The FDA has recommended a follow-up analysis, but has granted conditional approval.
More than two years after the launch of a program that aims to help patients fight life-threatening diseases, the FDA has approved as safe and effective two drugs that boost patient white blood cell counts. The FDA’s C.D.C. panel of experts voted 7-2 on Thursday to endorse the use of Moderna Therapeutics Inc.’s RNAi gene therapy and four years into the non-profit CAR-T Therapy Initiative’s efforts to improve a group of therapies known as chimeric antigen receptor T cells (CAR-T).
The FDA had set a 30-day public comment period for its decision, and it will eventually make a final decision about whether to approve the drugs. The decision comes a day after Celgene Corp. announced that the FDA had accepted the company’s request to review its drug for use in prostate cancer, called GED-0301.
The C.D.C. meeting considered whether each of these therapies ought to be designated so that they may be bought through the Medicaid program for the poor. Even though the program pays for cancer treatments, the status of the drugs in terms of their labeling, in the eyes of the health care systems that operate within it, will be significant.
C.D.C. Panel Endorses Moderna and J.&J. Boosters for Millions
The panel’s members were quite unanimous in their endorsement of these particular therapies, in terms of their safety profiles. In light of that consensus, they approved only one condition: because of the stocks of the components used in the drugs, that the FDA conduct a review of material from the different manufacturers. Should that same manufacturing process use represent a direct threat to the health of people, the FDA will continue to refer to one particular manufacturer by its generic name, “Centriq.”
While the C.D.C. panel ultimately agreed that both of these drugs could be authorized to treat two very rare cancers — hematological malignancies and non-Hodgkin lymphoma — they found that only Moderna’s therapy for leukemia was likely to have value for those patients. The panel expressed concern that demand for the drug could create an “abundance of modern versions” of the therapy and drive down the prices. That worry, of course, has been somewhat alleviated by the fact that patients would not have to pay for the drug out of pocket.
Moderna’s share price jumped as high as 20 percent after the announcement, closing at $115.28. Shares of Kite Pharma Inc., which raised $1.6 billion in a public offering after being acquired by Johnson & Johnson, ticked up as well.
In the case of the J.&J. CAR-T, the patients who stand to benefit are generally people with blood cancers that had already run out of other treatment options, so the agency already knows that demand for the therapy is high. But the approval still counts for large sums for the companies involved. One analysis by EvaluatePharma estimated that the approval of GED-0301 could mean a win for the biopharmaceutical company of $5.3 billion over a decade.
Also on hand Thursday to talk to the C.D.C. panel were the representatives of manufacturing companies of these two therapies. Each business had a chance to argue that their drugs should be licensed, and, if not licensed, why not a different one, of the same quality and effectiveness. The panel heard a presentation from the third manufacturer, Novartis. Each company presented different manufacturers for its CAR-T drugs, and each represented its own qualities and strengths. Still, both presenters seemed pleased with the outcome of the vote and promised to work with the FDA in its review process.
Dr. Theresa Eisenman, chair of the C.D.C. panel, expressed satisfaction at the way the entire process played out. “At the end of the day, the FDA listened to the panel and accepted the recommendations made by the panel,” she said in a statement.
