Incyte ($INCY) has won an accelerated FDA approval for ruxolitinib (originally dubbed INC424), its JAK1 and Jak2 inhibitor for myelofibrosis, a potentially lethal though rare bone marrow disease. That's also good news for Novartis ($NVS), which holds ex-U.S. rights and believes the treatment is on a short path to blockbuster status.
Ruxolitinib, which will be sold as Jakafi, has been in the clinic for the past four years, including time spent on two late-stage studies. Another late-stage trial is under way for advanced polycythemia vera, a program also partnered with Novartis. Add it all up, and Helvea analysts see $660 million in peak sales. But Novartis, which has considerable experience in winning niche approvals and then swiftly adding on new indications that broaden the market potential, has listed the treatment as one of its top drug prospects.
For the FDA, the treatment is an example of how a better understanding of molecular pathways can earn an approval after a relatively quick clinical program.
"Jakafi represents another example of an increasing trend in oncology where a detailed scientific understanding of the mechanisms of a disease allows a drug to be directed toward specific molecular pathways," said Dr. Richard Pazdur, director of the Office of Hematology and Oncology Products in the FDA's Center for Drug Evaluation and Research. "The clinical trials leading to this approval focused on problems that patients with myelofibrosis commonly encounter, including enlarged spleens and pain."
The treatment belongs to a drug class that has tremendous market potential. Pfizer ($PFE) has been advancing its JAK inhibitor, tofacitinib, for the blockbuster rheumatoid arthritis market.
- here's the press release
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