It's been a long road to FDA approval for Jazz Pharmaceuticals' ($JAZZ) Defitelio. But the Irish biotech is finally ready to launch the product--and a surprise on the product's label may help make that effort a success.
U.S. regulators tagged Defitelio with a longer-than-expected maximum number of days of therapy when they Wednesday approved the product to treat patients who develop hepatic veno-occlusive disease (VOD) with additional kidney or lung abnormalities after a hematopoietic stem-cell transplantation procedure, Leerink Partners analyst Jason Gerberry wrote in a note to clients. And that high ceiling "may translate to revenue upside," he pointed out.
Jazz's hefty price on the product won't hurt in that department, either, though as Gerberry noted, the $825 per vial--which comes out to about $140,000 per treatment course, based on an average number of vials consumed by the anticipated mix of adults and children--is more or less in line with what analysts expected.
Analysts expect to see Jazz roll out the product immediately. It's the first FDA-approved med for severe hepatic VOD, a life-threatening liver condition; of 20,000 stem cell transplantation recipients in the U.S. each year, about 10% suffer from the condition.
But don't expect to see things take off right away, Gerberry cautioned investors. He predicts initial uptake will be faster in children, and those sales will generate less revenue: Defitelio is priced per vial with weight-based dosing, and pediatric patients weight less than their adult counterparts.
Meanwhile, across the pond, Defitelio is already rolling; last year, it raked in $70.7 million in European sales.
- read the release
Special Report: Top 20 orphan drugs by 2018