Seeking to expand CAR-T's reach, Novartis snags new regulatory milestones for Kymriah

Novartis' Kymriah picked up new regulatory milestones from the FDA and EMA. (Novartis)

Even as Celgene reportedly considers buying CAR-T player Juno Therapeutics, Novartis has its own plans to keep a leadership position in the field. On Wednesday, the drugmaker announced that Kymriah won regulatory milestones on both sides of the Atlantic as it seeks to grow its prospects with the cutting-edge technology.

CAR-T drug Kymriah picked up an FDA priority review in relapsed/refractory diffuse large B-cell lymphoma (r/r DLBCL) for adults who can't have an autologous stem cell transplant or for those who relapse after the procedure, the drugmaker announced. In Europe, regulators granted an accelerated assessment for children and adults with r/r B-cell acute lymphoblastic leukemia (r/r ALL) and for adults with r/r DLBCL.

Novartis won the world's first CAR-T nod in August for Kymriah to treat r/r ALL patients up to 25 years old. CAR-T drugs are one-time treatments made of cells that are collected from each patient, re-engineered and then infused back into the patient to attack cancer. 

RELATED: Novartis seeks Kymriah nod in lymphoma, setting up Gilead CAR-T showdown 

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Since Novartis' FDA nod, Gilead's Yescarta picked up an approval to treat adults with certain types of large B-cell lymphoma, including r/r DLBCL. Gilead entered the field through its $12 billion Kite Pharma buyout in August. 

If Novartis does end up winning a nod in r/r DLBCL, the companies will be in a direct market showdown. The Swiss drugmaker applied for approval for the indication in October, shortly after Gilead's nod.

Novartis has said it will price differently in each indication; in r/r ALL, its product costs $475,000 and has a money-back guarantee for certain patients who don't respond. Gilead's Yescarta costs $373,000 for a one-time treatment. Its drug is currently approved for a larger group of patients.

RELATED: Juno jumps afterhours as reports suggest Celgene wants a buyout 

It won't always be the two drugmakers in direct competition, of course. With its eyes on the market, Celgene is reportedly considering purchasing Juno Therapeutics, a biotech that once held a leadership position in CAR-T before safety issues set it back.

A host of other players are active in the field as regulators and payers seek to keep up with the groundbreaking technology. In gene therapy, Spark Therapeutics recently won the first U.S. approval, Luxturna, which can cure an inherited disease that can lead to blindness.

RELATED: Payers point to Spark’s gene therapy as a model for innovative pricing plans

Luxturna costs $850,000 for two eyes, but the drugmaker rolled out a three-pronged strategy to help make its drug more affordable. It's offering rebates based on the drug's effectiveness and direct contracts with commercial payers rather than treatment centers. Spark is also working with the Centers for Medicare & Medicaid Services on ways to collect payments over multiple years.