "committed to doing everything we can to ensure that children and young adults who can benefit from [tisagenlecleucel] have access to the therapy and we are working with payers to ensure they fully understand the value of [it] and provide coverage for patients".

"Through our collaboration with Novartis, we are creating the next wave of immunocellular cancer treatments, and are eager to progress CAR-T therapy in a host of hematologic and other cancer types".

At $475,000 a pop, the therapy isn't cheap, but most people would consider that a small price to pay for a cure from a bad, life-threatening disease.

Of those, 600 United States patients would likely meet the criteria of being under 25 and having relapsed or refractory ALL, according to Novartis.

Novartis also 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.

Emily Whitehead in May, five years after she became the first pediatric patient in the world to receive the vehicle T therapy.

Shares of Gilead Sciences Inc, which this week announced an $11.9 billion deal to buy Kite Pharma, were up 6.2 percent to $80.47 on Wednesday afternoon, as Kite is widely expected to receive the next US approval of a CAR-T therapy for a different blood cancer. It's a type of cancer immunotherapy - or a therapy that harnesses the body's immune system to take on cancer cells.

Some details that underscore this complexity were highlighted by STAT.

The cancer-killers are then put back inside the patient and once they find their target they multiply.

Novartis had received a trifecta of designations that helped speed approval: the FDA gave fast-track, breakthrough therapy, and priority review status to tisagenlecleucel's application.

Now 12, she was among those calling on the FDA to approve Kymriah for other patients like her. The program is meant to provide healthcare professionals with an informed understanding of the risks associated with the CAR-T treatment. This week, Gilead Sciences Inc. agreed to pay about $11 billion for Kite Pharma Inc., in an ambitious bet on the approach. (Currently, CAR-T companies have been focusing on blood cancers.) "We also want to see if we can expand it to other diseases down the line", he said.

The FDA based its decision on a clinical trial involving 63 patients with B-cell ALL. Novartis estimates there are about 32 centers around the country suited to manage the technology and said at least 20 facilities could begin offering care within the next month.

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