J&J nabs an early OK for 'breakthrough' multiple myeloma blockbuster contender Darzalex

CDER's Richard Pazdur

Johnson & Johnson ($JNJ) scored an early FDA approval for its "breakthrough" blockbuster contender daratumumab, earning a swift OK as a new treatment for multiple myeloma four months ahead of the PDUFA date. The drug will be marketed as Darzalex.

The pharma giant signed up for a $1.1 billion deal to gain rights to the drug from Genmab, which also benefits considerably from today's marketing approval. An early winner of breakthrough drug status at the FDA, J&J will now get a jump on Bristol-Myers Squibb ($BMY) and AbbVie ($ABBV), which have another "breakthrough" multiple myeloma drug, elotuzumab, in late-stage development.

The approval comes hard on the heels of another early cancer drug approval, with AstraZeneca ($AZN) picking up an early OK on AZD9291, to be sold as Tagrisso. The FDA, and particularly the oncology group inside the agency, have been lopping months off the approval timeline for new therapies.

"Targeting proteins that are found on the surface of cancer cells has led to the development of important oncology treatments," said Dr. Richard Pazdur, director of the Office of Hematology and Oncology Products in FDA's Center for Drug Evaluation and Research. "Darzalex provides another treatment option for patients with multiple myeloma who have become resistant to other therapies."

Daratumumab is given once a week for the first two months of treatment, then the infusions wind down to once monthly in the second 6 months of the year, says a spokesperson for J&J. For the first full year--with a total of 23 doses given at a cost of $5,850 per infusion--the average monthly wholesale acquisition cost is $11,212, says J&J, or $135,550 for the first year. In year two, and any year thereafter, patients receive a total of 13 doses, for a monthly wholesale cost of $6,337, or $76,044 annually. J&J also offers programs to limit patient's out-of-pocket costs and will be offering payers discounts during price negotiations.

Focusing on the group of patients getting the top dose, investigators reported in The New England Journal of Medicine last August that the drug scored an impressive 36% overall response rate among 42 patients with late-stage multiple myeloma--a step up from the 29% overall rate that was reported at ASCO earlier in the year. And two-thirds of the responder group benefited from progression-free survival after 12 months of therapy. The dose-escalating study also satisfied researchers that they would be able to use the 16-mg dose for all future studies of the drug, an IgG1k antibody that binds to CD38 on the surface of multiple myeloma cells.

Daratumumab has excited a range of analysts happy to roll out multibillion-dollar projections for this drug. Kyprolis won an approval for Onyx with a 23% ORR, and then was bought out by Amgen ($AMGN), which recently scored a new combo approval for relapsed cases, looking for more of an edge in its fight to steal market share from Celgene's ($CELG) Pomalyst.

- here's the release

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