Novartis building out cell and gene therapy platform that'll lead the pack, CEO promises

Novartis' first-ever CAR-T cell therapy may have been hamstrung by early manufacturing problems, but the company still figures it can build a cell and gene therapy platform strong enough to deliver success not only with Kymriah but also a full "game board" of treatments for many more diseases, its CEO said on Monday.

During his first J.P. Morgan Healthcare Conference presentation, Novartis chief Vas Narasimhan, M.D., contended that his company's work to establish capabilities in cell and gene therapies now will pay off in the long run.

The company is partnering with groups around the world to produce its cell therapy Kymriah, a complicated process that the Big Pharma has struggled with early on. And in gene therapy, Novartis scooped up AveXis last year for $8.7 billion and teamed up with Spark Therapeutics on ex-U.S. launches for blindness drug Luxturna.

Then there's the commercial side. One particular challenge for gene therapies—pricing and striking innovative deals with payers—is a key focus for the drugmaker. The cutting-edge treatments will be far more pricey than even the most expensive rare disease drugs now on the market.

Consider its spinal muscular atrophy gene therapy candidate Zolgensma, acquired in the AveXis buyout. It's a "potential foundational medicine," Novartis argues, and Narasimhan said nearly all parents and providers would want the drug for their patients if it's approved. Complicating that, of course, is price. According to cost watchdogs at ICER, the drug could be cost-effective at a price of $1.6 million to $5.4 million, the helmsman pointed out.

To support a price tag that high, Novartis is "working very hard on novel approaches to enable healthcare systems to manage the spend appropriately over time," Narasimhan said. 

Meanwhile, the company is also exploring radio-drug conjugates across a "full range of solid tumors," according to the company's presentation. Novartis built out its portfolio in that field last year with the buyout of Advanced Accelerator Applications and has since nabbed an FDA approval for that company's Lutathera, which has racked up solid early sales.

In 2017, Novartis became the first drugmaker to win an approval globally for a cell therapy—Kymriah—and has since been able to broaden its approved uses to set up a competition with Gilead Sciences’ second-to-market Yescarta. So far in the rollout, Novartis has suffered a manufacturing setback that it’s working to overcome. Since the Kymriah and Yescarta approvals, Spark Therapeutics has also won an FDA nod for blindness gene therapy Luxturna. 

CAR-T drugs—made of T cells that are collected from each patient, modified and then infused back into the patient to attack cancer—provide the potential for a one-time treatment and cure for patients with few other options. While cell and gene therapies now boast limited approved uses, Narasimhan said he believes the classes will have broad applications in the long run.