Exclusive Life Science Feature
Big Pharma
Bets Big On
Breakthroughs
J&J;Õs Approach To Drug Discovery
By Rob Wright, chief editor
O
ne year ago, the Food and Drug Administration Safety
and Innovation Act (FDASIA) was signed into law. Its
most current version includes a provision that allows sponsor companies to request their drug be designated as a
"breakthrough therapy." In February, Johnson & Johnson
(J&J;) became only the second company (behind Vertex
Pharmaceuticals) to have a drug receive this designation.
The drug, ibrutinib, is an oral Bruton's tyrosine kinase inhibitor. Thus far, ibrutinib has
garnered three breakthrough designations (mantle cell lymphoma, Waldenström's macroglobulinemia, and a subset of patients with chronic lymphocytic leukemia who have a particularly poor prognosis) of the more than 40 requests the FDA has received. In addition
to this success, J&J; recently won accelerated FDA approval for Sirturo (bedaquiline), the
first drug with a new MOA (mechanism of action) for tuberculosis (TB) to be approved in
40 years. It seems J&J;'s pharmaceutical business is on a roll.
Driven by its R&D; engine, Janssen Research & Development, J&J; has been capitalizing
on FDA initiatives that not only incentivize drug innovation but also reward successful
companies. Bill Hait, M.D., Ph.D., is the global head of Janssen R&D.; He explains some
of the company's approaches to drug discovery that have resulted in J&J; recently being
ranked by Forbes as the most productive drug firm in the last 10 years.
FOCUS ON UNMET MEDICAL NEED CAN OFFER BIG WINS
It might seem clichéd in drug discovery to say, "Focus on the unmet medical need as
opposed to commercial viability." But being altruistic also can lead to big dividends.
For example, in 1998 Remicade (infliximab) was the first drug specifically approved for
patients with moderate to severe Crohn's disease, a bowel ailment that afflicts approximately 500,000 Americans. This equates to .16% of the U.S. population. At the time, there
were conventional, less-expensive treatments available, including steroids and antibiotics.
If Centocor (now Janssen) had not pursued approval of this drug based on the relatively
small commercial market (designated fast-track, orphan drug status, priority review),
the company would have missed out on significant sales revenue, including $6.1 billion
July 2013
LifeScienceLeader.com
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