Imbruvica: Creating a new standard for leukemia therapy

Project name: ibrutinib
Disease: Cancer
Peak sales estimate: $6 billion
Approved: Nov. 13
Companies: Pharmacyclics and Johnson & Johnson

The Scoop:
A year ago, Pharmacyclics ($PCYC) and J&J ($JNJ) were among the first of the biopharma companies to be rewarded with the FDA's new breakthrough therapy designation for ibrutinib--on three different oncology indications. And in November the agency came through with an early approval for one of the most closely watched applications of the year for a new drug that has earned a steadily rising forecast for peak sales.

The therapy blocks a key enzyme involved in the growth of cancer and helped control the cases of 68% of 116 mantle cell lymphoma patients--with a relatively improved safety profile among treatment-resistant patients. The data have driven Pharmacyclics' shares to considerable heights, making CEO Robert Duggan a paper billionaire in the process.

J&J stepped in late in 2011, providing a rich $975 million deal to collaborate on development and marketing. The move demonstrated yet again the pharma giant's expertise at executing a blockbuster deal at the right moment.

About a month after the FDA's first approval for ibrutinib, investigators pulled back the results from a 27-month extension study of leukemia patients which helped illustrate this drug's potential. Virtually all of the treatment-naïve patients in the study continued to benefit from progression-free cases, with roughly three-quarters of the treatment-resistant patients seeing similar results.

Researchers told colleagues that about half of all leukemia patients would typically see disease progression after two years, making ibrutinib a standout. And with an absence of severe side effects, it appears to be the kind of drug that patients can take over a prolonged period. -- John Carroll (email | Twitter)

For more:
Special Reports: 10 top drugs in biopharma's late-stage pipeline - Ibrutinib | Top  20 Orphan Drugs by 2018 - Ibrutinib | Fierce's 10 top biotech billionaires - Robert Duggan
J&J, Pharmacyclics win early approval for 'breakthrough' cancer drug ibrutinib
J&J, Pharmacyclics slap $130,000 price on Imbruvica for rare lymphoma
'Breakthrough' ibrutinib NDA makes rapid arrival at FDA

Imbruvica: Creating a new standard for leukemia therapy

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