With 2-pronged filing, Novartis' CAR-T med Kymriah aims to jump into Europe with both feet

Novartis has been building up its manufacturing capabilities to handle the intensive nature of producing CAR-T therapies.

Novartis is blazing ahead with its bid to take its new CAR-T therapy, Kymriah, into new realms with a double-pronged approval application in Europe.

After winning an initial FDA approval in children and young adults with acute lymphoblastic leukemia (ALL) and filing for an FDA nod in diffuse large B-cell lymphoma (DLBCL), Novartis has asked European Union regulators to clear Kymriah to treat both diseases. Both groups of patients are in "dire need" of new treatments, Novartis said in a statement.

The DLBCL approval would be the larger, because pediatric ALL cases are more rare—and it would be the more competitive, what with Gilead Sciences and its drug Yescarta, which won a broader FDA approval in lymphoma last month. Yescarta, the centerpiece of Gilead's $12 billion deal for developer Kite Pharma, was submitted for EU approval in August.

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Kymriah is a key piece of Novartis' oncology strategy as the company relies increasingly on cancer drugs for growth, and at a time when generics are taking a big bite out of its longtime blockbuster, Gleevec. It was the first in an expected wave of cell therapies that target deadly diseases in an entirely new way, by modifying a patients' own cells to fight them.

Others are on their way to market, however, and all are likely to bear very large price tags, presenting challenges to the companies and the payers that will foot the lion's share of the bill. Kymriah's U.S. list price is $475,000 in ALL, with a money-back guarantee, but Novartis has said it will price the drug differently for different indications, so its price in DLBCL in the U.S. could be lower. Yescarta's U.S. sticker is $373,000.

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In Europe, payer challenges will be different. Review for reimbursement tends to take longer, and winning coverage is a country-by-country affair. Negotiated prices tend to be far lower in European countries than they are in the U.S. But cancer is one field where cost-effectiveness gatekeepers have been under increasing pressure to make new meds available, and EU sales are a sizable portion of the blockbuster projections for the latest wave of cancer treatments.

Kymriah's EU application is partly based on data from the Juliet trial in DLBCL, which will be presented next month at the American Society of Hematology meeting. Top-line results revealed in June showed up to 43% of patients saw a complete response to the therapy, with an overall response rate of up to 51%.

"[T]he submission to the EMA is a major step toward our goal of delivering it to more critically ill cancer patients around the world," Novartis R&D chief and CEO-to-be Vas Narasimhan said in a Monday statement. "We look forward to working with the EMA to make CTL019 available to the children and adults who may benefit from this novel therapy."