CAR-T, FDA, New Products

FDA follows through with a groundbreaking OK for Yescarta, Gilead’s new CAR-T breakthrough

Just two weeks after Gilead closed on its $12 billion Kite buyout, the FDA has followed through with a groundbreaking approval of Yescarta (axi-cel), putting its CAR-T drug neck-and-neck with Novartis’ pioneer Kymriah as the two drugs are prepped for a launch.

In a virtual heartbeat, Gilead used its considerable cash reserves to reach a deal to buy out Kite and axi-cel 6 weeks ago, acquiring its newly approved CAR-T and all the next-gen technology now in the works. The acquisition made Gilead an overnight leader in CAR-T. While beaten to a historic first FDA approval for a CAR-T by an aggressive group at Novartis — initially green-lighted at the end of August for pediatric and young adult patients with a form of acute lymphoblastic leukemia — Gilead is retaining most of the Kite gang and honing its manufacturing effort, shaving off the time it takes to turn around these personalized therapies.

In classic aggressive form, Gilead came out gunning, offering the therapy at a price of $373,000 — $102,000 less than its rival at Novartis. On the other hand, Novartis offered a value-based deal to a number of payers in which they only get reimbursement for patients who respond to therapy, evening the playing field.

Once again, FDA commissioner Scott Gottlieb took the honors in congratulating the developers, as he did when Novartis came out in front. And this time, he promised to lend the agency’s help for the rest of the burgeoning cell therapy field. Said Gottlieb:

“Today marks another milestone in the development of a whole new scientific paradigm for the treatment of serious diseases. In just several decades, gene therapy has gone from being a promising concept to a practical solution to deadly and largely untreatable forms of cancer. This approval demonstrates the continued momentum of this promising new area of medicine and we’re committed to supporting and helping expedite the development of these products. We will soon release a comprehensive policy to address how we plan to support the development of cell-based regenerative medicine. That policy will also clarify how we will apply our expedited programs to breakthrough products that use CAR-T cells and other gene therapies. We remain committed to supporting the efficient development of safe and effective treatments that leverage these new scientific platforms.”

The CD19 T cell immunotherapy — which reengineers patient cells into a potent cancer therapy — has been approved for adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy. That label includes diffuse large B-cell lymphoma (DLBCL), primary mediastinal large B-cell lymphoma, high-grade B-cell lymphoma, and DLBCL arising from follicular lymphoma.

Alessandro Riva

The R&D work will now be led by Alessandro Riva, a former Novartis scientist promoted yesterday to executive vice president in charge of oncology R&D, with a seat on the leadership team.

Left behind in the race to CAR-T dominance, for now at least, is Juno Therapeutics. Its rival drug was mired down by a lethal toxicity issue that killed 5 patients, forcing the biotech to switch over to its number 2 program.

Novartis managed to surprise quite a few analysts by pegging Kymriah at $475,000, which is significantly lower than the $600,000 maximum provided in some of the spreads. In a story we published yesterday, however, a number of experts said the full cost of therapy may well end up at $1 million to $1.5 million.

The data, though, are jaw dropping.

The FDA’s approval comes through on the data for ZUMA-1, which demonstrated that 72% of the patients involved had an objective response rate to the therapy. And 51% demonstrated a complete remission, with no sign of the cancer left.

In a recent farewell letter to the staff, Kite CEO Arie Belldegrun summed it up by writing:

In a span of just a few short years, we grew from fewer than 10 employees to almost 700. The company’s value increased 2300% from the time of our IPO to nearly $12 billion with the acquisition by Gilead Sciences. Our closing $180 per share price represents not just a 960% appreciation from the IPO price of $17 per share, but the largest ever pre-commercial biopharma acquisition.

Now Gilead can see if it’s aggressive team can make the most of the blockbuster peak sales estimates that have yet to be climbed.

Today, Belldegrun had this to say:

“The FDA approval of Yescarta is a landmark for patients with relapsed or refractory large B-cell lymphoma. This approval would not have been possible without the courageous commitment of patients and clinicians, as well as the ongoing dedication of Kite’s employees. We must also recognize the FDA for their ability to embrace and support transformational new technologies that treat life-threatening illnesses. We believe this is only the beginning for CAR T therapies.”



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