Jakavi

Incyte/Novartis
Projected 2018 sales:
$1.559 billion
2012 sales: $150 million

Jakavi, like many rare-disease drugs, is expensive--and, like many expensive drugs, has failed to get the go-ahead from the U.K.'s cost-effectiveness watchdogs, the National Institute for Health and Care Excellence. In June, the institute ruled Jakavi--a JAK1 and JAK2 inhibitor approved for the treatment of symptoms in adults with primary myelofibrosis, a bone-marrow disorder--unworthy of its $5,000-per-month pricetag.

Jakavi is approved in the EU, however, where it has been used to treat the rare blood cancer since getting the OK from regulators in August of last year. Novartis' ($NVS) drug was the first of its kind, as myelofibrosis affects fewer than 1 in 100,000 people a year. But as is often the case with orphan drugs, its costliness helps offset its small market.

Jakavi is just a piece of Novartis' R&D success as of late. CEO Joe Jimenez has been lauded for the deep oncology pipeline that has helped Novartis avoid deep research cuts that have hit fellow pharma giants Pfizer ($PFE) and AstraZeneca ($AZN).

For more:
U.K. gatekeepers shut out Novartis cancer treatment

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Jakavi