[Corrected 9/5/17, 12:22 p.m. See below.] The far-out idea of reprogramming a patient’s immune cells to fight cancer, known as CAR-T, entered the mainstream this week, and two numbers highlight the tale. The first is $11.9 billion, the price Gilead Sciences is paying for Kite Pharma and its cancer-fighting cell therapy pipeline. It’s one of biopharma’s biggest deals in recent years and a massive bet that Gilead can build a cancer business, starting with, but not limited to, Kite’s nearest-term target of non-Hodgkin lymphoma.
The second number is $475,000, which Novartis intends to charge for its cell therapy for pediatric leukemia. The FDA approved it this week ahead of schedule. That wasn’t a surprise, thanks to a unanimous FDA advisory vote in July, but the cost of treatment, and Novartis’s unusual pledge not to collect cash if it doesn’t work initially, has kicked CAR-T squarely into the national debate over drug prices.
We also send our best wishes to Houston and other parts of southeast Texas. We’ll all pull together to help, even in these divisive times. If you’re wondering where to start, here’s a good guide.
A shout-out as well to our Texas editor Angela Shah, based in Houston and undaunted. She has been covering the local tech community during the storm. You can follow her coverage here.
For the CAR-T news and more, let’s get to the roundup.
—The FDA approved tisagenlecleucel (Kymriah), the first CAR-T cell therapy to pass regulatory muster. The treatment for kids with severe cases of acute lymphoblastic leukemia will cost $475,000, said its owner Novartis (NYSE: NVS). But Novartis pledged not to charge a dime to patients whose treatment doesn’t help within a month. The price was lower than industry analysts expected, but higher than some patient advocates felt was necessary.
—Why the one-month “no charge” threshold? Novartis said it wanted to stick to the data that the FDA used for its approval. But there is no rule saying drug prices must be set according to a drug’s label.
—Matthew Herper of Forbes described the Kymriah price tag as a Solomonic choice, as if Novartis had to forge a compromise that would not likely appease anyone. CEO Joe Jimenez told Herper that Novartis spent more than $1 billion bringing the complex cell therapy to market.
—The next CAR-T likely to gain approval will come from Gilead Sciences (NASDAQ: GILD). That’s because Foster City, CA-based Gilead is buying the CAR-T’s owner, Kite Pharma (NASDAQ: KITE) of Santa Monica, CA, for $11.9 billion. Gilead officials said the therapy, nicknamed axi-cel, would be the foundation of its nascent cancer business.
—The crowded field laid to waste one prospective CAR-T competitor this week. Johnson & Johnson (NYSE: JNJ) ended a partnership with Macrogenics (NASDAQ: MGNX) , of Rockville, MD, on an early-stage antibody drug, duvortuxizumab, that J&J paid $125 million to license in 2014. It was a candidate to treat some of the blood cancers that the first wave of CAR-T therapies have gone after. Macrogenics is scrapping the drug, citing neurological side effects and too much competition from CAR-T. [This paragraph has been changed to clarify the description of duvortuxizumab.]
FROM THE REGULATION REALM
—The FDA approved benznidazole, the first U.S. treatment for Chagas disease, a parasitic infection. Because it is the first drug approved in the U.S. for a neglected tropical disease, the developer, a consortium of for-profit and nonprofit entities, receives a valuable voucher. The consortium has pledged to put half the revenues from the voucher into its non-profit work. Martin Shkreli once had a plan to develop the same drug.
—Four years after the FDA rejected Aveo Oncology’s tivozanib (Fotivda), European regulators approved the treatment for kidney cancer patients. Aveo is eyeing a comeback in the U.S. as well, with plans to file for approval should a Phase 3 study succeed next year.
—The Medicines Co. won FDA approval of meropenem/vaborbactam (Vabomere) for complicated urinary tract infections. It has yet to announce a price.
—Merck’s (NYSE: MRK) cholesterol-lowering drug anacetrapib met its main goal in a 30,449-patient study called “Reveal,” but the New Jersey drug maker did not say if it would seek FDA approval. It could be the end of a class of drugs called CETP inhibitors, once seen as potential breakthrough heart medicines, if Merck doesn’t move anacetrapib forward.
—Eli Lilly (NYSE: LLY) of Indianapolis plans to reapply for FDA approval for rheumatoid arthritis drug baricitinib (Olumiant) by the end of January. Lilly said in July that the FDA’s safety concerns could take … Next Page »